| Crizanlizumab Improves Tissue Oxygen Supply Demand Matching in Patients With Sickle Cell Anemia | Children's Hospital Los Angeles | | 1 Jul 2023 | 1 Jul 2028 |
| Feasibility and Preliminary Efficacy of Acceptance and Commitment Therapy (ACT) for Sleep Disturbances in Adults With Sickle Cell Disease (SCD) | National Cancer Institute (NCI) | Insomnia, Pain, Nonpharmacological, Actigraph, Anemia, Sleeplessness | 17 Aug 2022 | 31 Jul 2024 |
| Correlation Between Pulmonary Functions and Physical Fitness in Children With β-thalassemia | Cairo University | | 1 Aug 2022 | 1 Dec 2022 |
| BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease | Beam Therapeutics Inc. | Gene Editing, Sickle Cell, Severe Sickle Cell | 1 Aug 2022 | 1 Feb 2027 |
| Acceptability of a New Paediatric Formulation of Hydroxycarbamide in Children With Sickle Cell Disease. | ADDMEDICA SASA | hydroxycarbamide, pediatric, acceptability | 8 Jul 2022 | 1 Nov 2022 |
| Study on the Effect of Different Risk Factors on the Growth Parameters of Thalassemic Patients in Assiut (AUCH) | Assiut University | | 2 Jul 2022 | 2 Mar 2024 |
| Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease | Vertex Pharmaceuticals Incorporated | | 1 Jul 2022 | 1 Feb 2025 |
| The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study | Sun Yat-sen University | | 1 Jul 2022 | 30 Apr 2023 |
| Research Study Investigating How Well NDec Works in People With Sickle Cell Disease | Novo Nordisk A/S | | 27 Jun 2022 | 29 Mar 2024 |
| A Phase 2/3 Study in Adult and Pediatric Participants With Sickle Cell Disease (SCD) | Global Blood Therapeutics | | 1 Jun 2022 | 1 Apr 2027 |
| Cognitive Remediation Intervention to Prepare for Transition of Care | University of Alabama at Birmingham | | 1 May 2022 | 30 Apr 2027 |
| Inhibitory Effect of a Polyphenol Supplement on Dietary Iron Absorption in Adults With Thalassemia | Swiss Federal Institute of Technology | Iron overload, Thalassemia, Polyphenols, Iron absorption | 1 May 2022 | 31 Dec 2022 |
| Study of Crizanlizumab for Prevention of Silent Cerebral Infarcts in SCA | Andria Ford | | 11 Apr 2022 | 1 Jul 2025 |
| The Montreal Cognitive Assessment.Test in Adults With Sickle Cell Disease | Assistance Publique - Hôpitaux de Paris | Sickle cell anemia, cognitive dysfunction, sensitivity and specificity, Diagnostic Techniques and Procedures, Neuropsychological Tests, Montreal cognitive assessment | 1 Apr 2022 | 1 May 2023 |
| Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) | Vertex Pharmaceuticals Incorporated | | 1 Apr 2022 | 1 May 2026 |
| A Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants With Sickle Cell Disease | Global Blood Therapeutics | blood disorders, hemoglobin, red blood cells, RBCs, sickle-like shape, mutation in hemoglobin gene, Sickle Cell Disease, SCD, Vaso-occlusive Crisis, VOC, SCA, Open Label | 29 Mar 2022 | 1 Mar 2027 |
| Thal-Fabs: Reduced Toxicity Conditioning for High Risk Thalassemia | The Hospital for Sick Children | thalassemia, reduced toxicity, abatacept, sirolimus, pre transplant immunosuppression | 22 Mar 2022 | 31 Dec 2026 |
| The Predictive Capacity of Machine Learning Models for Progressive Kidney Disease in Individuals With Sickle Cell Anemia | University of Tennessee | Machine Learning Models, Sickle Cell Disease, Chronic Kidney Disease, eGFR, Anemia, Sickle Cell, Albuminuria, Renal Insufficiency, Chronic, Renal Insufficiency, APOL1 | 15 Mar 2022 | 31 Jan 2026 |
| A Randomized Controlled Double-Blind Trial for Prevention of Recurrent Ischemic Priapism in Men With Sickle Cell Disease: A Pilot Study | Vanderbilt University Medical Center | | 1 Mar 2022 | 1 Jan 2027 |
| Sickle Cell Disease Obstetric Multi-Disciplinary Care Programme: Prospective Multi-Centre Cohort Study | Vanderbilt University Medical Center | | 1 Mar 2022 | 1 Jan 2026 |
| Cooperative Assessment of Late Effects for SCD Curative Therapies | Vanderbilt University Medical Center | Myeloablative Autologous Gene Editing, Myeloablative Autologous Gene Therapy, Myeloablative allo-HSCT, Nonmyeloablative allo-HSCT, Disease-Modifying Therapy | 1 Mar 2022 | 1 Feb 2026 |
| Cutaneous Hydration Assessment in SCD | Enrico M Novelli | | 1 Mar 2022 | 31 Oct 2024 |
| SCD Fit Homebase Program | University of Alabama at Birmingham | | 1 Mar 2022 | 1 Jan 2023 |
| A Study to Evaluate an Electronic Patient Diary in Adult Participants With Sickle Cell Disease | Pfizer | Vaso-occlusive crisis (VOC), Hemoglobin S (HbS) | 10 Feb 2022 | 26 Jul 2023 |
| Observational Study to Deeply Phenotype Major Organs in Sickle Cell Disease After Curative Therapies | National Heart, Lung, and Blood Institute (NHLBI) | Hydroxyurea, Hydroxyurea-Increased Fetal Hemoglobin, sickle cell anemi, Patterns of mortality in sickle cell disease, Mortality rates and age at death from sickle cell, Natural History | 3 Feb 2022 | 1 Jun 2035 |
| Screening for Renal Complications in Children and Young Adults With Major Sickle Cell Disease | Centre Hospitalier Universitaire de Nice | Sickle cell disease, renal complication | 1 Feb 2022 | 1 Feb 2024 |
| Nasopharyngeal Bacterial Carriage and Antibiotic Resistance in Children With Sickle Cell Disease in Ile-De-France | Assistance Publique - Hôpitaux de Paris | Sickle celle disease, Nasopharyngeal bacterial carriage | 1 Feb 2022 | 1 Feb 2023 |
| Voxelotor Cerebral Hemodynamics Study | Global Blood Therapeutics | | 1 Feb 2022 | 1 Apr 2025 |
| Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients | Westat | Transfusion, Red Blood Cell, Sickle Cell Disease, Thalassemia, Pediatric, Oncology, RBC survival, Genetic, Non-genetic, Observational, Blood donor | 1 Feb 2022 | 1 Mar 2025 |
| Voxelotor Neurocognitive Function Study | Global Blood Therapeutics | | 1 Feb 2022 | 1 Oct 2023 |
| Hemolysis Related Complications in SCD. A Phase II Study With Voxelotor | Assistance Publique - Hôpitaux de Paris | Sickle Cell Disease, Hemolysis, Plasma Hemoglobin | 15 Jan 2022 | 15 Jan 2024 |
| Assessment of Cognitive Function, Fatigue and Health Related Quality of Life in Children With Beta Thalassemia | Cairo University | | 15 Jan 2022 | 30 Dec 2022 |
| The Prevelence of HBB c.93-21 G-A in β Thalassemia Patients | Assiut University | | 1 Jan 2022 | 1 Jun 2024 |
| Assessment of Cognitive Functions, Psychological Symptoms, and Quality of Life Among B Thalassemia Patients and Their Clinical Correlates. | Assiut University | | 20 Dec 2021 | 20 Feb 2023 |
| An Observational Long-term Safety and Efficacy Follow-up Study After Ex-vivo Gene Therapy With BIVV003 in Severe Sickle Cell Disease (SCD) and ST-400 in Transfusion-dependent Beta-thalassemia (TDT) With Autologous Hematopoietic Stem Cell Transplant | Bioverativ, a Sanofi company | | 20 Dec 2021 | 13 Mar 2043 |
| A Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab as Adjunct Treatment in Prevention of Vaso-Occlusive Episodes (VOE) in Sickle Cell Disease (SCD) | Hoffmann-La Roche | Vaso-occlusive episodes, Pain crisis | 17 Dec 2021 | 10 Jan 2025 |
| Severe Acute Respiratory Syndrome CoV 2 COVID-19 Survey and Vaccination Coverage in the Sickle Cell Population in Ile-De-France | Assistance Publique - Hôpitaux de Paris | seroprevalence, SARS-CoV-2, sickle cell | 10 Dec 2021 | 10 Sep 2022 |
| A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease | Agios Pharmaceuticals, Inc. | | 1 Dec 2021 | 1 Jan 2030 |
| Interest of Famotidine in Children With Sickle Cell Disease | Assistance Publique - Hôpitaux de Paris | Sickle cell disease, Famotidine, Histamine, P-selectin, Vaso-occlusive crisis | 1 Dec 2021 | 1 Jul 2023 |
| COVID-19 Vaccine Response in Sickle Cell Disease | ASH Research Collaborative | | 1 Dec 2021 | 31 Dec 2022 |
| L-Arginine in Children Having Sickle Cell Disease With Increased Tricuspid Regurgitant Jet Velocity | Tanta University | | 25 Nov 2021 | 1 Sep 2024 |
| Primary Prevention of Stroke in Children With Sickle Cell Anaemia in Nigeria in the Community | Barau Dikko Teaching Hospital | Sickle Cell Anaemia, Stroke, Transcranial Doppler Ultrasound, Hydroxyurea, Task-shifting | 6 Nov 2021 | 1 Jul 2025 |
| THromboprophylaxis In Sickle Cell Disease With Central Venous Catheters (THIS) | Kevin H.M. Kuo, MD, MSc, FRCPC | | 1 Nov 2021 | 1 Mar 2024 |
| Pharmacokinetics and Pharmacodynamics of Rifaximin Novel Formulations in Patients With Sickle Cell Disease | Bausch Health Americas, Inc. | | 1 Nov 2021 | 1 Jan 2023 |
| Telemedicine for Children With Sickle Cell Disease | Indiana University | | 1 Nov 2021 | 1 Sep 2025 |
| A Study of FT-4202 in Patients With Thalassemia or Sickle Cell Disease | Forma Therapeutics, Inc. | SCD, sickle cell disease, sickle cell, anemia, sickle cell anemia, hemolytic, hemoglobin, vaso-occlusive crisis, VOC, vaso-occlusive events, sickle cell crisis, pain crisis, pain episode, congenital anemia, hemolytic anemia, hematologic disease, hemoglobinopathy, hemoglobinopathies, genetic disease, inborn disease, sickle cell trait, pyruvate kinase, PKR, thalassemia, beta-thalassemia, alpha-thalassemia, transfusions, hemoglobin H, transfusion, transfusion-dependent, non-transfusion dependent, hemoglobin E | 30 Sep 2021 | 31 Jul 2025 |
| Efficacy of Transdermal Microneedle Patch for Topical Anesthesia Enhancement in Paediatric Thalassemia Patients | Universiti Kebangsaan Malaysia Medical Centre | Microneedle, Maltose Microneedle, Thalassemia in Children, Paediatric Thalassemia, Transdermal microneedle patch, EMLA, Intravenous cannulation, Blood transfusion, Intravenous line insertion | 15 Sep 2021 | 11 Aug 2022 |
| Thrombin Generation in Beta-thalassemia Major | Central Hospital, Nancy, France | Beta Thalassemia Major; hypercoagulability; thrombin generation test | 1 Sep 2021 | 1 Sep 2024 |
| Functional and Mechanistic Characterization of Limb Ulcers in Patients With Sickle Cell Disease | Hospices Civils de Lyon | limb ulcers, sickle cell disease, microcirculation, cytokine, wound fluid | 1 Sep 2021 | 1 Jan 2024 |
| Long-term Comparative Cerebrovascular Outcome After Transplantation vs Standard Care in Sickle Cell Anemia | Centre Hospitalier Intercommunal Creteil | transplantation | 1 Sep 2021 | 1 May 2025 |
| Characteristics of Patients With Sickle Cell Disease | Novartis Pharmaceuticals | Crizanlizumab,, sickle cell disease | 30 Aug 2021 | 27 Oct 2021 |
| Study to Evaluate Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs) | Celgene | REBLOZYL, Myelodysplastic Syndromes, Healthcare Professionals, Post-authorization safety study, Additional educational measures, Beta-thalassemia, Luspatercept | 20 Jul 2021 | 30 Sep 2022 |
| Integrative Medicine in Pain Management in Sickle Cell Disease | Indiana University | Sickle cell disease, Pain, Acupuncture, Quantitative Sensory Testing, Magnetic Resonance Imaging, Circulating Biomarkers, Electroencephalography, Functional near-infrared spectroscopy | 29 Jun 2021 | 31 May 2026 |
| Preservation and Transfer of HBV Immunity After Allogeneic HSCT for SCD. | Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) | Sickle Cell Disease, Allogeneic stem cell transplantation, Mixed chimerism, Preservation of immunity, Adoptive transfer of immunity, Hepatitis B virus vaccination | 8 Jun 2021 | 1 Dec 2024 |
| A Study to Evaluate GBT021601-012 Single Dose and Multiple Dose in Participants With Sickle Cell Disease (SCD) | Global Blood Therapeutics | | 28 May 2021 | 30 Sep 2021 |
| Designing an Implementation Strategy for Delivering Routine Mental Health Screening and Treatment | University of Pittsburgh | | 1 May 2021 | 1 Sep 2022 |
| Moya Moya Syndrome With or Withtout Sickle Cell Disease | University Hospital, Montpellier | Moya Moya syndrome, Moya Moya | 1 May 2021 | 1 Dec 2022 |
| Avascular Bone Necrosis in Sickle Cell Disease: a Pediatric Study. | San Luigi Gonzaga Hospital | Avascular necrosis | 18 Mar 2021 | 27 Oct 2021 |
| The Role of Oxygen Reserve in Brain Growth | Washington University School of Medicine | sickle cell anemia, cerebrovascular reactivity, cerebral oxygen metabolism, cortical thickness, gray matter, carbon dioxide, magnetic resonance imaging, brain development | 1 Mar 2021 | 31 Mar 2026 |
| Association of Proteinuria and Progression of Kidney Dysfunction in Sickle Cell Disease (CSEG101A0FR01) | Soutien aux Actions contre les Maladies du Globule Rouge | | 1 Mar 2021 | 1 Dec 2021 |
| A Long-term Follow-up Study in Subjects Who Received CTX001 | Vertex Pharmaceuticals Incorporated | | 1 Feb 2021 | 1 Sep 2039 |
| A Study to Evaluate GBT021601 in Single and Multiple Doses in Healthy Participants | Global Blood Therapeutics | | 9 Dec 2020 | 1 Apr 2022 |
| SELF-BREATHE RCT for Chronic Breathlessness | King's College Hospital NHS Trust | | 3 Dec 2020 | 30 Jun 2022 |
| US Phase I Study of ECT-001-CB in Patients With Sickle-Cell Disease | ExCellThera inc. | | 1 Dec 2020 | 2 Feb 2024 |
| Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients | BGI-research | β-thalassemia major, β-globin restoration, autologous HSCT | 1 Nov 2020 | 30 Nov 2024 |
| COVID-19 Testing in Underserved and Vulnerable Populations | Jesse Nodora | | 1 Nov 2020 | 15 Nov 2021 |
| Implementing an Individualized Pain Plan (IPP) for ED Treatment of VOE's in Sickle Cell Disease | Duke University | sickle cell disease, vaso occlusive-episodes, Individualized Pain Plans, implementation science | 20 Oct 2020 | 1 Feb 2022 |
| Building Adaptive Coping and Knowledge to Improve Daily Life | Emory University | Pediatrics, Cognitive behavioral therapy, Behavioral intervention | 1 Oct 2020 | 1 Jun 2022 |
| Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD) | Novartis Pharmaceuticals | Gene therapy, genome-edited hematopoietic stem and progenitor cellular therapy, sickle cell, autologous transplant, BCL11A | 29 Sep 2020 | 20 Nov 2023 |
| Voxelotor Sickle Cell Exercise Study | Elizabeth Yang, MD, PhD | | 21 Sep 2020 | 20 Sep 2021 |
| Isoquercetin in Sickle Cell Anemia | Jeffrey Zwicker, MD | Sickle Cell Disease, Sickle Cell-Beta0-Thalassemia, isoquercetin | 1 Sep 2020 | 31 Dec 2024 |
| Medication Adherence and Non-adherence in Adults With Rare Disease | Raremark | Adherence, MAR-Scale, Rare disease, Observational | 1 Sep 2020 | 1 Sep 2022 |
| Dose Ranging Study of Benserazide in Thalassemia Intermedia | Phoenicia BioScience | Non-transfusion dependent beta thalassemia | 30 Aug 2020 | 1 Apr 2023 |
| Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC | Paul Szabolcs | Severe Combined Immune Deficiency (SCID) with NK cell activity, Omenn Syndrome, Bare Lymphocyte Syndrome (BLS), Combined Immune Deficiency (CID) syndromes, Wiskott-Aldrich Syndrome, Leukocyte adhesion deficiency, Chronic granulomatous disease (CGD), Hyper IgM (XHIM) syndrome, IPEX syndrome, Chediak-Higashi Syndrome, Autoimmune Lymphoproliferative Syndrome (ALPS), Hemophagocytic Lymphohistiocytosis (HLH) syndromes, Lymphocyte Signaling defects, Congenital Amegakaryocytic Thrombocytopenia (CAMT), Osteopetrosis, Hurler syndrome (MPS I), Hurler syndrome (MPS II), Krabbe Disease, also known as Globoid Cell Leukodystrophy, Metachromatic leukodystrophy (MLD), X-linked adrenoleukodystrophy (ALD), Alpha Mannosidosis, Gaucher Disease, Thalassemia major, Sickle cell disease (SCD), Diamond Blackfan Anemia (DBA), Crohn's Disease, Inflammatory Bowel Disease, IPEX or IPEX-like Syndromes, Rheumatoid Arthritis | 20 Aug 2020 | 30 Jun 2026 |
| Autologous Testicular Tissue Transplantation | Universitair Ziekenhuis Brussel | | 29 Jul 2020 | 29 Oct 2030 |
| Red Cell Half Life Determination in Patients With and Without Sickle Cell Disease | National Heart, Lung, and Blood Institute (NHLBI) | Red Cell Survival, Biotin, Sickle Cell Anemia, Sickle Cell Disease, Sickle Cell Trait | 28 Jul 2020 | 31 Dec 2021 |
| Frequency of COVID-19 Antibodies in Patients With Hereditary Hematological Diseases | University of Milano Bicocca | | 14 Jul 2020 | 30 Oct 2020 |
| A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell Disease | Agios Pharmaceuticals, Inc. | Sickle Cell Disease | 10 Jul 2020 | 1 Dec 2022 |
| Improving Scientific Rigor of Renal Clinical Endpoints for Sickle Cell Anemia | University of Alabama at Birmingham | | 1 Jul 2020 | 30 Jun 2025 |
| Desmopressin for Bedwetting in Children With SCD | Montefiore Medical Center | | 1 Jul 2020 | 1 Dec 2025 |
| Cognitive Behavioral Therapy and Real-Time Pain Management Intervention for Sickle Cell Via Mobile Applications | University of Pittsburgh | sickle cell disease, pain, opioids, depression, cognitive behavioral therapy, mobile technology, digital health, mHealth, implementation science, community-based participatory research | 1 Jul 2020 | 1 Aug 2024 |
| Sickle Cell Disease and the Genomic Needs of Parent and Pediatric Stakeholders | St. Jude Children's Research Hospital | Sickle Cell Disease, Sickle Cell Anemia, Hemoglobin SC Disease, Hemoglobin SS Disease, Hemoglobin S beta zero thalassemia, Adolescent, Parent | 1 Jul 2020 | 30 Jun 2021 |
| A Study of IMR-687 in Subjects With Sickle Cell Disease | Imara, Inc. | Sickle Cell Disease | 1 Jul 2020 | 1 Aug 2022 |
| A Stress and Pain Self-management m-Health App for Adult Outpatients With Sickle Cell Disease | University of Florida | | 1 Jul 2020 | 1 Jun 2025 |
| Low Dose Aspirin for Preventing Intrauterine Growth Restriction and Preeclampsia in Sickle Cell Pregnancy (PIPSICKLE) | University of Lagos, Nigeria | Sickle cell disease, Low dose aspirin, Preeclampsia, Intrauterine growth restriction (IUGR), Machine-learning, Pregnancy complications | 1 Jul 2020 | 30 Jun 2023 |
| Study to Evaluate the Effect of Ticagrelor Versus Placebo in Reducing Vaso-Occlusive Crises Rate in Pediatric Patients With Sickle Cell Disease. | AstraZeneca | Ticagrelor, Vaso-Occlusive Crises, Paediatric Patients, 6 Months to <18 Years, Sickle Cell Disease, Sickle Cell Anemia, Platelet aggregation, Brillinta, Acute Chest Syndrome, painful crisis | 30 Jun 2020 | 10 Oct 2022 |
| Living With Sickle Cell Disease in the COVID-19 Pandemic | National Human Genome Research Institute (NHGRI) | Mental Health, Quality of Life, Sickle Cell Pandemic Wellness | 30 Jun 2020 | 31 Dec 2024 |
| RHD Genotype Matching for Anti-D | Children's Hospital of Philadelphia | Chronic Transfusion | 1 Jun 2020 | 1 Jul 2024 |
| Thalassemic Iron Overload Cardiomyopathy is Ameliorated by Taurine Supplementation | University Health Network, Toronto | | 1 Jun 2020 | 31 May 2023 |
| Prevalence of Osteoporosis in Sickle Cell Disease | Hospices Civils de Lyon | sickle cell disease | 1 Jun 2020 | 1 Dec 2021 |
| Preliminary Feasibility and Efficacy of Behavioral Intervention to Reduce Pain-Related Disability in Pediatric SCD | Children's National Research Institute | | 1 Jun 2020 | 31 May 2021 |
| Actigraphy Improvement With Voxelotor (ActIVe) Study | Global Blood Therapeutics | | 1 Jun 2020 | 1 Nov 2021 |
| Red Blood Cell Survival in Sickle Cell Disease | Emory University | | 1 Jun 2020 | 1 May 2025 |
| Evaluation of Impact of Disease on Quality of Life, Education and Socio-professional Integration of Adults and Parents of Children Living With Sickle- Cell Disease in France | Argo Sante | Sickle-cell disease, quality of life, education, socio-professional integration, france | 1 Jun 2020 | 1 Jun 2021 |
| A Study of IMR-687 in Subjects With Beta Thalassemia | Imara, Inc. | Transfusion, TDT, NTDT | 1 Jun 2020 | 1 May 2022 |
| Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia | Institute of Hematology & Blood Diseases Hospital | Beta-Thalassaemia, Hematopoietic Stem-Cell Transplantation | 21 May 2020 | 15 Oct 2023 |
| Venous Thrombosis Biomarkers in Sickle Cell Disease and Sickle Cell Trait | National Heart, Lung, and Blood Institute (NHLBI) | hypercoagulable state, Recurrence, Biomarkers, Venous Thromboembolism, Vascular Mortality | 5 May 2020 | 15 Oct 2022 |
| SMILES: Study of Montelukast in Sickle Cell Disease | Great Ormond Street Hospital for Children NHS Foundation Trust | | 1 May 2020 | 31 Aug 2022 |
| Low Dose Ketamine and Acute Pain Crisis | Rhode Island Hospital | ketamine, emergency department, acute pain crisis | 15 Apr 2020 | 1 Jan 2021 |
| A Trial to Assess Haploidentical T-depleted Stem Cell Transplantation in Patients With SCD | University of Regensburg | | 1 Apr 2020 | 31 Mar 2027 |
| A Voxelotor for Sickle Cell Anemia Patients at Highest Risk for Progression of Chronic Kidney Disease | University of Illinois at Chicago | | 1 Apr 2020 | 1 Apr 2024 |
| Reduced Intensity Transplantation for Severe Sickle Cell Disease | St. Jude Children's Research Hospital | | 1 Apr 2020 | 1 Jul 2025 |
| Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemia | Vifor (International) Inc. | | 1 Apr 2020 | 1 May 2021 |
| Dermatological Abnormalities in Beta-thalassemia Major | Assiut University | | 2 Mar 2020 | 2 Apr 2021 |
| SMYLS: A Self-management Program for Youth Living With Sickle Cell Disease | Medical University of South Carolina | | 2 Mar 2020 | 31 Dec 2020 |
| β-globin Restored Autologous HSC in β-thalassemia Major Patients | Shanghai Bioray Laboratory Inc. | | 1 Mar 2020 | 1 Mar 2022 |
| Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells | Shanghai Bioray Laboratory Inc. | | 1 Mar 2020 | 1 Jan 2022 |
| Links Between Cognitive Functions and Clinical, Biological and Neuroradiological Outcomes in Adults With Sickle Cell Disease. | Hospices Civils de Lyon | Sickle Cell Disease, Drepanocytosis | 1 Mar 2020 | 1 Sep 2022 |
| Safety of Single Ascending Doses of CSL889 in Adult Patients With Stable Sickle Cell Disease | CSL Behring | | 1 Mar 2020 | 1 Mar 2021 |
| Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (THALA-RAP) | Università degli Studi di Ferrara | rapamycin, erythroid differentiation, γ-globin, fetal haemoglobin | 28 Feb 2020 | 30 May 2021 |
| Effect of Exercise on Biomarkers in SCT | St. Louis University | Sickle Cell Trait | 17 Feb 2020 | 14 May 2021 |
| A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease | bluebird bio | | 14 Feb 2020 | 1 Nov 2023 |
| A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease | EpiDestiny, Inc. | | 3 Feb 2020 | 3 Mar 2021 |
| Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease | Global Blood Therapeutics | Sickle Cell Disease, Transcranial Doppler Ultrasound (TCD) | 1 Feb 2020 | 1 Mar 2026 |
| High-flow Oxygen for Vaso-occlusive Pain Crisis | Assistance Publique - Hôpitaux de Paris | Sickle Cell Disease, vaso-occlusive pain crisis, HFNO, ACS | 1 Feb 2020 | 29 May 2021 |
| RH Genotype Matched RBC Transfusions | Children's Hospital of Philadelphia | Chronic Transfusion | 1 Feb 2020 | 1 Jul 2024 |
| Dissemination and Implementation of Stroke Prevention Looking at the Care Environment | University of Alabama at Birmingham | | 1 Feb 2020 | 1 Jun 2022 |
| Daily Vitamin D for Sickle-cell Respiratory Complications | Gary M Brittenham, MD | Sickle Cell Disease, Acute Chest Syndrome, Respiratory Complication, Vitamin D Deficiency | 1 Feb 2020 | 1 Dec 2024 |
| Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS TMPRSS6-LRx | Ionis Pharmaceuticals, Inc. | Thalassaemia, Beta Thalassemia, IONIS TMPRSS6-LRx | 1 Feb 2020 | 1 Oct 2022 |
| Acupuncture for Adults With Sickle Cell Disease (SCD): A Feasibility Study | University of Illinois at Chicago | sickle cell disease, pain, chronic pain, acupuncture | 15 Jan 2020 | 15 Apr 2020 |
| Qualitative Survey of Potential Exercise Activity in Adults With Sickle Cell Anemia (SCA) | University of Alabama at Birmingham | | 15 Jan 2020 | 15 Jan 2021 |
| FOCUS for Pediatric Sickle Cell Disease and Cancer | Georgia State University | Pediatric cancer, Pediatric Sickle Cell Disease, Quality of Life, Randomized Controlled Trial, Intervention, Photography, Narrative Psychology, Adolescence | 10 Jan 2020 | 30 Jul 2020 |
| Dose Escalation Study to Evaluate the Safety, Tolerability, PK and PD of Voxelotor in Patients With SCD | Global Blood Therapeutics | Sickle Cell Disease, SCD | 9 Jan 2020 | 1 Dec 2021 |
| Folic Acid Supplementation in Children With Sickle Cell Disease | University of British Columbia | Folic Acid, Unmetabolized Folic Acid, Pediatrics | 3 Jan 2020 | 1 Jan 2022 |
| Sickle Cell Disease and CardiovAscular Risk - Red Cell Exchange Trial (SCD-CARRE) | Mark Gladwin | | 1 Jan 2020 | 30 Apr 2026 |
| Ketamine Infusion for Sickle Cell Pain Crisis | Duke University | Ketamine, Intravenous Infusions, Anemia; Sickle-Cell, with Crisis, Acute Pain | 1 Jan 2020 | 31 Aug 2020 |
| Thrombophilia In Beta Thalassemia | Assiut University | | 1 Jan 2020 | 1 Jan 2021 |
| Is the Preoperative Preparation of Sickle Cell Patients Optimal: Assessment of Practices and Post-operative Complications | Queen Fabiola Children's University Hospital | | 1 Jan 2020 | 30 Jun 2020 |
| Clinical Experience of Thalidomide in Thalassemic Patients | Children's Hospital Karachi | | 1 Jan 2020 | 31 Jan 2021 |
| Gum Arabic as Anti-oxidant, Anti-inflammatory and Fetal Hemoglobin Inducing Agent in Sickle Cell Anemia Patients | Al-Neelain University | sickle cell anemia, Gum Arabic, Fetal Hemoglobin | 15 Dec 2019 | 15 Jul 2020 |
| Study Exploring the Effect of Crizanlizumab on Kidney Function in Patients With Chronic Kidney Disease Caused by Sickle Cell Disease | Novartis Pharmaceuticals | SEG101, SCD, Crizanlizumab, Sickle cell nephropathy, chronic kidney disease, CKD, albuminuria (ACR), renal function, standard of care | 10 Dec 2019 | 17 Sep 2022 |
| Haploidentical Hematopoietic Stem Cell Transplantation (HSCT) for Patients With Severe Sickle Cell Disease | University of Chicago | | 1 Dec 2019 | 1 Nov 2027 |
| A Study to Assess the Safety and Pharmacokinetics of HBI-002, an Oral Carbon Monoxide Therapeutic, in Healthy Volunteers | Hillhurst Biopharmaceuticals, Inc. | Anemia, Sickle Cell, Anemia, Hemolytic, Congenital, Anemia, Hemolytic, Anemia, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Carbon Monoxide, CO, Heme Oxygenase, HO-1 | 1 Dec 2019 | 1 May 2020 |
| DREPAMASSE Study - Evaluation of a Newborn Screening for Sickle Cell Disease by Tandem Mass Spectrometry | Hospices Civils de Lyon | | 1 Dec 2019 | 1 Dec 2020 |
| Quantitative Susceptibility Mapping (QSM) to Guide Iron Chelating Therapy | Weill Medical College of Cornell University | Quantitative Susceptibility Mapping, Iron chelating therapy, Iron overload, Liver iron concentration, erythrocyte transfusion, liver transplantation, magnetic resonance imaging, thalassemia major | 1 Dec 2019 | 31 Jan 2024 |
| Phase 2a Pilot Study of NBMI Treatment in Patients With Beta Thalassemia Major, Requiring Iron Chelation | EmeraMed | | 21 Nov 2019 | 30 Jun 2020 |
| Pediatric Open-Label Extension of Voxelotor | Global Blood Therapeutics | | 18 Nov 2019 | 1 Jan 2026 |
| Safety and Efficacy of Gene Therapy of the Sickle Cell Disease by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains CD34+ Cells Transduced ex Vivo With the GLOBE1 Lentiviral Vector Expressing the βAS3 Globin Gene in Patients With Sickle Cell Disease (DREPAGLOBE) | Assistance Publique - Hôpitaux de Paris | | 15 Nov 2019 | 15 Nov 2024 |
| Integration of mHEALTH Into the Care of Patients With Sickle Cell Disease to Increase Hydroxyurea Utilization | St. Jude Children's Research Hospital | implementation science, sickle cell anemia, digital medicine, adherence, hydroxycarbamide, health innovation | 11 Nov 2019 | 1 Jun 2022 |
| Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Subjects Who Require Regular RBC Transfusions Due to Beta (β)-Thalassemia. | Celgene | ACE-536, Luspatercept, Pharmacokinetics, Beta-Thalassemia, Red Blood Cell Transfusion | 7 Nov 2019 | 2 Sep 2026 |
| iPeer2Peer Program for Youth With Sickle Cell Disease | The Hospital for Sick Children | | 1 Nov 2019 | 1 May 2020 |
| Intranasal Ketamine For Pain Control In Patients With Sickle Cell Disease And Vaso-occlusive Episode (VOE) In The PED | Abigail Nixon | | 1 Nov 2019 | 1 Jan 2021 |
| Algorithm for Apherisis Monitoring and Prescription Assistance in Sickle Cell Patients (ALGODREP) | Etablissement Français du Sang | Algorithm, Apherisis, Sickle cell patients | 1 Nov 2019 | 1 Nov 2021 |
| Cystatin c and Beta 2 Microglobulin in Thalassemic Children. | Assiut University | renal impairment | 1 Nov 2019 | 1 Mar 2021 |
| Hydroxy Urea, Omega 3, Nigella Sativa,Honey on Oxidative Stress and Iron Chelation in Pediatric Major Thalassemia | Beni-Suef University | thymoquinone, omega3, hydroxyurea, pediatric Thalassemia Major, oxidative stress, iron chelation, iron overload, chelation activity of thymoquinone, antioxidant effect of omega3, anti-hemolysis effect of hydroxyurea | 1 Nov 2019 | 1 Nov 2020 |
| Different Treatment Modalities in the Management of the Painful Crisis in Pediatric Sickle- Cell Anemia | Beni-Suef University | omega3, Vaso-occlusive painful crisis, Pediatric sickle cell anemia, comparative effective analysis in VOC, Anti-inflammatory effect of Vit-D, Anti-hemolytic effect of Vit-D, Anti-hyperlipidemia of Vit-D, effect of omega-3 on blood rheology, effect of omega-3 on blood viscosity, Antiaggregation effect of omega-3, Anti-inflammatory effect of statins, Anti-inflammatory effect of zinc supplements, effect of zinc supplements on blood viscosity, effect of zinc supplements on blood rheology, Antiaggregation effect of zinc supplements, pediatric sickle cell disease | 1 Nov 2019 | 1 Nov 2020 |
| Peer i-Coaching for Activated Self-Management Optimization in Adolescents and Young Adults With Chronic Conditions | Duke University | | 29 Oct 2019 | 29 Feb 2024 |
| A Pilot Study on Neuroimaging in SCD: Part of The Boston Consortium to Cure Sickle Cell Disease | Boston Children’s Hospital | Sickle Cell, MRI, Near Infrared Spectroscopy (NIRS) | 25 Oct 2019 | 1 Aug 2020 |
| Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels | National Heart, Lung, and Blood Institute (NHLBI) | Graft-Versus-Host Disease, Donor Apheresis, Host-Donor Chimerism | 24 Oct 2019 | 1 Jan 2030 |
| A Study of SHP655 (rADAMTS13) in Sickle Cell Disease | Shire | | 21 Oct 2019 | 27 Aug 2021 |
| Cerebral Oxygen Metabolism in Children | Washington University School of Medicine | extracorporeal membrane oxygenation | 17 Oct 2019 | 1 Dec 2027 |
| A Study to Evaluate the Safety and Efficacy of Crizanlizumab in Sickle Cell Disease Related Priapism | Novartis Pharmaceuticals | Priapism, sickle cell disease, crizanlizumab, P-selectin, SEG101, monoclonal antibody, prolonged erection, painful erection | 16 Oct 2019 | 4 Mar 2022 |
| Comparison of Allogeneic Matched Related Haematopoietic Stem Cell Transplantation After a Reduced Intensity Conditioning Regimen With Standard of Care in Adolescents and Adults With Severe Sickle Cell Disease | Assistance Publique - Hôpitaux de Paris | | 15 Oct 2019 | 15 Oct 2024 |
| CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease | CSL Behring | | 2 Oct 2019 | 1 Jul 2021 |
| Effect of MitoQ on Platelet Function and Reactive Oxygen Species Generation in Patients With Sickle Cell Anemia | University of Pittsburgh | Basal platelet activation, MitoQ, mitochondrial ROS (Reactive Oxygen Species) | 1 Oct 2019 | 31 Dec 2020 |
| The Impact of Oxidative Stress on Erythrocyte Bilogy | Versiti | | 1 Oct 2019 | 1 Oct 2023 |
| AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion | Mitchell Cairo | allogeneic stem cell transplantation, t-cell depletion, alpha beta cell depletion | 1 Oct 2019 | 31 Dec 2024 |
| The Role of OCTA in Patients Affected by Beta Thalassemia | Federico II University | | 1 Oct 2019 | 28 Feb 2020 |
| Collection of Human Biospecimens for Basic and Clinical Research Into Alpha Globin Variants | National Institute of Allergy and Infectious Diseases (NIAID) | Assay Development, Malaria, Sickle Cell Disease, Alpha Thalassemia | 25 Sep 2019 | 31 Jul 2028 |
| To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease | Axcella Health, Inc | Amino Acid, Food Study | 25 Sep 2019 | 1 Oct 2020 |
| Effect of Platelet Inhibition and / or Lipid Lowering in Non-ACS-patients With Positive Troponin | Dr. med. Mahir Karakas | Aspirin, Atorvastatin, Troponin | 1 Sep 2019 | 1 Apr 2022 |
| Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant | Emory University | Pediatrics, Non-malignant hematologic diseases, Severe aplastic anemia, Fanconi anemia, Sickle cell disease, Hematopoietic stem cell transplantation, Thalassemia, Mismatched unrelated donor | 22 Aug 2019 | 1 Dec 2023 |
| Study in Beta-thalassaemia or Myelodysplastic Syndrome Patients to Investigate the Safety and Tolerability of SLN124 | Silence Therapeutics plc | | 20 Aug 2019 | 14 Oct 2021 |
| A Study to Evaluate Long-term Safety in Subjects Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials | Celgene | ACE-536, Luspatercept, MDS, Beta-thalassemia, Myeloproliferative neoplasm (MPN)-associated myelofibrosis | 12 Aug 2019 | 24 Jan 2028 |
| Comparing Individualized vs. Weight Based Protocols to Treat VOE in SCD Occlusive Episodes in Sickle Cell Disease | Duke University | | 6 Aug 2019 | 7 Jan 2022 |
| Deferoxamine for Sickle Cell Chronic Leg Ulcer Treatment | TauTona Group | chronic sickle cell leg ulcer | 1 Aug 2019 | 1 Apr 2021 |
| Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients | Novartis Pharmaceuticals | Sickle Cell Disease, SEG101, Crizanlizumab, Hydroxyurea/ Hydroxycarbamide Therapy, Vaso-Occlusive Crises, SCA, blood disorders, hemoglobin, red blood cells, sickle-like shape, mutation in hemoglobin gene, sickle-cell trait, sickle-cell crisis | 26 Jul 2019 | 6 Dec 2027 |
| Dronabinol for Pain and Inflammation in Adults Living With Sickle Cell Disease | Yale University | | 26 Jul 2019 | 31 Dec 2020 |
| Hydroxyurea Exposure Limiting Pregnancy and Follow-Up Lactation | Children's Hospital Medical Center, Cincinnati | Hydroxyurea | 22 Jul 2019 | 1 Jul 2022 |
| Neuropathic Pain in Jamaicans With Sickle Cell Disease | The University of The West Indies | Quantitative sensory testing, Jamaica | 15 Jul 2019 | 1 Jul 2020 |
| Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Multiple Oral Doses of AG-348 in Subjects With Stable Sickle Cell Disease | National Heart, Lung, and Blood Institute (NHLBI) | HbS polymerization, pyruvate kinase, 2,3- diphosphoglycerate, ATP in red blood cells, acute sickle pain | 11 Jul 2019 | 31 Dec 2020 |
| Sickle Cell Pain: Intervention With Capsaicin Exposure | Children's Hospital of Michigan | Capsaicin, Topical Analgesia, Quantitative Sensory Testing, Pain | 3 Jul 2019 | 30 Jun 2020 |
| Screening for Hemoglobinopathies in Pregnant Women | University Hospital, Basel, Switzerland | thalassemia, sickle cell anaemia, molecular genetic analysis | 3 Jul 2019 | 1 Jul 2020 |
| Sickle Cell Uric Acid (SCUA) - Cohort Repository | Virginia Commonwealth University | Uric acid | 1 Jul 2019 | 1 Oct 2024 |
| Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients | Rare Partners srl Impresa Sociale | Fetal hemoglobin, Sirolimus, Red Blood Cells | 27 Jun 2019 | 30 Sep 2020 |
| Near Infrared Spectroscopy in Sickle Cell Pediatric Patients | Children's Research Institute | | 26 Jun 2019 | 30 Jun 2022 |
| Clinical Transplant-Related Long-term Outcomes of Alternative Donor Allogeneic Transplantation | Center for International Blood and Marrow Transplant Research | | 14 Jun 2019 | 1 Mar 2025 |
| A Program to Increase Sickle Cell Trait Knowledge Among Parent of Infants Identified in Newborn Screening | Nationwide Children's Hospital | Sickle Cell Trait, Education | 13 Jun 2019 | 31 Dec 2021 |
| Hydroxyurea Therapy: Optimizing Access in Pediatric Populations Everywhere | St. Jude Children's Research Hospital | | 10 Jun 2019 | 30 Jun 2020 |
| The Effect of N-acetylcysteine on Oxidative Stress Status and Iron Overload in Thalassemia Major | Beni-Suef University | | 3 Jun 2019 | 30 Dec 2019 |
| Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study | First Affiliated Hospital of Guangxi Medical University | thalassemia major, hematopoietic stem cell transplantation | 1 Jun 2019 | 31 May 2023 |
| Red Cell Distribution Width Index Versus Red Cell Distribution Width as Discriminating Guide for Iron Deficiency Anaemia and Beta Thalassemia Trait . | Assiut University | | 1 Jun 2019 | 1 Jun 2022 |
| SIKAMIC (SIklos on Kidney Function and AlbuMInuria Clinical Trial) | ADDMEDICA SASA | albuminuria, hydroxycarbamide | 28 May 2019 | 1 Jun 2021 |
| Best Noninvasive Predictor of Renal Function in Assessing Adult Sickle Nephropathy | National Heart, Lung, and Blood Institute (NHLBI) | Sickle Nephropathy, Iothalamate, Cystatin-C | 24 May 2019 | 1 May 2021 |
| An Extension Study of IMR-687 in Adult Patients With Sickle Cell Anemia | Imara, Inc. | | 22 May 2019 | 1 Jun 2024 |
| Pharmacokinetic (PK) and Pharmacodynamics (PD) Study of Ilera Specific Products | Children's Hospital of Philadelphia | | 15 May 2019 | 31 Dec 2020 |
| Safety Study for Beta Thalassemia Subjects on PTG-300 | Protagonist Therapeutics | Chronic anemia, β-thalassemia | 10 May 2019 | 10 May 2023 |
| Expanded Access to Voxelotor for Patients With Sickle Cell Disease Who Have No Alternative Treatment Options | Global Blood Therapeutics | | 9 May 2019 | 19 Dec 2019 |
| TAPS2 Transfusion Antenatally in Pregnant Women With SCD | Guy's and St Thomas' NHS Foundation Trust | pregnancy, exchange transfusion, perinatal complications | 2 May 2019 | 1 May 2021 |
| Combined Use of a Respiratory Broad Panel Multiplex PCR and Procalcitonin to Reduce Antibiotics Exposure in Hospitalized Sickle-cell Adults With Acute Chest Syndrome. A Bi-centric, Open, Parallel-group, Randomized Controlled Study | Assistance Publique - Hôpitaux de Paris | Acute chest syndrome, SCD, PCR multiplex, antibiotic treatment | 1 May 2019 | 1 Jun 2020 |
| Choroidal Thickness in Beta-thalassemia Patients | University Hospital of Patras | beta-thalassemia, choroidal thickness, enhanced depth imaging, optical coherence tomography | 1 May 2019 | 1 Sep 2019 |
| SP-420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias | Abfero Pharmaceuticals, Inc | chelator | 1 May 2019 | 1 Jan 2022 |
| Stroke Prevention With Hydroxyurea Enabled Through Research and Education (SPHERE) | Children's Hospital Medical Center, Cincinnati | Sickle Celll Anemia, Transcranial Doppler, Tanzania, Hydroxyurea, Stroke | 24 Apr 2019 | 31 Mar 2022 |
| Sexual Maturation in β-Thalassemia Major Patients in Assiut University Hospital | Assiut University | | 15 Apr 2019 | 15 Apr 2020 |
| Pulmonary Ultrasound in the Diagnosis of Acute Thoracic Syndrome in Vaso-occlusive Sickle Cell Crisis | Centre Hospitalier Intercommunal Creteil | | 4 Apr 2019 | 1 Aug 2020 |
| Observing the Changes of Endocrine and Metabolism in Patients With Thalassemia Major | National Taiwan University Hospital | | 1 Apr 2019 | 1 Apr 2020 |
| Early Human Leukocyte Antigen (HLA) Matched Sibling Hematopoietic Stem Cell Transplantation | Emory University | Pediatrics, Hematopoietic stem cell transplantation, HLA matched sibling | 22 Mar 2019 | 1 Jan 2027 |
| Apotransferrin in Patients With β-thalassemia | Sanquin Plasma Products BV | | 21 Mar 2019 | 1 May 2021 |
| Role of Ajwa Derived Polyphenols in Dyslipidaemias | Aga Khan University | Ajwa (Pheonix Dactylfera) Derived polyphenol, Dyslipidemia, Cardioprotection, HDL, Transferulic Acid, Gallic Acid | 20 Mar 2019 | 7 Dec 2019 |
| Collect of Cord Blood From Subjects at Risk for Sickle Cell Disease, for the Purpose of Laboratory Research | Institut National de la Santé Et de la Recherche Médicale, France | | 1 Mar 2019 | 1 Mar 2022 |
| Detection Of β-thalassemia Carriers In Assiut | Assiut University | HbA2, HPLC | 1 Mar 2019 | 1 Jun 2021 |
| Resistance Exercise on Postprandial Hyperglycemia in Patients With B-thalassemia Exhibiting Resistance to Insulin | University of Thessaly | | 11 Feb 2019 | 30 Oct 2019 |
| Whole Exome Sequencing and Whole Genome Sequencing for Non-immune Fetal/Neonatal Hydrops | Thomas Jefferson University | hydrops, prenatal diagnosis, amniocentesis, whole genome sequencing | 15 Jan 2019 | 1 Dec 2021 |
| Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia | Protagonist Therapeutics | chronic anemia, β-thalassemia | 19 Dec 2018 | 1 Dec 2020 |
| A SAD/MAD to Assess the Safety, Pharmacokinetics and Pharmacodynamics of FT-4202 in Healthy Volunteers and Sickle Cell Disease Patients | Forma Therapeutics, Inc. | | 11 Dec 2018 | 1 Sep 2020 |
| Short-term Clinical Study of CN128 in Thalassemia Patients | Hangzhou Zede Pharma-Tech Co., Ltd. | | 3 Dec 2018 | 17 Dec 2019 |
| Outcomes Mandate National Integration With Cannabis as Medicine | OMNI Medical Services, LLC | | 1 Dec 2018 | 31 Dec 2025 |
| Impact on the Length of Stay in Incentive Spirometry and Pain in the Decompensation of Sickle Cell Disease: . | Centre Hospitalier Régional d'Orléans | Sickle cell disease, incentive spirometry, pain, children | 6 Nov 2018 | 6 Apr 2019 |
| Community Health Workers and mHealth for Sickle Cell Disease Care | Children's Hospital of Philadelphia | Sickle Cell Disease | 1 Nov 2018 | 30 Nov 2022 |
| Acupuncture for Pain in Sickle Cell Disease | Deepika Darbari | Pain, Acupuncture | 23 Oct 2018 | 23 Oct 2026 |
| PINPOINT: Gaming Technology for SCD Pain | Klein Buendel, Inc. | | 19 Sep 2018 | 30 Jun 2021 |
| Study of a Red Blood Cell Deformability Parameter in Patients With Sickle Cell Disease | Assistance Publique Hopitaux De Marseille | | 14 Sep 2018 | 14 Sep 2021 |
| Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients | Novartis Pharmaceuticals | SEG101, Sickle cell disease, crizanlizumab, pediatric, pharmacokinetic, P-selectin | 30 Aug 2018 | 31 Mar 2022 |
| Fetal Electrophysiologic Abnormalities in High-Risk Pregnancies Associated With Fetal Demise | Medical College of Wisconsin | Fetal Magnetocardiography, Stillbirth, Intrauterine Fetal Demise, Mobile Medical Technologies, Fetal Heart Rate Variability, Fetal Arrhythmias, High Risk Pregnancy, Pregnancy, Fetal Anomaly, Fetal Echocardiography, Non-Stress Testing, New Technology, Birth Defects, Fetal Research | 1 Jul 2018 | 30 Apr 2022 |
| PK of SOF/LED in HCV - Infected Adolescents With Haematological Disorders | Ain Shams University | Hepatitis C virus, Pharmacokinetic Modeling, Pharmacokinetics, Sofosbuvir, Ledipasvir, Direct acting antivirals, Beta thalassemia major | 11 Jun 2018 | 1 Dec 2020 |
| Impact of the Preparation Method of Red Cell Concentrates on Transfusion Indices in Thalassemic Patients | Università degli Studi di Ferrara | Packed RBCs, leukodepletion, buffy-coat, transfusion | 14 May 2018 | 1 Jun 2019 |
| Vitamin D Supplementation in Children With Sickle Cell Disease | St. Justine's Hospital | Bolus supplementation, Pilot randomised controlled trial, Vitamin D | 1 May 2018 | 31 Jul 2019 |
| Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (HABIT): Efficacy Trial | Columbia University | Hydroxyurea, Community health worker (CHW), Text messages, Medication adherence | 1 May 2018 | 1 Jun 2021 |
| Complications in Children With B- Thalassemia Major | Assiut University | | 1 May 2018 | 1 Oct 2019 |
| Fetal Birth Defects: Toward a Precision-based Approach | University of California, San Francisco | hydrops fetalis, birth defect, fetal anomaly, whole exome sequencing | 1 Apr 2018 | 1 Dec 2025 |
| Immunomodulatory Vitamin D in Thalassemia | Universitas Padjadjaran | | 1 Apr 2018 | 15 Aug 2018 |
| Hemoglobin Desaturation in Sickle Cell Disease | University Hospitals Cleveland Medical Center | | 23 Mar 2018 | 1 Dec 2022 |
| Adding Azathioprine/Hydroxyurea Preconditioning to Alemtuzumab/TBI to Reduce Risk of Graft Failure in MSD HSCT in Adult SCD Patients | Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) | Sickle Cell Disease, Allogeneic stem cell transplantation, Mixed chimerism | 8 Mar 2018 | 1 Dec 2023 |
| Hyperbaric Oxygen Therapy in Sickle Cell Pain | University of Nebraska | | 1 Mar 2018 | 1 Mar 2019 |
| A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT) | Sangamo Therapeutics | Beta thalassemia, Beta-thalassemia, Thalassemia major, Cooley's anemia, ZFN mediated genome editing, zinc finger nuclease | 1 Mar 2018 | 1 May 2022 |
| Shared-Decision Making for Hydroxyurea | Children's Hospital Medical Center, Cincinnati | shared decision making, parent-provider communication, hydroxyurea, sickle cell disease | 1 Mar 2018 | 30 Nov 2020 |
| Quantitative MRI for Patients With Sickle Cell Disease Undergoing Hematopoietic Cell Transplant | University of Michigan | bone marrow cellularity, bone marrow transplant | 1 Mar 2018 | 1 Mar 2023 |
| Stem Cell Transplant in Patients With Severe Sickle Cell Disease | Kathleen Dorritie | | 15 Feb 2018 | 15 Feb 2023 |
| Risk Factors for Allo-immunization in Sickle Cell Disease | Hanane EL KENZ | Sickle cell disease, Allo-immunization | 1 Feb 2018 | 1 Dec 2018 |
| Transfusion in Sickle Cell Disease: Risk Factors for Alloimmunization | Hanane EL KENZ | Sickle cell disease, Allo-immunization, Blood transfusion | 1 Feb 2018 | 1 Jan 2020 |
| Transfusion in Sickle Cell Disease: Screening of Sickle Cell Disease Trait in Blood Donors | Hanane EL KENZ | Sickle cell, Heterozygous, Blood donor | 1 Feb 2018 | 1 Jan 2020 |
| Predictors and Outcomes in Patients With Sickle Cell Disease | St. Jude Children's Research Hospital | Health Care Transition, Adolescent, Young Adult | 1 Feb 2018 | 1 Feb 2022 |
| HRV-B for Symptom Management in Sickle Cell Patients | Greenville Health System | Heart Rate Variability Biofeedback | 22 Jan 2018 | 1 Oct 2018 |
| Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome | New York Medical College | | 12 Jan 2018 | 1 Jan 2022 |
| Autonomic Nervous System and Sickle Cell Disease | Assistance Publique - Hôpitaux de Paris | sickle cell disease; asthma; beta-agonist; hear rate variability | 5 Jan 2018 | 31 May 2019 |
| iCanCope With Sickle Cell Pain | Seattle Children's Hospital | | 1 Jan 2018 | 1 Dec 2022 |
| A Phase - IIa - IIb, Open Label Trial to Study the Safety, Tolerability and Efficacy of Memantine as a Long-term Treatment of SCD | HaEmek Medical Center, Israel | | 1 Jan 2018 | 30 Jun 2020 |
| Efficacy and Safety of RBCs Derived From Mirasol-treated Whole Blood in Patients Requiring Chronic Transfusion (PRAISE) | Terumo BCTbio | Thalassemia, pathogen reduction therapy | 1 Jan 2018 | 1 Jun 2020 |
| A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia | Celgene | (β)-Thalassemia, Beta-Thalassemia, Phase 2, Luspatercept, ACE-536, Safety, Efficacy, Placebo, Red Blood Cell Transfusions, Erythrocyte transfusion, Hb increase, NTDT | 1 Jan 2018 | 1 Feb 2020 |
| A Study of IMR-687 in Adult Patients With Sickle Cell Anaemia (Homozygous HbSS or Sickle-β0 Thalassemia) | Imara, Inc. | | 1 Jan 2018 | 1 Jun 2019 |
| Role of Virtual Reality (VR) in Patients With Sickle Cell Disease (SCD) | Duke University | | 1 Jan 2018 | 1 Jul 2018 |
| Ketamine for Acute Painful Crisis in Sickle Cell Disease Patients | Dammam University | | 1 Jan 2018 | 27 Feb 2019 |
| A Study of the Effect of IW-1701, a Stimulator of Soluble Guanylate Cyclase (sGC), on Patients With Sickle Cell Disease (SCD) | Ironwood Pharmaceuticals, Inc. | Sickle Cell Disease, SCD | 1 Dec 2017 | 1 Jul 2019 |
| Effect of Virtual Reality Technology for Pain Management of Vaso-Occlusive Crisis in Patients With Sickle Cell Disease | St. Jude Children's Research Hospital | Virtual reality, Pain, Nonpharmacological | 1 Dec 2017 | 1 Dec 2020 |
| Quality of Life and Treatment Satisfaction in β-Thalassemia Patients Receiving Deferasirox | Assiut University | | 1 Dec 2017 | 1 Mar 2020 |
| Study on the Mechanism of Colla Corri Asini in the Treatment of Thalassemia Patients With Pregnancy Anemia | The First Affiliated Hospital, Guangzhou University of Traditional Chinese Medicine | Thalassemia, Pregnancy, Globin expression, Colla corii asini | 1 Dec 2017 | 28 Feb 2019 |
| Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer | Roswell Park Cancer Institute | | 20 Nov 2017 | 6 Sep 2022 |
| Non-Myeloablative Conditioning Regimen With Haploidentical T-Cell-Depleted Peripheral Blood Transplant for Patients With Severe Sickle Cell Disease | City of Hope Medical Center | Sickle Cell Disease, Sickle Cell Disorders, Hemoglobinopathies, Thalassemia, Anemia, Sickle Cell, Haploidentical Transplant, Nonmyeloablative Conditioning, CD4+ T cell, CD4+ T cell-depleted Hematopoietic Cell Transplant, Mixed Chimerism | 1 Nov 2017 | 1 Nov 2021 |
| Re-Aiming at Hydroxyurea Adherence for Sickle Cell With mHealth | St. Jude Children's Research Hospital | mHealth, Medication adherence, Barriers to adherence | 1 Nov 2017 | 1 Nov 2021 |
| A Study of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent Beta-Thalassemia | Hoffmann-La Roche | | 30 Oct 2017 | 1 May 2018 |
| Sickle Cell Disease: Targeting Alloantibody Formation Reduction; Risk Factors, and Genetics | Sanquin Research & Blood Bank Divisions | | 8 Oct 2017 | 31 Dec 2019 |
| Enhancing Preventive Therapy of Malaria In Children With Sickle Cell Anemia in East Africa (EPiTOMISE) | Duke University | | 1 Oct 2017 | 1 Oct 2019 |
| Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease | City of Hope Medical Center | Sickle Cell Disease, Hematopoietic stem cell transplantation, Haploidentical stem cell transplantation, Post-transplant Cytoxan | 1 Oct 2017 | 1 Aug 2022 |
| Gene Transfer for Sickle Cell Disease | David Williams | Gene therapy, lentivirus vector, BCL11A, Fetal Hemoglobin | 1 Oct 2017 | 1 Oct 2024 |
| Hyalornic Acid Level in β-Thalassemic Children Treated for Hepatitis C Virus | Tanta University | | 1 Oct 2017 | 1 Mar 2019 |
| Muscle Function and Its Biological and Physiological Determinants in Sickle Cell Disease | Hospices Civils de Lyon | Sickle Cell Disease, Muscle function, Hemorheological disorders | 30 Sep 2017 | 30 Sep 2019 |
| Sickle Cell Trait and Exercise, Effect of Hot Environment | University of the French West Indies and French Guiana | Exercise, sickling, rhamdomyolysis, oxidative stress | 25 Sep 2017 | 1 Jul 2019 |
| Gene Therapy for Beta-Thalassemia Major Using Autologous Hematopoietic Stem Cell Genetically Modified | Nanfang Hospital of Southern Medical University | Beta thalassemia, Gene therapy, Beta-globin, Hematopoietic stem cells, Lentiviral vector | 15 Sep 2017 | 15 Sep 2021 |
| iCanCope With Sickle Cell Disease: a Computerized Cognitive Behavioral Therapy Program for Pain Management | University of Pittsburgh | | 1 Sep 2017 | 1 Sep 2019 |
| Adiponectin, IL-6 and hsC-RP in Relation to Carotid Intima-media Thickness in B-thalassemia Patients | Asmaa Nady Hussein | | 1 Sep 2017 | 1 Mar 2020 |
| HLA Haploidentical Bone Marrow Transplant in Patients With Severe Sickle Cell Disease | Centre Hospitalier Intercommunal Creteil | sickle cell disease, haploidentical, graft, marrow | 1 Sep 2017 | 1 Sep 2020 |
| sPLA2 in EBC During Acute Chest Syndrome | Virginia Commonwealth University | acute chest syndrome, secretory phospholipases A2, exhaled breath condensate, sickle cell disease | 1 Sep 2017 | 28 Feb 2018 |
| Haploidentical Bone Marrow Transplantation in Sickle Cell Patients | Medical College of Wisconsin | Reduced Intensity Conditioning, Haploidentical, Bone Marrow, Transplant, Sickle Cell Disease | 1 Sep 2017 | 1 Dec 2024 |
| Kidney Function in Sickle Cell Anemia | University of North Carolina, Chapel Hill | | 1 Sep 2017 | 31 Aug 2022 |
| Mesenchymal Stromal Cells for Haplo Hematopoietic Cell Transplantation for Sickle Cell Disease | Emory University | Hematopoietic cell transplantation (HCT), Haploidentical, Mesenchymal stromal cells | 1 Sep 2017 | 1 Sep 2021 |
| The Implementation of the Automated Erythrocytapheresis in Egyptian Sickle Cell Disease Center | Ain Shams University | | 16 Aug 2017 | 1 Jan 2020 |
| A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT | Phoenix Children's Hospital | | 1 Aug 2017 | 1 Oct 2018 |
| Zinc Supplementation in Patients With β-Thalassemia Major Complicated With Diabetes Mellitus | Ain Shams University | Zinc, blood glucose, diabetes, beta thalassemia | 1 Aug 2017 | 28 Aug 2018 |
| Peripheral Blood Stem Cell Collection for Sickle Cell Disease (SCD) Patients | National Heart, Lung, and Blood Institute (NHLBI) | Plerixafor, Leukapheresis, Mobilization, Stem Cells, Sickle Cell Disease | 25 Jul 2017 | 28 Feb 2027 |
| Sickle Cell Disease, Hemechip | University Hospitals Cleveland Medical Center | | 11 Jul 2017 | 30 May 2022 |
| Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor | University of Calgary | sickle cell disease, stem cell transplant, red blood cell engraftment, nonmyeloablative, pure red cell aplasia | 5 Jul 2017 | 1 Jul 2023 |
| Laboratory-based Hypnosis Intervention on Pain Responsivity in Adolescents With Sickle Cell Disease and Healthy Controls | University of California, Los Angeles | Adolescence, Autonomic Nervous System, Hypnosis, Pain, Sickle Cell Disease | 1 Jul 2017 | 1 Jun 2020 |
| BEATS 2: Music Therapy in Sickle Cell | University Hospitals Cleveland Medical Center | | 26 Jun 2017 | 31 Dec 2018 |
| An Open Label Randomized Controlled Trial to Evaluate the Efficacy and Safety of HYDROXYUREA in Management of Beta Thalassemia Patients in Karachi Pakistan | Dr.Saqib Hussain Ansari | | 13 Jun 2017 | 30 Jan 2018 |
| Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers | Ionis Pharmaceuticals, Inc. | Thalassaemia, IONIS TMPRSS6-Lrx | 6 Jun 2017 | 31 Mar 2018 |
| Erythrocyte Glutamine/Glutamate Ratio Relation to Pulmonary Hypertension Risk in Thalassemic Children | Assiut University | Thalassemia, Glutamine/glutamate ratio, Pulmonary hypertension | 1 Jun 2017 | 1 Jun 2018 |
| Sleep and Pain in Sickle Cell Disease | Johns Hopkins University | | 1 Jun 2017 | 30 May 2021 |
| Evaluation of Nutritional Status in Thalassemia Major Patients in Assiut Children Hospital | Assiut University | thalassemia - nutrition -growth | 1 Jun 2017 | 1 Dec 2018 |
| Examining the Knowledge, Attitudes, and Beliefs of Sickle Cell Disease Patients, Parents of Patients With Sickle Cell Disease, and Providers Towards the Integration of CRISPR in Clinical Care | National Human Genome Research Institute (NHGRI) | CRISPR, Gene Editing, Qualitative, Participation Clinical Trials, Genetic Literacy | 1 Jun 2017 | 30 Jun 2018 |
| A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Subjects With Transfusion-Dependent β-Thalassemia, Who Have a β0/β0 Genotype | bluebird bio | | 1 Jun 2017 | 1 Apr 2021 |
| A Study to Compare if the Uptake of Ticagrelor in the Body Differs When Different Tablets Are Administered. | AstraZeneca | Abnormal hemoglobin (called hemoglobin S or sickle hemoglobin) in the red blood cells, Thrombotic cardiovascular events, Acute coronary syndrome, P2Y12 platelet inhibitor, Brilinta | 4 May 2017 | 3 Jul 2017 |
| The Effective and Safety of Thalidomide in TI | Xiao-Lin Yin | Thalidomide TI | 2 May 2017 | 2 May 2019 |
| A Study to Evaluate Safety, Pharmacokinetic, and Biological Activity of INCB059872 in Subjects With Sickle Cell Disease | Incyte Corporation | Sickle cell disease (SCD), sickle cell SS, lysine demethylase 1 (LSD1) inhibition | 1 May 2017 | 1 Jun 2017 |
| Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease | University of Illinois at Chicago | | 28 Apr 2017 | 28 Oct 2026 |
| Precision Exercise in Children With Malignant Hemopathies | University of Milano Bicocca | exercise, children, adolescent, cancer, malignant hemopathies, leukemia, lymphoma, hematopoietic stem cell transplant, survivor, oxidative metabolism, strength, balance, flexibility, quality of life | 3 Apr 2017 | 3 Apr 2022 |
| Denosumab Versus Zoledronic Acid in Thalassemia-Induced Osteoporosis | Hamad Medical Corporation | Osteoporosis, Beta-Thalassemia Major, Denosumab, Zoledronic Acid | 1 Apr 2017 | 1 Apr 2018 |
| EPO-4-Rhesus: Role of Darbepoetin Alfa in Reducing Late Anaemia in Neonates With Red Blood Cell Alloimmunization After Intrauterine Transfusions | Sanquin-LUMC J.J van Rood Center for Clinical Transfusion Research | Hemolytic disease of the fetus and newborn, HDFN, Red blood cell alloimmunization | 1 Apr 2017 | 1 Aug 2020 |
| Evaluation of Knowledge Among Adolescents With Sickle Cell Disease. | Adele CARLIER-GONOD | | 10 Mar 2017 | 1 Mar 2018 |
| Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease | National Heart, Lung, and Blood Institute (NHLBI) | Peripheral Blood Stem Cells, Host-Donor Chimerism, Graft Versus Host Disease, Haploidentical, Donor Apheresis | 8 Mar 2017 | 31 Aug 2026 |
| Sofosbuvir/Ledipasvir for Hepatitis C Genotype 1-6 in Patients With Transfusion-Dependent Thalassemia: An Open Label Trial | Ala'a Sharara | Thalassemia, HCV, Sofosbuvir, Ledipasvir | 15 Feb 2017 | 15 Aug 2017 |
| In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM) | University of California, San Francisco | intrauterine transfusion, in utero human stem cell transplantation | 1 Feb 2017 | 1 Feb 2024 |
| Hydroxyurea Management in Kids: Intensive Versus Stable Dosage Strategies | St. Jude Children's Research Hospital | Sickle cell, Hydroxyurea, Infants | 1 Feb 2017 | 1 Jun 2020 |
| New Hemolysis Parameters in Sickle Cell Disease | Queen Fabiola Children's University Hospital | | 1 Feb 2017 | 27 Feb 2019 |
| Pathophysiology of Acute Pain in Patients With Sickle Cell Disease | National Heart, Lung, and Blood Institute (NHLBI) | Pain, Cell Free DNA, Inflammatory Markers, Microparticles, NETosis | 31 Jan 2017 | 31 Oct 2021 |
| Gaming Technology to Engage Adolescent Sickle Cell Patients in Pain Management | Klein Buendel, Inc. | Sickle Cell Disease, Adolescence, Pain Assessment, Tablet Application, Pain, mHealth | 15 Jan 2017 | 30 Sep 2017 |
| Hydoxycarbamide and L-Carnitine Therapy in Sickle Cell Anemia | Assiut University | | 10 Jan 2017 | 10 Jul 2021 |
| Omega-3 Fatty Acids in Sickle Cell Disease | Robin E. Miller | Sickle Cell Disease, omega-3 fatty acids, Docosahexaenoic Acid (DHA), Eicosapentaenoic Acid (EPA) | 1 Jan 2017 | 1 Apr 2019 |
| Dose-Finding Study of SC411 in Children With Sickle Cell Disease | Sancilio and Company, Inc. | | 1 Jan 2017 | 1 Jul 2017 |
| A Pilot Study of Fecal Microbiome and Neutrophil Cellular Adhesion Molecules in Patients With Sickle Cell Disease (SCD) | Rhode Island Hospital | | 1 Jan 2017 | 1 Dec 2017 |
| A Phase Ib Study of NVX-508 in Sickle Cell Disease | Amma Owusu-Ansah, MD | | 1 Jan 2017 | 1 Dec 2017 |
| The Effect of Alpha-tocopherol in Hemolysis and Oxidative Stress Marker on the Red Cell Membrane Beta-thalassemia Major | Indonesia University | Haptoglobin, Hemopexin, Hemolysis, Malondialdehyde, Glutathione, Oxidative stress, Alpha-Tocopherol | 30 Dec 2016 | 1 Aug 2017 |
| Sickle-cell Disease Registry of the GPOH | University Hospital Heidelberg | Anemia, Sickle Cell, Sickle Cell disease | 15 Dec 2016 | 31 Dec 2040 |
| A Study of EPEG in Beta Thalassemia Patients | Prolong Pharmaceuticals | | 1 Dec 2016 | 1 Jun 2017 |
| Precision Medical Research of Non-immune Fetal Hydrops (NIFH)-From Prenatal Diagnosis to Intrauterine Treatment | Shanghai First Maternity and Infant Hospital | | 1 Dec 2016 | 1 Aug 2018 |
| The Potential Hepatoprotective Effect of Metformin in Patients With Beta Thalassemia Major | Cairo University | Beta Thalassemia, Metformin, Oxidative stress, Iron overload, Liver function | 1 Dec 2016 | 1 May 2018 |
| Pilot and Feasibility Trial of Plerixafor for Hematopoietic Stem Cell (HSC) Mobilization in Patients With Sickle Cell Disease Pilot and Feasibility Trial of Plerixafor for Hematopoietic Stem Cell (HSC) Mobilization in Patients With Sickle Cell Disease | Alessandra Biffi | Plerixafor | 1 Dec 2016 | 1 Jan 2018 |
| Study to Evaluate the Effect of GBT440 Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE) | Global Blood Therapeutics | | 1 Dec 2016 | 1 Jun 2019 |
| Fetal Hemoglobin Induction Treatment Metformin | Baylor College of Medicine | Metformin, Sickle Cell Anemia, Sickle Cell Disease, Hemoglobinopathies, Hemoglobin Disorder, Blood Disease, Hemoglobin Disease; Sickle-Cell, Thalassemia, Hemoglobin Disease | 1 Nov 2016 | 1 Nov 2022 |
| A Study of IMR-687 in Healthy Adult Volunteers | Imara, Inc. | Sickle Cell Disease, Sickle Beta 0 Thalassemia | 1 Nov 2016 | 1 Jun 2017 |
| SCD-PROMIS: A Software Platform to Enhance Self-efficacy and Patient-provider Engagement for Patients With Sickle Cell Pain | Children's Research Institute | | 1 Nov 2016 | 1 Sep 2018 |
| Hydoxyurea Exposure in Lactation: A Pharmacokinetics Study (HELPS) | Children's Hospital Medical Center, Cincinnati | Hydroxyurea | 1 Oct 2016 | 1 Nov 2019 |
| Monocytic Expression of Heme Oxidase-1 (HO-1) in Sickle Cell Patients and Correlation With the Humoral Immune Response to Vaccine and With Allo-immunization. | Francis Corazza | Sickle Cell Disease, Heme oxidase-1 (HO-1) | 1 Oct 2016 | 1 Oct 2018 |
| Oxygen Therapy and Pregnancy in Sickle Cell Disease | Assistance Publique - Hôpitaux de Paris | sickle cell disease, oxygen therapy, pregnancy, pregnancy in sickle cell disease | 1 Sep 2016 | 1 Jun 2021 |
| Mobile-Directly Observed Therapy on Adherence to Hydroxyurea | Muhimbili University of Health and Allied Sciences | Sickle cell disease, Hydroxyurea, Adherence, randomized trial, medication possession ratio | 1 Sep 2016 | 1 Aug 2017 |
| Macrolide Therapy to Improve Forced Expiratory Volume in 1 Second in Adults With Sickle Cell Disease | Vanderbilt University | sickle cell disease, pulmonary complications, reduced FEV1, azithromycin, lung function | 1 Sep 2016 | 1 Oct 2018 |
| The Effective and Safety of Thalidomide in NTDT | Xiao-Lin Yin | Thalidomide NTDT | 1 Sep 2016 | 1 Mar 2018 |
| Patient-Empowered Mobile Technology in Hospitalized Patients: Technology Resources to Understand Pain Assessment in Patients With Pain (TRU-PAIN) | Duke University | oncology, bone marrow transplant, sickle cell disease | 1 Aug 2016 | 1 Aug 2018 |
| Monitoring of Chimerism After Transplantation in Patients With β Thalassemia Major and the Treatment Strategies for the Reduction of Chimerism | First Affiliated Hospital of Guangxi Medical University | thalassemia, transplantation, chimerism, donor ymphocytes | 1 Aug 2016 | 31 Dec 2017 |
| Extended Evaluation of Deferasirox Film-coated Tablet (FCT) Formulation | Novartis Pharmaceuticals | Anemia., Myelodysplastic Syndrome, Thalassemia, Film coated tablet | 1 Jul 2016 | 1 Sep 2018 |
| Ketamine as an Adjuvant Therapy for Acute Vaso Occlusive Crisis in Pediatric Patients With Sickle Cell Disease | Georgia Regents University | | 1 Jul 2016 | 1 Jul 2018 |
| Abatacept for GVHD Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell Disease | Monica Bhatia | Sickle Cell Disease, Graft Versus Host Disease, Abatacept, Hematopoietic Stem Cell Transplantation | 1 Jul 2016 | 1 Dec 2017 |
| L-arginine Versus Sildenafil in Children With Beta Thalassemia Associated With Pulmonary Hypertension | Tanta University | | 1 Jul 2016 | 1 Dec 2023 |
| Transfusion Treatment in Patients With SCD | Società Italiana Talassemie ed Emoglobinopatie | | 1 Jul 2016 | 31 Dec 2020 |
| Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN 1503) | Medical College of Wisconsin | Sickle Cell Disease, Young Adults, Phase II Trial, Hematopoietic Cell Transplantation (HCT), Human Leukocyte Antigen (HLA) | 1 Jun 2016 | 1 Oct 2022 |
| Benefits of Nigella Sativa in Children With Beta Thalassemia Major | Tanta University | | 1 Jun 2016 | 1 Aug 2016 |
| Adjuvant Low-dose Ketamine in Pediatric Sickle Cell Vaso-occlusive Crisis | Children's Hospital & Research Center Oakland | Ketamine, Sickle Cell Disease, Vaso-Occlusive Crisis | 1 Jun 2016 | 1 Dec 2018 |
| Endothelial Monocyte-activating Polypeptide-II in Egyptian Sickle Patients | Ain Shams University | | 1 Jun 2016 | 1 Jun 2019 |
| Zinc Supplementation in Children With Sickle Cell Disease in Western Kenya | Lucas Otieno Tina, MD MSc | Children, Sickle Cell Disease, Zinc | 20 May 2016 | 19 Jan 2017 |
| Rejuvesol® Washed RBC in Sickle Cell Patients Requiring Frequent Transfusions | Duke University | | 1 May 2016 | 1 Jun 2017 |
| SACRED A Prospective Research Study to Reduce Stroke in Children With Sickle Cell Anemia | Children's Hospital Medical Center, Cincinnati | | 1 May 2016 | 1 Oct 2020 |
| HLA-Identical Sibling Donor Bone Marrow Transplantation for Individuals With Severe Sickle Cell Disease Using a Reduced Intensity Conditioning Regimen | National Guard Health Affairs | | 1 May 2016 | 1 Dec 2019 |
| Identifying Barriers and Strategies to Support Self-efficacy for Medication Adherence With Text Messaging | Vanderbilt University | | 1 May 2016 | 1 May 2017 |
| Study of Hydroxyurea to Treat Sickle Cell Disease | Mersin University | Sickle cell disease, Hydroxyurea, Compliance | 1 May 2016 | 1 Jul 2016 |
| A Multi-Center Study of Riociguat in Patients With Sickle Cell Diseases | Gregory J. Kato, MD | SCD, Sickle Cell Disease, Riociguat, Adempas | 1 Apr 2016 | 1 Jun 2019 |
| A Study of SANGUINATE for the Treatment of Vaso-occlusive Crisis (VOC) in Adult Sickle Cell Disease Patients | Prolong Pharmaceuticals | | 1 Apr 2016 | 1 Sep 2017 |
| The Feasibility of the PAINReportIt Guided Relaxation Intervention-INPATIENT | University of Florida | | 1 Apr 2016 | 1 Apr 2018 |
| Transplantation Using Reduced Intensity Approach for Patients With Sickle Cell Disease From Mismatched Family Donors of Bone Marrow | Emory University | bone marrow transplant | 1 Apr 2016 | 1 Dec 2020 |
| Improving Self-Management in Adolescents With Sickle Cell Disease | Children's Hospital Medical Center, Cincinnati | Anemia, Sickle Cell, Self Care, Adolescent, Young Adult, Disease Management, Behavioral Activation, Peer Communication | 1 Apr 2016 | 1 Apr 2018 |
| Coordinated HEalthcare for Complex Kids | University of Illinois at Chicago | care coordination, Children, Young adults, Chronic disease | 1 Apr 2016 | 31 Aug 2018 |
| Nonmyeloablative Conditioning for Mismatched Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Disease | Washington University School of Medicine | | 1 Mar 2016 | 1 May 2020 |
| Investigation of the Genetics of Hematologic Diseases | St. Jude Children's Research Hospital | Genetics, Whole genome sequencing, Pediatrics and hematology | 1 Mar 2016 | 1 Aug 2026 |
| Plasma DNA and Vascular Remodelling in Patients With Sickle Cell Disease | ADDMEDICA SASA | | 1 Mar 2016 | 1 Sep 2018 |
| Evaluating Thromboelastography (TEG) and ETP in Sickle Adults | Guy's and St Thomas' NHS Foundation Trust | | 1 Mar 2016 | 1 Jun 2016 |
| Myeloablative Conditioning and Haplo AlloSCT for Patients With Sickle Cell Disease | New York Medical College | stem cell transplantation, sickle cell disease, haploidentical, defibrotide | 1 Feb 2016 | 1 Dec 2021 |
| Screening for Alpha Thalassemia in Healthy Volunteers | National Heart, Lung, and Blood Institute (NHLBI) | Alpha Thalassemia, Double Deletion | 1 Feb 2016 | 1 Dec 2018 |
| A Phase 1 Study of Continuous Intravenous L-citrulline During Sickle Cell Pain Crisis or Acute Chest Syndrome | University of Mississippi Medical Center | sickle cell disease, citrulline | 1 Feb 2016 | 1 Dec 2017 |
| Arginine Therapy for Sickle Cell Disease Pain | Emory University | Arginine Therapy | 1 Jan 2016 | 1 Aug 2019 |
| SANGUINATE™ in Sickle Cell Disease Associated Leg Ulcer | Prolong Pharmaceuticals | Leg Ulcer, LU, SANGUINATE, QOL, SCD, Sickle Cell Disease, Sickle Cell, Anemia, Leg Wound, Leg, Skin Lesion | 1 Jan 2016 | 1 Jan 2017 |
| Multi-center Study of SC411 for Sickle Cell Disease | Sancilio and Company, Inc. | hemoglobin SS, hemoglobin SC, hemoglobin S/β°-thalassemia | 1 Jan 2016 | 1 Dec 2017 |
| Desmopressin as a Therapy for Bedwetting in Children With Sickle Cell Disease | Montefiore Medical Center | | 1 Jan 2016 | 1 Jun 2018 |
| Measures of Respiratory Health (MRH) | The Hospital for Sick Children | CF, Multiple Breath Washout, Lung Clearance Index, Pediatrics | 1 Jan 2016 | 1 Dec 2018 |
| Treatment of Adult Patients With Hemoglobin SC Disease (SCYTHE) | Baylor College of Medicine | | 1 Dec 2015 | 1 Dec 2020 |
| Effect of Spirulina on Serum Hyaluronic Acid in Beta Thalassemic Children With Hepatitis C | Tanta University | | 1 Dec 2015 | 1 Dec 2016 |
| Effect of Spirulina on Liver Fibrosis by Transient Elastography in Beta Thalassemic Children With Hepatitis C | Tanta University | | 1 Dec 2015 | 1 Dec 2016 |
| The Prevalence and Severity of HCV Infection in Thalassemia Major and Thalassemia Intermedia in Siriraj Hospital | Mahidol University | Transient elastography, Fibrotest | 1 Nov 2015 | 1 Feb 2017 |
| Sub-dissociative Intranasal Ketamine for Pediatric Sickle Cell Pain Crises | Cameroon Baptist Convention Health | Ketamine, Intranasal, Pain crisis, Vasoocclusive Pain, Sickle cell disease | 1 Oct 2015 | 1 Feb 2017 |
| the Potential Immunomodulatory Effects of Spirulina on Thalassemic Children | Tanta University | | 1 Oct 2015 | 1 Oct 2017 |
| Evaluation of the Impact of Renal Function on the Pharmacokinetics of SIKLOS ® (DARH) | ADDMEDICA SASA | Pharmacokinetics of Hydroxyurea | 1 Sep 2015 | 1 Jun 2016 |
| Prevention of Vaso-occlusive Painful Crisis by Using Omega-3 Fatty Acid Supplements | Sultan Qaboos University | Prevention of Vaso Occlusive Painful Crisis | 1 Sep 2015 | 1 Mar 2019 |
| Evaluation of the Hemostatic Potential in Sickle Cell Disease Patients | Brugmann University Hospital | Hemostatic potential, Sickle cell disease, Thrombin generation test | 1 Sep 2015 | 1 Sep 2017 |
| A Single-Dose Relative Bioavailability Study Of GBT440 300 mg Capsules in Healthy Subjects | Global Blood Therapeutics | | 1 Sep 2015 | 1 Feb 2016 |
| Drug Interaction Study of GBT440 With Caffeine, S-warfarin, Omeprazole, and Midazolam in Healthy Subjects | Global Blood Therapeutics | anemia, sickle cell | 1 Sep 2015 | 1 Feb 2016 |
| Nonpharmacological Approaches and Parental Education in Children With Sickle Cell Disease | Akdeniz University | sickle cell disease, nonpharmacological approaches, pain, parent, nurse | 1 Sep 2015 | 2 Feb 2016 |
| Comparison of Two Methods of Transfusion for Stroke Prevention in Sickle Cell | Chattanooga-Hamilton County Hospital Authority | | 1 Aug 2015 | 1 Jun 2017 |
| Comparison of Patient Centered Outcomes for People With SCD in the Acute Care Setting | Johns Hopkins University | | 1 Jul 2015 | 1 Dec 2018 |
| Safety Of Rivipansel (GMI-1070) In The Treatment Of One or More Vaso-occlusive Crises In Hospitalized Subjects With Sickle Cell Disease | Pfizer | Rivipansel, GMI-1070, Selectin Inhibitor, SCD, VOC | 1 Jul 2015 | 1 Feb 2020 |
| Sevuparin Infusion for the Management of Acute VOC in Subjects With SCD | Dilaforette AB | | 1 Jul 2015 | 1 Jun 2016 |
| Carbon Monoxide Measurement to Screen for Sickle Cell Disease | Children's Hospital & Research Center Oakland | Sickle Cell Anemia | 1 Jul 2015 | 1 Dec 2015 |
| Macitentan in Pulmonary Hypertension of Sickle Cell Disease | Boston University | Pulmonary Hypertension, Sickle Cell Disease, Pulmonary Arterial Hypertension, Macitentan | 1 Jul 2015 | 1 Dec 2016 |
| Evaluation of Repeat Administration of Purified Poloxamer 188 | Mast Therapeutics, Inc. | Sickle cell, vaso-occlusive crisis | 1 Jun 2015 | 1 Jun 2016 |
| Music Therapy in Sickle Cell Transition Study | University Hospital Case Medical Center | | 1 Jun 2015 | 1 Aug 2016 |
| Amlodipine as Adjuvant Treatment to Iron Chelation for Prevention of Cardiac Iron Overload in Thalassemia Patients | Kevin H.M. Kuo, MD, MSc, FRCPC | | 1 Jun 2015 | 1 Jun 2018 |
| SMART Mobile Application Technology Utilization in the Treatment of Sickle Cell Disease Post Day Hospital Discharge | Duke University | Sickle Cell Disease | 1 Jun 2015 | 1 May 2017 |
| A Study to Evaluate the Effect of Ticagrelor in Reducing the Number of Days With Pain in Patients With Sickle Cell Disease | AstraZeneca | Sickle cell disease, Young adults, Hestia2, Ticagrelor | 1 Jun 2015 | 1 Jun 2016 |
| A Study of the Absorption, Metabolism, and Excretion of GBT440 in Healthy Male Subjects | Global Blood Therapeutics | | 1 Jun 2015 | 1 Dec 2015 |
| Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias | ApoPharma | Iron overload, Sickle cell disease, Deferiprone, Ferriprox, Iron chelation | 1 May 2015 | 1 Oct 2018 |
| Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease | Emory University | | 1 May 2015 | 1 Jul 2019 |
| Gene Therapy for Transfusion Dependent Beta-thalassemia | IRCCS San Raffaele | Beta thalassemia, Gene therapy, Lentiviral vector | 1 May 2015 | 1 Aug 2019 |
| Study of SANGUINATE™ In the Treatment of Sickle Cell Disease Patients With Vaso-Occlusive Crisis | Prolong Pharmaceuticals | | 1 Apr 2015 | 1 Apr 2016 |
| Effect of Spirulina Compared to Amlodipine on Cardiac Iron Overload in Children With Beta Thalassemia | Tanta University | | 1 Apr 2015 | 1 Apr 2017 |
| Mental Health Treatment in Sickle Cell Disease | University of Pittsburgh | | 1 Mar 2015 | 1 Dec 2015 |
| Effect of Spirulina on Liver Iron Concentration in Beta Thalassemic Children With Hepatitis C | Tanta University | | 1 Mar 2015 | 1 Dec 2016 |
| Comparing Acute Pain Management Protocols for Patients With Sickle Cell Disease | Duke University | Sickle Cell Disease, Emergency Department, Vaso-occlusive Crisis, Pain Management, Pilot Project | 1 Feb 2015 | 1 Sep 2016 |
| Dose-Escalation Study of SCD-101 in Sickle Cell Disease | Invenux, LLC | Homozygous Sickle Cell Disease S/Beta 0 Thalassemia | 1 Feb 2015 | 1 Jan 2016 |
| Sperm DNA Damage in β-thalassemia Major: Is There a Role for Antioxidants? | Ain Shams University | | 1 Feb 2015 | 1 Dec 2017 |
| A Study of the Safety, Blood Levels and Biological Effects of GBT440 in Healthy Subjects and Subjects With Sickle Cell Disease | Global Blood Therapeutics | | 1 Jan 2015 | 1 May 2016 |
| Ledipasvir/Sofosbuvir Fixed-Dose Combination for 12 Weeks in Genotype 1 or 4 HCV Infected Adults With Sickle Cell Disease | Gilead Sciences | Sickle Cell Disease, Gilead, Hepatitis C | 1 Dec 2014 | 1 Mar 2016 |
| Treatment of Hemoglobin Sickle Cell Disease | Baylor College of Medicine | Hemoglobin SC disease, hydroxyurea, quality of life, viscosity, red cell density | 1 Dec 2014 | 1 Dec 2019 |
| Biological, Genetic and Environmental Involved in the Complications of Sickle Cell Disease | Erasme University Hospital | sickle cell disease, nephropathy | 20 Oct 2014 | 1 Jan 2025 |
| PATient Navigator to rEduce Readmissions | University of Illinois at Chicago | Patient Navigators, Readmissions, Peer Coaching | 1 Oct 2014 | 1 Apr 2016 |
| A New Reagent Assay Examining Natural Parvovirus B19 Infection in Sickle Cell Disease | St. Jude Children's Research Hospital | Parvovirus B19 Infection | 1 Oct 2014 | 1 May 2017 |
| ACE-536 Extension Study - Beta Thalassemia | Acceleron Pharma, Inc. | | 1 Oct 2014 | 1 Apr 2017 |
| Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia | Sideris Pharmaceuticals | Chelation, Iron Overload, Thalassemia, Transfusion, Iron Chelation | 1 Oct 2014 | 1 Jul 2015 |
| Feasibility and Efficacy of a Home-based, Computerized Cognitive Training Program in Pediatric Sickle Cell Disease | Steven J. Hardy | Neurocognitive, Working memory, Cogmed, Cognitive training | 1 Oct 2014 | 1 Aug 2017 |
| Vitamin D3 in Patients With Sickle Cell Disease | Icahn School of Medicine at Mount Sinai | Sickle Cell Disease, Vitamin D | 1 Oct 2014 | 1 Dec 2017 |
| Assessment of Tolerance of Mobilizing Peripheral Hematopoietic Stem Cells by Plerixafor in Sickle Cell Patients | Assistance Publique - Hôpitaux de Paris | Sickle cell disease, Open monocenter study, Phase I/II, hematopoietic stem cells, Plerixafor | 1 Sep 2014 | 1 Jun 2017 |
| A Pharmacokinetic (PK) and Pharmacodynamic (PD) Dose-ranging Phase II Study of Ticagrelor Followed by a 4 Weeks Extension Phase in Paediatric Patients With Sickle Cell Disease | AstraZeneca | sickle cell anemia, paediatric | 1 Sep 2014 | 1 Sep 2015 |
| Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases | Bellicum Pharmaceuticals | Severe Combined Immune Deficiency, Congenital T-cell Defect, Congenital T-cell Deficiency, Chronic Granulomatous Disease, Shwachman Diamond Syndrome, Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Sickle Cell Disease, Thalassemia, Mucopolysaccharidosis, Sphingolipidoses | 1 Sep 2014 | 1 Dec 2018 |
| Music Therapy in Sickle Cell Pain RCT | University Hospital Case Medical Center | Music Therapy, Sickle Cell Disease, Pain | 1 Sep 2014 | 1 Jun 2016 |
| Decision Aid for Therapeutic Options In Sickle Cell Disease | Emory University | Hydroxyurea, Bone marrow transplant, Chronic blood transfusion, Treatment options for sickle cell disease, Decision making | 1 Sep 2014 | 1 Dec 2016 |
| Denosumab vs Placebo in Patients With Thalassemia Major and Osteoporosis | Ersi Voskaridou | | 1 Sep 2014 | 1 Dec 2016 |
| A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in Severe Sickle Cell Disease | bluebird bio | | 1 Aug 2014 | 1 Jan 2018 |
| Haploidentical Hematopoietic Stem Cell Transplantation for Children With Sickle Cell Disease and Thalassemia Using CD34+ Positive Selected Grafts | Catherine Bollard | | 1 Aug 2014 | 1 Dec 2019 |
| Fertility Preservation in Women Who Will be Undergoing Gonadotoxic Therapy, Hematopoietic Stem Cell Transplantation, and in Women With Sickle Cell Disease | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Bone Marrow Transplant, Fertility, Ovary | 1 Aug 2014 | 1 Aug 2019 |
| Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease | National Heart, Lung, and Blood Institute (NHLBI) | Hydroxyurea, Sickle Cell Disease, Fetal Hemoglobin Induction | 1 Aug 2014 | 1 May 2018 |
| Working Memory and School Readiness in Preschool-Aged Children With Sickle Cell Disease | St. Jude Children's Research Hospital | | 1 Aug 2014 | 1 Mar 2017 |
| Study of Beet Juice for Patients With Sickle Cell Anemia | Daniel Kim-Shapiro | Erythrocyte Deformability, Platelet Activation, Platelet Aggregation, Nitrite, Sickle Cell Anemia, Diet Therapy | 1 Jul 2014 | 1 Apr 2015 |
| Stem Cell Gene Therapy for Sickle Cell Disease | Donald B. Kohn, M.D. | Sickle Cell Disease (SCD), Gene Therapy, Lentiviral Vector, Beta Globin | 1 Jul 2014 | 1 Apr 2018 |
| Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Study Of PF-04447943, Co-Administered With And Without Hydroxyurea, In Subjects With Stable Sickle Cell Disease | Pfizer | | 1 Jun 2014 | 1 Apr 2015 |
| Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia Major | Affiliated hospital of guangxi medical university,china | beta-Thalassemia major | 1 Jun 2014 | 1 Dec 2018 |
| Patient Centered Comprehensive Medication Adherence Management System in Patients With Sickle Cell Disease | Emory University | | 1 Jun 2014 | 1 Dec 2018 |
| Sickle Cell Disease (SCD) Decision Aid | Emory University | | 15 May 2014 | 31 Mar 2015 |
| Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet) | Novartis Pharmaceuticals | Iron overload, Chelation, Thalassemia, Myelodysplastic syndrome (MDS), Transfusional hemisiderosis, Deferasirox, ICL670, Dispersible tablet, Film-coated tablet | 1 May 2014 | 1 Dec 2015 |
| Use of Mobile Technology for Intensive Training in Medication Management | Duke University | Sickle cell, Thalassemia, Adherence | 1 May 2014 | 1 May 2016 |
| Medication Adherence in Youth With Sickle Cell Disease (SCD) | St. Jude Children's Research Hospital | Medication Adherence, Hydroxyurea | 1 May 2014 | 1 May 2015 |
| Insights Into Microbiome and Environmental Contributions to Sickle Cell Disease and Leg Ulcers | National Human Genome Research Institute (NHGRI) | Genetic Disorders, Sickle Cell Disease, African American, Leg Ulcers, Skin | 1 May 2014 | 1 Apr 2016 |
| Sickle Cell Hemoglobinopathies and Bone Health | University of Connecticut Health Center | Sickle Cell Disease (SCD), Sickle Cell Trait (SCT) | 1 May 2014 | 1 May 2017 |
| Feasibility Study of Unfractionated Heparin in Acute Chest Syndrome | University of Pittsburgh | Sickle cell disease, Acute chest syndrome, Hemoglobinopathy, Hemolytic anemia, Heparin, Anticoagulant | 1 Apr 2014 | 1 Apr 2016 |
| Gum Arabic as Fetal Hemoglobin Agent in Sickle Cell Anemia | Al-Neelain University | | 1 Apr 2014 | 1 Jan 2015 |
| Androgen Regulation of Priapism in Sickle Cell Disease | Johns Hopkins University | Priapism, Sickle cell disease, Male | 1 Mar 2014 | 1 Sep 2017 |
| Hematopoietic Stem Cell Transplant for Sickle Cell Disease | Case Comprehensive Cancer Center | Sickle Cell Disease, Sickle Cell Anemia, GvHD, fludarabine | 1 Mar 2014 | 1 Nov 2016 |
| Effect of Deferasirox on Endocrine Complications in Subjects With Transfusion Dependent Thalassemia | Novartis Pharmaceuticals | Endocrine complications, transfusion dependent thalassemia | 1 Mar 2014 | 1 Mar 2018 |
| Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure | National Heart, Lung, and Blood Institute (NHLBI) | Sickle Cell Disease, Allogeneic Hematopoietic Stem Cell Transplant, Pentostatin (Nipent), Cyclophosphamide, Alemtuzumab (Campath) | 1 Mar 2014 | 1 Aug 2021 |
| Sickle Cell Clinical Research and Intervention Program | St. Jude Children's Research Hospital | Sickle Cell Anemia, Sickle Cell Disease, Survival, Mortality, End-Order Dysfunction | 1 Mar 2014 | 1 Dec 2044 |
| Hepcidin Levels in Sickle Cell Disease (SCD) | Kenneth Ataga, MD | sickle cell disease | 1 Mar 2014 | 1 May 2015 |
| Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias | ApoPharma | sickle cell disease, Iron overload, Deferiprone, Ferriprox, deferoxamine, Chelation | 1 Feb 2014 | 1 Mar 2016 |
| Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia. | Novartis Pharmaceuticals | thalassemia, spleen enlargement | 1 Feb 2014 | 1 Feb 2016 |
| Amlodipine for Myocardial Iron in Thalassemia | Aga Khan University | Thalassemia, Amlodipine, Chelation, T2*, MRI, Myocardial Iron | 1 Feb 2014 | 1 May 2015 |
| The Effect of Rivaroxaban in Sickle Cell Disease | University of North Carolina, Chapel Hill | sickle cell anemia, sickle cell disease, rivaroxaban, direct Xa inhibition, coagulation, anticoagulation | 1 Feb 2014 | 1 Sep 2016 |
| Novel Use Of Hydroxyurea in an African Region With Malaria | University of Minnesota - Clinical and Translational Science Institute | Hydroxyurea | 1 Jan 2014 | 1 Jan 2017 |
| Allograft for Sickle Cell Disease and Thalassemia | University of Texas Southwestern Medical Center | Sickle Cell Disease, Thalassemia | 1 Jan 2014 | 1 Jan 2018 |
| Reduced Intensity Conditioning in Patients Aged ≤30 With Non-Malignant Disorders Undergoing Cord Blood Transplantation | University of Pittsburgh | Severe Combined Immune Deficiency (SCID) with NK cell activity, Omenn Syndrome, Bare Lymphocyte Syndrome (BLS), Combined Immune Deficiency (CID) syndromes, Combined Variable Immune Deficiency (CVID) syndrome, Wiskott-Aldrich Syndrome, Leukocyte adhesion deficiency, Chronic granulomatous disease (CGD), X-linked Hyper IgM (XHIM) syndrome, IPEX syndrome, Chediak -Higashi Syndrome, Autoimmune Lymphoproliferative Syndrome (ALPS), Hemophagocytic Lymphohistiocytosis (HLH) syndromes, Lymphocyte Signaling defects, Other primary immune defects where HSCT may be beneficial, Dyskeratosis Congenita (DC), Congenital Amegakaryocytic Thrombocytopenia (CAMT), Osteopetrosis, Mucopolysaccharidoses, Hurler syndrome (MPS I), Hunter syndrome (MPS II), Sanfilippo syndrome (MPS II), Leukodystrophies, Krabbe Disease, also known as globoid cell leukodystrophy, Metachromatic leukodystrophy (MLD), X-linked adrenoleukodystrophy (ALD), Other inherited metabolic disorders, alpha mannosidosis, Other inheritable metabolic diseases where HSCT may be beneficial., Thalassemia major, Sickle cell disease (SCD), Diamond Blackfan Anemia (DBA), other congenital transfusion dependent anemias | 1 Dec 2013 | 1 Nov 2018 |
| Survey in a Population of Sickle Cell Disease Patients to Evaluate the Transition Between the Queen Fabiola Children Hospital and the CHU Brugmann Hospital, and the Quality of the Hospital Care Within the CHU Brugmann Hospital. | Brugmann University Hospital | pediatric hospital care, adult hospital care, sickle cell disease | 1 Dec 2013 | 1 Dec 2016 |
| Phase 2 Study of Montelukast for the Treatment of Sickle Cell Anemia | Vanderbilt University | | 1 Nov 2013 | 1 Oct 2017 |
| SCD-Haplo: A Phase II Study of HLA-Haploidentical Stem Cell Transplation to Treat Clinically Aggressive Sickle Cell Disease | Damiano Rondelli, MD | | 1 Nov 2013 | 1 Oct 2018 |
| CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant | Diane George, MD | Unrelated donor transplant, Allogenic Stem Cell Transplant, Adult Bone Marrow Transplant, Pediatric Bone Marrow Transplant, Related donor transplant, Haploidentical donor transplant, Peripheral blood stem cell transplantation, Non-malignant disease, Malignant disease, Bone marrow failure syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Schwachman Diamond Syndrome, Primary Immunodeficiency Syndrome, Acquired Immunodeficiency Syndrome, Histiocytic Syndrome, Familial Hemophagocytic Lymphocytosis, Lymphohistiocytosis, Macrophage Activation Syndrome, Langerhans Cell Histiocytosis (LCH), Hemoglobinopathies, Reduced-Intensity Conditioning, Sickle Cell Disease, Sickle Cell-beta-thalassemia | 1 Nov 2013 | 1 Dec 2017 |
| Added Value of Speckle Tracking in the Evaluation of Patients With Sickle Cell Disease | Brugmann University Hospital | Sickle Cell Disease, Echocardiography, Speckle tracking | 1 Nov 2013 | 1 Mar 2016 |
| Feasibility of a Stress Reduction Intervention Study in Sickle Cell Disease | University of Illinois at Chicago | | 1 Nov 2013 | 1 Nov 2014 |
| Ph. 2 Study of MP4CO to Treat Vaso-occlusive Sickle Crisis | Sangart | Sickle cell anemia, Sickle cell disease, Sickling crisis, Vaso-occlusive crisis, Carboxyhemoglobin, Oxygen therapeutic, Ischemic rescue therapy, Hemoglobin solution, Pegylated hemoglobin | 1 Oct 2013 | 1 Oct 2015 |
| Treatment of Iron Overload Requiring Chelation Therapy | Shire Development LLC | | 1 Oct 2013 | 1 Jul 2015 |
| Pain Management in Children and Young Adults With Sickle Cell Disease | St. Jude Children's Research Hospital | Pain control | 1 Oct 2013 | 1 Oct 2017 |
| Evaluation of Nocturnal Enuresis and Barriers to Treatment Among Pediatric Patients With Sickle Cell Disease | St. Jude Children's Research Hospital | Nocturnal enuresis, Barriers to treatment | 1 Oct 2013 | 1 Oct 2015 |
| Hydroxyurea Therapy, Sickle Cell Disease, NDEPTH | Baylor College of Medicine | Sickle cell, Hydroxyurea, Dose prediction, Maximum tolerated dose | 1 Oct 2013 | 1 Oct 2018 |
| Utility of Fibroscan in Estimating Hepatic Iron Concentration | University of British Columbia | Beta Thalassemia Major | 1 Oct 2013 | 1 Oct 2014 |
| Abciximab (ReoPro) as a Therapeutic Intervention for Sickle Cell Vaso-Occlusive Pain Crisis | St. Louis University | Sickle Cell Pain Crisis, Abciximab, Integrins, Cell Adhesion Molecules | 1 Sep 2013 | 1 Sep 2015 |
| Deferasirox BID in Transfusion Dependent Thalassemia Patients With Indadequate Response to High Doses | Novartis Pharmaceuticals | | 1 Sep 2013 | 1 Sep 2015 |
| Hydroxyurea Adherence for Personal Best in Sickle Cell Treatment: HABIT | Columbia University | Sickle cell disease, Medication adherence, Hydroxyurea, Community health worker intervention, Sickle Cell Treatment | 1 Sep 2013 | 1 May 2015 |
| Pneumococcal Vaccination for Splenectomised Thalassemia Major Patients in Indonesia | Fakultas Kedokteran Universitas Indonesia | | 1 Sep 2013 | 1 Feb 2014 |
| Quantifying the Presence of Lung Disease and Pulmonary Hypertension in Children With Sickle Cell Disease | Duke University | sickle cell disease, pulmonary hypertension, airway hyperreactivity, acute chest syndrome | 1 Aug 2013 | 1 Feb 2020 |
| Improving Disease Knowledge in Adolescents With Sickle Cell Disease | The University of The West Indies | Educational intervention, Formal Counselling, Knowledge, Illness Perception | 1 Aug 2013 | 1 Aug 2015 |
| Evaluation of Sickle Cell Liver Disease | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Microbiome, Sickle Cell Disease, Bone Marrow Transplant | 1 Aug 2013 | 1 May 2018 |
| Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain Crises | Selexys Pharmaceuticals Corporation | SelG1, P-selectin, monoclonal antibody, sickle cell disease, sickle cell anemia, sickle cell, pain crisis, pain crises, vasoocclusion, vaso-occlusion, priapism, hepatic sequestration, splenic sequestration, chest syndrome | 1 Jul 2013 | 1 Aug 2015 |
| An Open Label Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Efficacy of Deferasirox Administered to Chinese Patients With β-thalassemia Major Aged From 2 to Less Than 6 Years Old | Novartis Pharmaceuticals | deferasirox | 1 Jun 2013 | 1 Oct 2014 |
| Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients | Consorzio per Valutazioni Biologiche e Farmacologiche | chronic iron overload, hereditary haemoglobinopathy, beta thalassaemia major, chelating agents, deferiprone, deferasirox, children, paediatrics | 1 Jun 2013 | 1 Dec 2014 |
| Exhaled Carbon Monoxide as a Marker of Hemolysis in Sickle Cell Disease- an Exploratory Study | University Hospital Case Medical Center | carbon mono oxide, sickle cell, hemolysis | 1 Jun 2013 | 1 Jun 2015 |
| Nitrous Oxide Analgesia Vaso-occlusive Crisis | Columbia University | Sickle cell disease, Vaso-occlusive crisis, Nitrous oxide, Emergency department | 1 Jun 2013 | 1 Jun 2014 |
| Efficacy, Safety Study and Benefit of Alkali Therapy in Sickle Cell Disease | University Hospital Case Medical Center | Sickle cell disease, Chronic kidney disease, Bicarbonate therapy | 1 Jun 2013 | 1 Jun 2015 |
| Reproductive Capacity and Iron Burden in Thalassemia | Children's Hospital & Research Center Oakland | Infertility, Thalassemia, Iron overload, Pituitary Iron Load, Oxidative Stress, REPRODUCTIVE CAPACITY AND ASSOCIATION TO IRON BURDEN AND CHELATION PATTERNS IN THALASSEMIA MAJOR PATIENTS | 1 Jun 2013 | 1 Jun 2016 |
| Evaluation of the Free α-hemoglobin Pool in the Red Blood Cells : Prognostic Marker and Severity Index in Thalassemic Syndromes | Assistance Publique - Hôpitaux de Paris | Alpha-Hemoglobin Stabilizing Protein, alpha-Hb pool, thalassemias, diagnostic method, biomarker | 1 Jun 2013 | 1 Jun 2016 |
| Absorption, Metabolism, and Excretion of a Single Dose of Ferriprox® in Patients With Sickle Cell Disease | ApoPharma | sickle cell disease, iron overload, pharmacokinetics, Ferriprox, deferiprone | 1 May 2013 | 1 Jul 2013 |
| Non-Myeloablative Conditioning and Bone Marrow Transplantation | Vanderbilt-Ingram Cancer Center | | 1 May 2013 | 1 May 2016 |
| Effect of Exercise With and Without HMB on Body Composition and Muscle Strength in Sickle Cell Anaemia | The University of The West Indies | | 30 Apr 2013 | 15 Nov 2019 |
| A Phase II Trial of Regadenoson in Sickle Cell Anemia | Dana-Farber Cancer Institute | | 1 Apr 2013 | 1 Apr 2015 |
| N-Acetylcysteine in Patients With Sickle Cell Disease | Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) | Sickle Cell Disease, Sickle Cell Anemia, Pain, N-Acetylcysteine, Acetylcysteine, Oxidative stress | 1 Apr 2013 | 1 Oct 2014 |
| A Study Evaluating the Safety and Efficacy of the Gene Therapy LentiGlobin® BB305 Drug Product in Subjects With Beta-Thalassemia Major | Bluebird Bio | gene therapy, beta thalassemia, hemoglobin, anemia, CD34 | 1 Mar 2013 | 1 Jul 2017 |
| A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD) | Eli Lilly and Company | | 1 Mar 2013 | 1 Jun 2015 |
| CD34+ Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation for Non-Malignant Disease | Diane George, MD | Unrelated donor transplant, Haploidentical donor transplant, Peripheral blood stem cell transplantation, CD34+ selection, Non-malignant disease, Bone marrow failure syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Schwachman Diamond Syndrome, Primary Immunodeficiency Syndrome, Acquired Immunodeficiency Syndrome, Histiocytic Syndrome, Familial Hemophagocytic Lymphocytosis, Lymphohistiocytosis, Macrophage Activation Syndrome, Langerhans Cell Histiocytosis (LCH), Hemoglobinopathies, Sickle Cell Disease, Sickle Cell-beta-thalassemia | 1 Mar 2013 | 1 Mar 2017 |
| Safety, Pharmacokinetic, and Pharmacodynamic Study of NKTT120 in Adult Patients With Stable Sickle Cell Disease (SCD) | NKT Therapeutics | Sickle cell disease | 1 Feb 2013 | 1 Aug 2014 |
| Sickle Cell Disease - Stroke Prevention in Nigeria Trial | Vanderbilt University | transcranial Doppler, stroke, sickle cell anemia, Nigeria, hydroxyurea, low income country | 1 Feb 2013 | 1 Aug 2014 |
| Implications of a Paediatrician-psychologist Tandem for Sickle Cell Disease Care and Impact on Cognitive Functioning | Fondation Ophtalmologique Adolphe de Rothschild | paediatric-psychological partnership, child, mental health, integrated care, well-being | 1 Feb 2013 | 1 Apr 2014 |
| Microvascular and Fibrosis Imaging Study | Duke University | Autoimmune diseases with cutaneous involvement, Systemic sclerosis, Scleroderma, Morphea, Dermatomyositis, Cutaneous lupus, Vasculitis, Sickle Cell Disease, Graft versus Host Disease | 1 Jan 2013 | 1 Jan 2017 |
| Vaporized Cannabis for Chronic Pain Associated With Sickle Cell Disease | University of California, San Francisco | | 1 Jan 2013 | 1 Mar 2016 |
| Anthropometric Measurements in Children Having Transfusion-dependent Beta Thalassemia | Aga Khan University | | 1 Jan 2013 | 31 Dec 2014 |
| Study to Evaluate Efficacy and Safety of S303 Treated Red Blood Cells (RBCs)in Subjects With Thalassemia Major Requiring Chronic RBC Transfusion | Cerus Corporation | S303 treated RBCs | 1 Dec 2012 | 1 Jun 2014 |
| Pharmacokinetic Study of Deferiprone in Paediatric Patients | Consorzio per Valutazioni Biologiche e Farmacologiche | chronic iron overload, hereditary haemoglobinopathy, beta thalassaemia major | 1 Dec 2012 | 1 Apr 2013 |
| Evaluation of Purified Poloxamer 188 in Children in Crisis (EPIC) | Adventrx Pharmaceuticals | sickle cell disease, vaso-occlusive crisis | 1 Dec 2012 | 1 Aug 2015 |
| An Epidemiological Study to Assess Iron Overload Using MRI in Patients With Transfusional Siderosis (TIMES Study) | Novartis Pharmaceuticals | | 1 Dec 2012 | 1 Jul 2014 |
| Study of ACE-536 to Evaluate the Effects of ACE-536 in Patients With Beta-thalassemia Intermedia | Acceleron Pharma, Inc. | | 1 Dec 2012 | 1 Nov 2014 |
| Evaluation of Spectra Optia RBC Exchange in Sickle Cell Patients | CaridianBCT | | 1 Nov 2012 | 1 May 2013 |
| Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders | Massachusetts General Hospital | Kidney, Chronic Kidney Disease, CKD, Bone Marrow, Bone Marrow Transplant, BMT, Leukemia, AML, ALL, CML, CLL, MM, NHL, MDS | 1 Nov 2012 | 1 Jan 2015 |
| Zinc and Diabetes in Patients With Thalassemia: a Pilot Study | Children's Hospital & Research Center Oakland | Thalassemia, Diabetes, Zinc, Iron-Overload | 1 Nov 2012 | 1 Dec 2014 |
| The Use of Warmed Saline in Vaso-occlusive Episodes | Nationwide Children's Hospital | | 1 Nov 2012 | 1 Jun 2014 |
| Single-center Prospective Evaluation of Sickle Cell Patient Care in the CHU Brugmann Emergency Department | Brugmann University Hospital | Sickle Cell Disease, Vaso-occlusive crisis, Emergency management | 1 Nov 2012 | 1 May 2015 |
| An Open Label, Multi-center, Efficacy and Safety Study of Deferasirox in Iron Overloaded Patients With Non-transfusion Dependent Thalassemia | Novartis Pharmaceuticals | Non-transfusion dependent thalassemia, NTDT | 1 Oct 2012 | 1 Apr 2014 |
| Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Observational Study II Protocol | National Heart, Lung, and Blood Institute (NHLBI) | | 1 Oct 2012 | 1 Dec 2016 |
| Cognitive Rehabilitation in Sickle Cell Disease | Duke University | sickle cell disease, pediatric, learning disability, cognitive impairment, memory, attention, Children with sickle cell disease | 1 Oct 2012 | 1 Mar 2014 |
| Carbon Monoxide Monitor for the Measurement of End-Tidal Carbon Monoxide Levels in Children With or Without Hemolysis | Ashutosh Lal | | 1 Oct 2012 | 1 Oct 2013 |
| Sickle Cell Trait in Football Players | University of South Florida | Sickle Cell Trait, Football, Sickling, Heat Illness, SCT | 1 Oct 2012 | 1 Aug 2013 |
| Enhancing Use of Hydroxyurea In Sickle Cell Disease Using Patient Navigators | Virginia Commonwealth University | sickle cell disease, hydroxyurea, patient navigator, community health worker, public health worker, fetal hemoglobin, health services research | 1 Oct 2012 | 1 Aug 2016 |
| Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia | Children's Hospital of Philadelphia | Thalassemia, Chelation, Transfusion, Safety | 1 Sep 2012 | 1 Aug 2015 |
| Unrelated Umbilical Cord Blood Following HLA-haploidentical Hematopoietic Stem Cell Transplantation in Patients With β-thalassemia Major | Nanfang Hospital of Southern Medical University | Cord Blood Transplant, Haploidentical HSCT, Thalassemia major | 1 Sep 2012 | 1 Jun 2015 |
| ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene | Memorial Sloan-Kettering Cancer Center | BUSULFAN, G-CSF, CliniMACS-CD34 Reagent System, Blood transfusion, 10-164, Autologous CD34+ cells transduced with TNS9.3.55, a lentiviral vector encoding the human β-globin gene | 1 Jul 2012 | 1 Jul 2014 |
| Liver Fibrosis in Sickle Cell Disease | University of Miami | sickle cell disease, hemochromatosis, liver fibrosis, liver cirrhosis, FibroScan, MRI R2*, liver iron | 1 Jul 2012 | 1 Dec 2017 |
| Post Hematopoietic Stem Cell Transplantation | Novartis Pharmaceuticals | hematopoietic stem cell transplantation, beta-thalassemia major, deferasirox, iron overload | 1 Jun 2012 | 1 Jul 2014 |
| Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin. | Ain Shams University | Hydroxyurea, Erythropoitin therapy, Thalassemia intermedia | 1 Jun 2012 | 1 Dec 2012 |
| Immunomodulatory Effects of Silymarin in Patients With Beta-Thalassemia Major | Shiraz University of Medical Sciences | Focus, immunomodulatory effect, of silymarin, on cell mediated immunity, Beta-Thalassemia, major patients. | 1 Jun 2012 | 1 Sep 2012 |
| Use of a Mobile-based App for SCD Patients | Jude C. Jonassaint | Sickle cell, Pain | 1 Jun 2012 | 1 Jun 2014 |
| Trial of HQK-1001 in Beta Thalassemia Intermedia in Lebanon | Boston University | Thalassemia intermedia, Fetal hemoglobin, Hemoglobin | 1 May 2012 | 1 Jan 2013 |
| Massive Iron Deposit Assessment | St. Jude Children's Research Hospital | Sickle cell disease, Thalassemia, Hemochromatosis, Cancer | 1 Apr 2012 | 1 Apr 2017 |
| Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia. | Celgene Corporation | Beta-Thalassaemia | 1 Mar 2012 | 1 Jun 2014 |
| Study of SDMB in Beta Thalassemia Intermedia in Thailand | Boston University | Thalassemia, Anemia, Fetal hemoglobin | 1 Mar 2012 | 1 Dec 2012 |
| Multicenter Observational Study on Myocardial Iron Overload in 3 Multitransfused Populations | Assistance Publique - Hôpitaux de Paris | Thalassemia, sickle cell disease, myelodysplasia, transfusion, chelation | 1 Mar 2012 | 1 Mar 2015 |
| Effects of Transfusion of Older Red Blood Cells on Patients With Hemoglobinopathies | Columbia University | iron, transfusion, red blood cells, sickle cell disease, thalassemia, blood donors | 1 Feb 2012 | 1 Feb 2015 |
| Study Of Efficacy,Safety of Combined Deferasirox and Deferiprone Versus Combined Deferiprone and Desferal In Conditions of Iron Overload | Ain Shams University | Iron chelation, Iron balance, Secondary iron overload, deferoxamine, deferasirox, Deferiprone, 1- Beta-thalassemia major patients;, Patients with high iron stores, Serum ferritin consistently > 2500 mcg/l and or increasing trend over previous 12 months, Liver iron >14 mg/g dry weight- by R2 MRI, 2- Sickle cell disease, 3- Other causes of transfusional iron hemosiderosis | 1 Feb 2012 | 1 Feb 2013 |
| Congenital Heart Surgery in Pediatric Patients With Beta-Thalassemia Major | Ain Shams University | | 15 Jan 2012 | 30 Aug 2019 |
| Prevalence of Pulmonary Hypertension (PAH) in Patients With Thalassemia | Ente Ospedaliero Ospedali Galliera | Thalassemia, Pulmonary Arterial Hypertension | 1 Jan 2012 | 1 Jan 2013 |
| Safety Study of MP4CO in Adult Sickle Cell Patients | Sangart | Sickle cell anemia, Sickle cell disease, Sickling crisis, Vaso-occlusive crisis, Carboxyhemoglobin, Oxygen therapeutic, Hemoglobin solutions, Pegylated hemoglobin | 1 Jan 2012 | 1 Mar 2013 |
| Effect of Metoprolol on Thalassemia Cardiomyopathy | Shiraz University of Medical Sciences | Thalassemia cardiomyopathy, metoprolol succinate, echocardiography, six minute walk test | 1 Jan 2012 | 1 Dec 2012 |
| Functional Neuroimaging of Pain Using EEG and fMRI | University of Minnesota - Clinical and Translational Science Institute | Sickle Cell | 1 Jan 2012 | 1 May 2015 |
| Demographic, Clinical, Laboratory and Genetical Characteristics of Patients With Beta Thalassemia Intermedia | HaEmek Medical Center, Israel | Beta globin gene, Alfa globin gene, xmn1 polymorphism | 1 Oct 2011 | 1 Dec 2012 |
| A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) | Center for International Blood and Marrow Transplant Research | | 1 Oct 2011 | |
| Providing Access to Cord Blood Units for Transplants | National Cancer Institute (NCI) | Adult and Pediatric, Unlicensed Cord Blood Unit IND, NMDP, Transplant, Hematologic Malignancies, Leukemia, Multiple Myeloma, Non-Hodgkin Lymphoma, CML, Chronic Myelogenous Leukemia, Myelodyplastic Syndrome, MDS, Thalassemia, Lysosomal Storage Diseases | 1 Oct 2011 | 1 Nov 2012 |
| Amlodipine in the Prevention and Treatment of Iron Overload in Patients With Thalassemia Major | University of Campinas, Brazil | Magnetic resonance imaging, Thalassemia, Iron overload | 1 Aug 2011 | 1 Dec 2013 |
| Study of Effects of YisuiShengxueGranules on Thalassemia | Guang'anmen Hospital of China Academy of Chinese Medical Sciences | traditional chinese medicine and thalassemia | 1 Jul 2011 | 1 Jan 2012 |
| Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload | Shire Development LLC | Beta-Thalassemia, Sickle Cell Anemia, Transfusional iron overload, Iron Overload, Iron Chelation | 1 Jul 2011 | 1 Dec 2013 |
| The Effect of Vitamin D Supplementation on Calcium Excretion in Thalassemia: a Dose Response Study | Weill Medical College of Cornell University | Thalassemia, hypercalciuria, vitamin D, To conduct a pilot study to determine the effect of various doses of, vitamin D supplementation on vitamin D stores and their association with calcium excretion | 1 Jun 2011 | 1 Dec 2014 |
| Stem Cell Transplantation for Sickle Cell Anemia | Hackensack University Medical Center | Stem cell transplant, Sickle cell, Stem cell transplantation, Related, Unrelated | 1 Jun 2011 | 1 Jun 2018 |
| Patient-Provider Tools to Improve the Transition to Adult Care in Sickle Cell Disease | Children's Hospital Medical Center, Cincinnati | Anemia, Sickle Cell, Transition to Adult Care, Self Care, Personal Electronic Health Records, Adolescent, Young Adult, Disease Management, Physician-Patient Relations | 1 Jun 2011 | 1 Jul 2015 |
| Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies | University of Louisville | other hemoglobinopathies, chimerism, sickle cell, thalassemia, Marrow/Enriched Hematopoetic Stem Cell Transplant | 1 May 2011 | 1 May 2030 |
| High-Tc Susceptometer to Monitor Transfusional Iron Overload | Columbia University | Iron, Iron overload, Hepatic iron concentration, High-temperature superconductivity, Magnetic susceptibility, Susceptometer | 1 Mar 2011 | 1 May 2014 |
| Topical Sodium Nitrite for Chronic Leg Ulcers in Adult Patients With Blood Disorders | National Heart, Lung, and Blood Institute (NHLBI) | Sickle Cell Disease, Hemolysis, Anemia, Nitric Oxide, Blood Flow, Leg Ulcer, Sickle Cell Anemia, Thalassemia, Hemolytic Blood Disorder | 1 Mar 2011 | 1 Apr 2013 |
| Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease | Abdullah Kutlar | sickle cell anemia, sickle cell thalassemia, HDAC inhibitor, hemoglobin F, sickle beta thalassemia | 1 Nov 2010 | 1 Dec 2014 |
| Evaluating the Safety and Effectiveness of Mozobil Mobilization in Adults With Beta-Thalassemia Major | University of Washington | Beta Thalassemia Major, Hematopoietic Stem Cell Mobilization, Gene Transfer Techniques | 1 Oct 2010 | 1 Dec 2012 |
| A Randomized Trial of LOVAZA in Pediatric Sickle Cell Disease (SCD) | Thomas Jefferson University | Sickle Cell Anemia, Sickle Cell Disease, Hemoglobin SS Disease, Hemoglobin S beta-0 Thalassemia, Inflammation, Quality of Life, Sickle Thalassemia, C-Reative Protein, Hemolytic Anemia, Hemostasis, Biomarkers, Coagulation, Omega-3 Fatty Acids, Eicosapentaenoic Acid, Docosahexaenoic Acid, Fish Oils, Drug: Placebo, Drug: LOVAZA | 1 Oct 2010 | 1 Mar 2012 |
| B Memory Cell Response to Vaccination With the 13-valent Pneumococcal Conjugate Vaccine in Asplenic Individuals | Aghia Sophia Children's Hospital of Athens | | 1 Oct 2010 | 1 Feb 2012 |
| Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation | Emory University | bone marrow transplant, sickle cell disease, thalassemia, Glanzmann thrombasthenia, Wiskott-Aldrich syndrome, chronic-granulomatous disease, severe congenital neutropenia, leukocyte adhesion deficiency, Schwachman-Diamond syndrome, Diamond-Blackfan anemia, Fanconi anemia, dyskeratosis-congenita, Chediak-Higashi syndrome, severe aplastic anemia | 1 Sep 2010 | 1 Sep 2015 |
| GDF 15 in Sickle Cell Disease and Hereditary Spherocytosis | Wolfson Medical Center | | 1 Sep 2010 | 1 Sep 2011 |
| Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload | Shire Development LLC | Beta-thalassemia, Sickle cell anemia, Transfusional iron overload, Iron overload, Iron chelation | 1 Aug 2010 | 1 Jan 2013 |
| Genes Influencing Iron Overload State | St. Jude Children's Research Hospital | Iron overload, Blood Transfusion | 1 Aug 2010 | 1 Jul 2016 |
| Safety and Efficacy of Deferasirox in β-thalassaemia Patients With Severe Cardiac Iron Overload | Novartis Pharmaceuticals | Severe cardiac iron overload, deferasirox, β-thalassaemia, cardiac dysfunction | 1 Jul 2010 | 1 Dec 2014 |
| Sickle Cell Trait and the Risk of Venous Thromboembolism | Albert Einstein College of Medicine of Yeshiva University | sickle cell trait, thrombosis, D-Dimer | 1 Jul 2010 | 1 Dec 2012 |
| Propanolol and Red Cell Adhesion Non-asthmatic Children Sickle Cell Disease | University of Miami | sickle cell disease, propanolol, red blood cell adhesion | 1 Jun 2010 | 1 Dec 2016 |
| A Phase III Safety and Efficacy Study of L-Glutamine to Treat Sickle Cell Disease or Sickle βo-thalassemia | Emmaus Medical, Inc. | Sickle Cell Anemia, Sickle Cell Pain Crises, Painful Crises, Sickle Cell Pain, Vaso-occlusive Crises | 1 May 2010 | 1 Feb 2013 |
| Perceptions of Thalassemia Major in Singapore: An Exploratory Study of Stigma | National Human Genome Research Institute (NHGRI) | Thalassemia Major, Stigma, Singapore, Qualitative | 1 Apr 2010 | |
| Beta-thalassemia and Microparticles | Assistance Publique Hopitaux De Marseille | TM, TI, MP | 1 Mar 2010 | 1 Mar 2013 |
| Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study (THALASSA) | Novartis Pharmaceuticals | Thalassemia,, thalassemia intermedia,, alpha-thalassemia,, beta-thalassemia,, deferasirox,, iron overload,, non-transfusion dependent | 1 Mar 2010 | 1 Jun 2012 |
| Multi-Center Study of Iron Overload: Survey Study (MCSIO) | Children's Hospital & Research Center Oakland | | 1 Mar 2010 | 1 Oct 2013 |
| Efficacy and Safety of Desferal Versus Osveral in Transfusional Iron Overload | Hormozgan University of Medical Sciences | Thalassemia, Iron overload, transfusion, osveral, desferal | 1 Feb 2010 | 1 May 2011 |
| Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia | Washington University School of Medicine | Thalassemia, Alemtuzumab, Hematopoietic cell transplant, non-myeloablative | 1 Jan 2010 | 1 Sep 2011 |
| This Study Will Evaluate Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndromes (MDS), Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload. | Novartis Pharmaceuticals | Iron Overload, hemotranfusion, deferasirox, ferritin, Rare anemia, Transfusional Iron Overload | 1 Dec 2009 | 1 Sep 2011 |
| Zoledronic Acid for the Prevention of Bone Loss Post-bone Marrow Transplantation for Thalassemia Major Patients | Tehran University of Medical Sciences | Thalassemia, BMT, HSCT | 1 Nov 2009 | 1 Dec 2011 |
| Multi-Center Study of Iron Overload: Pilot Study | Children's Hospital & Research Center Oakland | | 1 Nov 2009 | 1 Sep 2012 |
| Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload | FerroKin BioSciences, Inc. | Beta-thalassemia, Sickle cell anemia, Transfusional iron overload, Iron overload, Iron chelation | 1 Nov 2009 | 1 Dec 2010 |
| Demographic, Clinical and Laboratory Characteristics of Children With Alpha Thalassemia in Northern Israel | HaEmek Medical Center, Israel | | 1 Oct 2009 | 1 Dec 2010 |
| Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Severe Congenital Anemias Including Sickle Cell Disease and Beta-Thalassemia | National Heart, Lung, and Blood Institute (NHLBI) | Peripheral Blood Stem Cell Transplantation, Anemia, Sickle Cell, Graft-Versus-Host Disease, Sirolimus (Rapamune(Registered Trademark)), Alemtuzumab (Campath(Registered Trademark)), Thalassemia, Sickle Cell Disease | 1 Sep 2009 | 1 Aug 2015 |
| Assessment of Pain in People With Thalassemia Who Are Treated With Regular Blood Transfusions | National Heart, Lung, and Blood Institute (NHLBI) | Transfusion-Dependant Thalassemia, Pain, Mild Pain | 1 Jul 2009 | 1 Oct 2010 |
| Traditional Chinese Medicine in the Supportive Management of Anaemic and Cytopenic (Leukopenia, Thrombocytopenia) Haematological Disorders | Singapore General Hospital | Chronic cytopenic haematological diseases, Chinese herbal concoction, Supportive management | 1 Jul 2009 | 1 Aug 2011 |
| Treosulfan and Fludarabine Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders | Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium | Nonmalignant diseases, nonmalignant inherited disorders, primary immunodeficiency diseases, primary immune deficiency disorders, Chronic granulomatous disease, IPEX syndrome, hemophagocytic lymphohistiocytosis, Wiskott Aldrich Syndrome, bone marrow failure syndromes, Shwachman Diamond Syndrome, Dyskeratosis Congenita, Diamond Blackfan Anemia, inborn errors of metabolism, metabolic diseases, hemoglobinopathies, sickle cell disease, thalassemia, reduced intensity transplantation, hematopoietic cell transplantation, bone marrow transplantation, umbilical cord blood transplantation | 1 Jul 2009 | 1 Jul 2017 |
| Families Taking Control (FTC): Family-based Problem-solving Intervention for Children With Sickle Cell Disease | University of Pennsylvania | | 1 Jul 2009 | 1 Aug 2012 |
| Effect of Antioxidant Cocktail in Beta-thalassemia/Hb E Patients | Mahidol University | Thalassemia, Oxidative stress, antioxidant cocktail | 1 Jun 2009 | 1 Jun 2011 |
| Blood Transfusions in Thalassemia Patients, Complications and Adverse Effects | HaEmek Medical Center, Israel | Blood transfusions, Adverse events, Hepatitis C, HIV | 1 May 2009 | 1 Nov 2010 |
| Phase 1/2 Study of HQK-1001 in Patients With Beta Thalassemia | HemaQuest Pharmaceuticals Inc. | | 1 Mar 2009 | 1 May 2010 |
| Sildenafil to Improve Exercise Capacity in People With Thalassemia and Pulmonary Hypertension | National Heart, Lung, and Blood Institute (NHLBI) | Pulmonary Hypertension | 1 Mar 2009 | 1 Nov 2010 |
| Therapeutic Effects of Silymarin in Patients With B-thalassemia Major | Isfahan University of Medical Sciences | iron overload condition, regular desferrioxamine administration, receiving continuous blood transfusions | 1 Mar 2009 | 1 Dec 2009 |
| Assessment of Pain in People With Thalassemia | National Heart, Lung, and Blood Institute (NHLBI) | Transfusion-Dependent Thalassemia, Non-Transfusion-Dependent Thalassemia | 1 Mar 2009 | 1 Dec 2010 |
| Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension | Children's Hospital & Research Center Oakland | Pulmonary Hypertension, Sickle Cell Disease, Thalassemia | 1 Mar 2009 | 1 Mar 2013 |
| Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs | Stanford University | | 1 Mar 2009 | 1 Aug 2013 |
| The Spleen in Sickle Cell Anemia and Sickle Cell Thalassemia | HaEmek Medical Center, Israel | Splenectomy, Thrombocytosis, Infections, Spleen, Sickle Cell Thalassemia | 1 Feb 2009 | 1 Aug 2010 |
| Effect of Deferasirox on Patients With Cardiac MRI T2* < 20 Msec | Novartis Pharmaceuticals | Thalassemia, cardiac MRI T2* | 1 Feb 2009 | 1 May 2011 |
| Iron Overload and Growth Velocity in Thalassemia and Sickle Cell Anemia | HaEmek Medical Center, Israel | Thalassemia Major, Thalassemia Intermedia, Sickle Cell Anemia, Sickle Cell Thalassemia, Iron Overload, Growth Velocity | 1 Jan 2009 | 1 Jul 2010 |
| Interferon and Ribavirin Treatment in Patients With Hemoglobinopathies | Azienda Ospedaliera V. Cervello | antiviral treatment in hemoglobinopathies, management of chronic viral hepatitis C infection, antiviral treatment | 1 Jan 2009 | 1 Dec 2013 |
| European Sickle Cell Disease Cohort - Hydroxyurea | ADDMEDICA SASA | | 1 Jan 2009 | 1 Dec 2018 |
| Fresh Versus Old Red Blood Cells for Transfusion | Columbia University | red blood cells, transfusion, storage, sickle cell disease, β-thalassemia | 1 Dec 2008 | 1 Mar 2011 |
| HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity | North Shore Long Island Jewish Health System | Reduced Intensity Conditioning Regimen | 1 Dec 2008 | 1 Jan 2017 |
| Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload | Novartis Pharmaceuticals | Thalassemia, thalassemia intermedia, alpha-thalassemia, beta-thalassemia, deferasirox, iron overload, non-transfusion dependent | 1 Nov 2008 | 1 Jun 2011 |
| Amlodipine Use in the Prevention and Treatment of Iron Overload in Patients With Thalassemia Major | University of Campinas, Brazil | thalassemia major, calcium channel blockade, magnetic resonance imaging | 1 Sep 2008 | 1 Jul 2011 |
| Clinical and Laboratory Assessment of Iron Overload in Sickle Cell Anemia and Sickle Cell Thalassemia | HaEmek Medical Center, Israel | Sickle Cell Anemia, Sickle Cell Thalassemia, Iron overload, Non transferrin binding iron, Hepcidin | 1 Sep 2008 | 1 Dec 2010 |
| Evaluation of Non-invasive Endothelial Function in Children Sickle by Vascular Ultrasound | University Hospital, Toulouse | endothelial function, flow-mediated dilation, vascular elastic properties | 1 Sep 2008 | 1 Sep 2016 |
| Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells | Children's Hospital Los Angeles | unrelated, BMT, HSCT, bone marrow transplantation, sickle cell disease, thalassemia, CGD, HLH, Blackfan-Diamond anemia, Hurler, leukodystrophy, LAD I, Genetic diseases, Red blood cell defects, Sickle cell disease, Thalassemia, Leukocyte defects and immune deficiencies, Hereditary Lymphohistiocytosis, chronic granulomatous disease, Wiskott-Aldrich syndrome, Chediak Higashi syndrome, CD40 ligand deficiency, Hyper IgM syndrome, leucocytes adhesion defect type 1, Osteopetrosis, congenital neutropenia, X-linked lymphoproliferative disease, Platelets defects, Congenital amegakaryocytic thrombocytopenia, Metabolic and storage disorders, Hurler disease, leukodystrophies, Niemann-Pick disease, Fucosidosis, Stem cell defects, reticular agenesis | 1 Aug 2008 | 1 Aug 2012 |
| Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders | Duke University | Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, SCIDS, Wiskott Aldrich, FEL, HLH, IPEX, LAD, Sickle Cell, Thalassemia, Omenn's Syndrome, Hurler's Syndrome, MLD, ALD, Sanfilippo, Krabbe, Hunter's syndrome, TaySachs, Diamond Blackfan Anemia, transplant, MPS, Gaucher | 1 Aug 2008 | 1 Dec 2013 |
| Iron Balance Study of Deferasirox, Deferoxamine and the Combination of Both | Weill Medical College of Cornell University | Iron chelation, Iron balance, Secondary iron overload, deferoxamine, deferasirox | 1 Aug 2008 | 1 Dec 2009 |
| Oral Nifedipine to Treat Iron Overload | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Nifedipine, Thalassemia, Hemochromatosis, Iron Overload, Iron Excretion | 1 Jun 2008 | 1 Sep 2010 |
| Sickle Cell Anemia Screening and Prevention in Northern Israel | HaEmek Medical Center, Israel | Thalassemia, Sickle cell anemia, Carrier detection, Prenatal Diagnosis, Genetic counseling | 1 May 2008 | |
| Clinical Diagnosis of Teenagers Admitted to Pediatric Departments | HaEmek Medical Center, Israel | | 1 May 2008 | |
| Clinical and Laboratory Characteristics of Sickle Cell Anemia Patients Admitted With Fever | HaEmek Medical Center, Israel | Fever, Blood cultures | 1 May 2008 | 1 Nov 2008 |
| Diabetic Retinopathy and Sickle Trait | Medical University of South Carolina | Diabetic Retinopathy, Sickle Cell Trait, Vision Loss, Eye disease | 1 May 2008 | 1 Jul 2010 |
| Assess the Feasibility and Safety of Granulocyte Colony Stimulating Factor (GCSF) Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major | Memorial Sloan-Kettering Cancer Center | GCSF | 1 Apr 2008 | 1 Feb 2011 |
| Pilot Study for Patients With Poor Response to Deferasirox | Children's Hospital Boston | Thalassemia, Iron, Chelation | 1 Mar 2008 | 1 Nov 2008 |
| Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia | National Heart, Lung, and Blood Institute (NHLBI) | Thalassemia Intermedia | 1 Jan 2008 | 1 Sep 2010 |
| MRI Evaluation of Iron Overload in the Heart, Liver and Pancreas in Patients Receiving Multiple Blood Transfusions. | Sheba Medical Center | iron overload, Thalassemia, Anemia, Sickle Cell, Magnetic Resonance Imaging | 1 Jan 2008 | 1 May 2010 |
| Far Infrared Radiation for Sickle Cell Pain Management | GAAD Medical Research Institute Inc. | HEMOGLOBIN SC DISEASE, HEMOGLOBIN C DISEASE, SICKLE CELL TRAIT, THALASSEMIA | 1 Jan 2008 | 1 Jan 2009 |
| Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload | Novartis Pharmaceuticals | Myelodysplastic Syndromes, beta-Thalassemia, Iron Overload, Iron Chelating Agents, deferasirox | 1 Dec 2007 | 1 Jul 2011 |
| Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload | Novartis Pharmaceuticals | iron overload, cardiac iron, haemosiderosis, myocardial T2*, left ventricular ejection fraction, LVEF, cardiac dysfunction, thalassaemia, Diamond Blackfan anemia, DBA, sideroblastic anemia, myelodysplastic syndromes, MDS (low and INT-1 risk as per the IPSS for MDS), liver MRI, deferasirox, deferoxamine, ICL670, DFO, cardiovascular magnetic resonance imaging | 1 Nov 2007 | 1 Mar 2013 |
| Blood Sampling for Research Related to Sickle Cell Disease | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Sickle Hemoglobin, Erythrocytes, Drug Screen, Sickle Cell Trait, Sickle Cell Disease | 1 Oct 2007 | |
| Iron Overload Assesment in Sickle Cell Anemia and Sickle Cell Thalassemia | HaEmek Medical Center, Israel | Sickle Cell Anemia, Sickle Cell Thalassemia, Iron Overload, MRI | 1 Sep 2007 | 1 Dec 2010 |
| Phase I Study to Examine the Effect of Deferasirox on Renal Hemodynamics in β-thalassemia Patients With Transfusional Iron Overload | Novartis Pharmaceuticals | β-thalassemia, transfusional iron overload, renal function, renal biomarkers, deferasirox | 1 Sep 2007 | 1 Sep 2012 |
| Combo Chelation Trial | Children's Hospital & Research Center Oakland | Thalassemia, Chelation, Iron Overload, Thalassemia patients with Iron overload | 1 Sep 2007 | 1 Oct 2012 |
| Retroviral Vector Mediated Globin Gene Transfer to Correct Sickle Cell Anemia or Thalassemia | St. Jude Children's Research Hospital | | 1 Jul 2007 | 1 Jul 2016 |
| Clinical Trial of Deferasirox Combination Treatment With Deferiprone In Thalassaemia Patients | China Medical University Hospital | | 1 Jul 2007 | 1 Jul 2008 |
| Combination Therapy of Hydroxyurea With L-Carnitine and Magnesium Chloride in Thalassemia Intermedia | Shiraz University of Medical Sciences | Hydroxyurea, magnesium chloride, L-carnitine, β-thalassemia intermedia | 1 Jun 2007 | 1 Dec 2007 |
| Evaluating People With Thalassemia: The Thalassemia Longitudinal Cohort (TLC) Study | New England Research Institutes | | 1 May 2007 | 1 Jun 2011 |
| Endocrine and Nutritional Assessment in B Thalassemia Major | HaEmek Medical Center, Israel | Thalassemia, Nutrition, Endocrine, BMI | 1 May 2007 | 1 Dec 2009 |
| Pegasys® Plus Ribavirin in Thalassemic Patients With Hepatitis C Virus Infection | Baqiyatallah Medical Sciences University | Hepatitis C, Thalassemia, Pegasys, Ribavirin | 1 May 2007 | 1 Sep 2009 |
| Long Term Follow up in Sickle Cell Patients Treated by Hydroxyurea | HaEmek Medical Center, Israel | Sickle cell anemia, Sickle cell thalassemia, Hydroxyurea | 1 May 2007 | 1 Dec 2008 |
| A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders | Celgene Corporation | Myelodysplastic Syndrome (MDS), Acute myelogenous Leukemia (AML), Acute Lymphocytic Leukemia (ALL), Sickle Cell Disease (SCD), Beta Thalassemia, Inborn Errors of Metabolism, Severe Combined Immunodeficiency Disease (SCID) | 1 May 2007 | 1 Dec 2013 |
| Sickle Cell Anemia in an Arab Bedouin Village in the Northern Israel | HaEmek Medical Center, Israel | Sickle cell disease, Sickle cell thalassemia, Population screening | 1 May 2007 | 1 Dec 2008 |
| Detection of β Thalassemia Carriers by Red Cell Parameters Obtained From the H2 Automatic Counter | HaEmek Medical Center, Israel | Thalassemia, Iron Deficiency, Carrier screening | 1 Mar 2007 | 1 Jun 2007 |
| T2* in Transfusion Dependant Anemia, MI, LVF, Normal Patients | Imperial College London | | 1 Jan 2007 | 1 Aug 2007 |
| Hydroxyurea and Magnesium Pidolate to Treat People With Hemoglobin Sickle Cell Disease | St. Jude Children's Research Hospital | Sickle Cell Disease, Vaso-occlusive Event, Painful Crises, Acute Chest Syndrome | 1 Jan 2007 | 1 Aug 2009 |
| Autologous Bone Marrow Stem Cell Transplantation for Hip Osteonecrosis in Sickle Cell Disease | Federal University of Bahia | Cell therapy, Sickle cell disease, Hip Osteonecrosis, Autologous implantation, Mesenchymal stromal cell | 1 Aug 2006 | 1 Aug 2015 |
| Autologous Bone Marrow Stem Cells for Chronic Leg Ulcer Treatment in Sickle Cell Disease | Federal University of Bahia | Cell Therapy, Sickle cell disease, Autologous implantation, Mesenchymal stromal cell, Edothelial Progenitor cell | 1 Aug 2006 | 1 Aug 2016 |
| Evaluating the Safety of G-CSF Mobilization in Individuals With Beta Thalassemia Major | University of Washington | Beta Thalassemia Major, Hematopoietic Stem Cell Mobilization, Gene Transfer Techniques | 1 Jul 2006 | 1 Aug 2010 |
| Cardiopulmonary Function Assessment and NO-Based Therapies for Patients With Hemolysis-Associated Pulmonary Hypertension | National Heart, Lung, and Blood Institute (NHLBI) | Nitric Oxide, PDE Inhibitors, Pulmonary Hypertension, Sickle Cell, Thalassemia, Sickle Cell Anemia | 1 Jul 2006 | 1 Mar 2010 |
| Efficacy and Safety of Deferasirox in Patients With Transfusion-Dependent Iron Overload | Novartis Pharmaceuticals | Thalassemia, Myelodysplastic Syndrome, MDS, Sickle cell, Diamond-Blackfan anemia, Transfusion, Anemia, Fanconi, Chelation, Deferasirox | 1 Jul 2006 | 1 May 2009 |
| The Change of Coagulation Markers in Children With β-Thalassemia Disease After Stem Cell Transplantation | Mahidol University | Coagulation markers, thalassemia disease post SCT | 1 Jun 2006 | 1 Dec 2009 |
| Zinc & Bone Health in Thalassemia: The Think Zinc Study | Children's Hospital & Research Center Oakland | zinc, thalassemia, bone mineral density | 1 Apr 2006 | 1 Feb 2011 |
| Compassionate Use of Deferiprone for Patients With Thalassemia and Iron-Induced Heart Disease | Children's Hospital of Philadelphia | Iron overload, Thalassemia, Iron induced heart disease, deferoxamine (Desferal), Deferiprone | 1 Mar 2006 | |
| Amniotic Fluid Tandem Mass Spectrometry for Pregnancies Complicated by NIH and Severe Symmetrical IUGR | Obstetrix Medical Group | NIH, Severe Symmetrical IUGR, Tandem Mass Spectrometry, Nonimmune Hydrops, Severe Symmetrical Intrauterine Growth Restriction | 1 Mar 2006 | 1 Aug 2009 |
| Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment | Novartis | Iron Chelation, Deferasirox, Chelator, Desferal, beta-thalassemia, Iron overload | 1 Feb 2006 | 1 Nov 2009 |
| Invasive Infections in Children With Hemoglobinopathies | HaEmek Medical Center, Israel | Thalassemia, Sickle cell anemia, Splenectomy, Infections, Streptococcal Pneumonia | 1 Jan 2006 | 1 Jun 2006 |
| Collection of Blood From Persons With Hemoglobin and Erythrocyte Polymorphisms for Laboratory Malaria Research | National Institute of Allergy and Infectious Diseases (NIAID) | Sickle, Hemoglobinopathy, Thalassemia, G6PD, Plasmodium, Erythrocyte Variant, Hemoglobin Variant, Healthy Volunteer, HV | 1 Jan 2006 | 1 Jul 2010 |
| Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload | Novartis Pharmaceuticals | Deferasirox, Congenital Anemias, Anemias, Red Blood Cell Disorders, Chronic Iron Overload, Transfusional Iron Overload, Iron Chelators, Oral Iron Chelators, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis, ICL670A | 1 Oct 2005 | 1 Oct 2008 |
| Examination of Protective Factors Against Severe Malaria | National Institute of Allergy and Infectious Diseases (NIAID) | Sickle Trait, Fetal Hemoglobin, Hemoglobin C, Beta-Thalassemia, Artesunate-Amodiaquine | 1 Aug 2005 | 1 Apr 2009 |
| Factors Promoting Increased Rate and Success of Pregnancy in the Thalassemia Population in Toronto | University Health Network, Toronto | | 1 Jul 2005 | |
| Bone Marrow Transplantation, Hemoglobinopathies, SCALLOP | Baylor College of Medicine | Sickle Cell Disease, SCD, Hemoglobin SS, Hemoglobin SC, Hemoglobin Sb0/+, HLA genotype, Severe anemia, Transfusion therapy | 1 Jul 2005 | 1 Jul 2015 |
| Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration) | Novartis Pharmaceuticals | Transfusional hemosiderosis, Beta-thalassemia major, Deferasirox, iron overload, rare anemia, iron overload due to transfusion | 1 Jun 2005 | 1 May 2008 |
| Combination Therapy Compared With Single-Drug Therapy in Patients With Cardiac Diseases | National Heart, Lung, and Blood Institute (NHLBI) | | 1 Jun 2005 | 1 Apr 2009 |
| Heart Disease in Sickle Cell Anemia | National Institutes of Health Clinical Center (CC) | Cardiac Arrhythmias, Diastolic Dysfunction, Pulmonary Hypertension, Sickle Cell Anemia, Sudden Death, Thalassemia | 1 Jun 2005 | 1 Oct 2007 |
| A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload | Novartis Pharmaceuticals | Thalassemia, Myelodysplastic Syndrome, MDS, Sickle cell, Diamond-Blackfan anemia, Transfusion, Anemia, Fanconi, Chelation, Deferasirox | 1 Apr 2005 | 1 Jun 2010 |
| Study Using Deferiprone Alone or in Combination With Desferrioxamine in Iron Overloaded Transfusion-dependent Patients | Lipomed | Deferiprone, L1, Desferrioxamine, Hemochromatosis, Iron overload, Thalassemia | 1 Mar 2005 | 1 May 2011 |
| Aspirin Prophylaxis in Sickle Cell Disease | University of Rochester | sickle cell disease, hemoglobin SS disease, hemoglobin S Beta-0 Thalassemia, silent infarction in sickle cell disease, overt stroke in sickle cell disease, aspirin, transcranial Doppler ultrasound, neurocognitive testing | 1 Mar 2005 | 1 Nov 2009 |
| Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia | National Institute of Allergy and Infectious Diseases (NIAID) | | 1 Jan 2005 | 1 Dec 2005 |
| An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670) in β-thalassemia Patients With Transfusional Iron Overload | Novartis Pharmaceuticals | β-thalassemia, iron overload, deferasirox | 1 Oct 2004 | 1 Apr 2008 |
| Genetic Factors Affecting the Severity of Beta Thalassemia | Hadassah Medical Organization | | 1 Jul 2004 | 1 Apr 2012 |
| A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis | Novartis | Thalassemia (beta-thal. major), Transfusional hemosiderosis, Deferasirox, Beta-thalassemia major patients, Unable to be chelated with deferoxamine or deferiprone | 1 May 2004 | 1 Nov 2006 |
| Combined Chelation Treatment With Deferiprone and Deferoxamine in Thalassemia Major | Royal Brompton & Harefield NHS Foundation Trust | Randomized Controlled Trial, Deferiprone, Deferoxamine, Iron chelation, Beta Thalassemia Major | 1 May 2004 | 1 Jun 2005 |
| Monitoring of Erythroid Lineage Specific Chimerism Following Allogeneic Hematopoietic Transplantation for Thalassemia Major | Dana-Farber Cancer Institute | chimerism, allogenic hematopoietic transplantation, assay | 1 Apr 2004 | 1 Oct 2005 |
| Genetics of Alpha Thalassemia in Israeli Ethnic Groups | Hadassah Medical Organization | | 1 Apr 2004 | 1 Apr 2012 |
| Natural History of Sickle Cell Disease and Other Hemolytic Disorders | National Heart, Lung, and Blood Institute (NHLBI) | Acute Chest Syndrome, Hemoglobin, Nitric Oxide, Pulmonary Hypertension, Pain Crisis, Treatment Options, Sickle Cell Disease, Sickle Cell Trait, Red Blood Cell Disorder, Thalassemia, Paroxysmal Nocturnal Hemoglobinuria, Hemolytic Disorders | 1 Apr 2004 | |
| L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia | Emmaus Medical, Inc. | sickle cell disease, sickle cell anemia, L-glutamine, Sickle Cell Anemia (homozygous), Sickle ß0-Thalassemia | 1 Mar 2004 | 1 Jul 2008 |
| Evaluation of Efficacy of Zoledronic Acid in Patients With Haemoglobin Syndromes (Thalassemia and Sicle Cell Anaemia) and Risk of Skeletal Events | Novartis Pharmaceuticals | thalassemia, zoledronic acid, skeletal events | 1 Mar 2004 | 1 Mar 2006 |
| Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies | Dana-Farber Cancer Institute | Hemoglobinopathies, Sickle cell anemia, sickle cell-hemoglobin C disease, sickle cell-B-thalassemia, transfusion-dependant thalassemia, allogeneic transplant, nonmyeloablative transplant, Stem cell transfusion, graft vs. host disease | 1 Mar 2004 | 1 Jul 2009 |
| Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients | Novartis Pharmaceuticals | β-thalassemia, rare chronic anemia, iron overload, deferasirox, chronic anemias, transfusional hemosiderosis | 1 Mar 2004 | 1 Oct 2008 |
| Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO | Baylor College of Medicine | transfusion-dependent, homozygous b0/+-thalassemia, severe variants of b0/+-thalassemia, chronic transfusion therapy, iron chelating agents, severe, transfusion-dependent homozygous b0/+-thalassemia or severe variants of b0/+-thalassemia | 1 Feb 2004 | 1 Sep 2015 |
| Efficacy and Safety of Neridronate (Nerixia®)to Treat Osteoporosis in Patients With TM and TI | Ente Ospedaliero Ospedali Galliera | Thalassemia, Neridronate, Osteopenia, Osteoporosis | 1 Jan 2004 | 1 May 2010 |
| Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia | Genzyme | Beta Thalassemia | 1 Sep 2003 | |
| A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload. | Novartis Pharmaceuticals | β-thalassemia major, iron overload, deferasirox, pediatric rare anemia | 1 Sep 2003 | 1 Feb 2008 |
| A Rehabilitation Program in Children With Sickle Cell Disease and Cognitive Deficits: a Pilot Study | National Heart, Lung, and Blood Institute (NHLBI) | Sickle Cell Disease, Sickle Cell, Memory, Learning, Achievement | 1 Sep 2003 | 1 Dec 2012 |
| A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity | Novartis Pharmaceuticals | Deferasirox, ICL670A, Iron chelators, Deferiprone, Transfusional hemosiderosis, Congenital aplastic anemia (Diamond Blackfan anemia), Red cell aplasia, Thalassemia, β thalassemia | 1 Jun 2003 | 1 Dec 2007 |
| Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions | Novartis Pharmaceuticals | Thalassemia, iron overload, deferoxamine, hemosiderosis | 1 May 2003 | 1 Nov 2004 |
| Evaluating the Safety of Two Medications to Treat Hepatitis C in People With Thalassemia (The HepC Study) | National Heart, Lung, and Blood Institute (NHLBI) | | 1 May 2003 | 1 Aug 2006 |
| Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias | Novartis Pharmaceuticals | beta-thalassemia, iron overload, deferoxamine, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan, Anemia, Aplastic | 1 May 2003 | 1 Nov 2004 |
| Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias | National Heart, Lung, and Blood Institute (NHLBI) | Stem Cell Transplant, Low Dose Radiation, Alemtuzumab (Campath), Sirolimus (Rapamune), Donor Apheresis, Graft-Versus-Host Disease, Graft-Versus-Marrow, Host-Donor Chimerism, Peripheral Blood Stem Cells, Low Dose Irradiation, Sickle Cell Anemia, SCA, Thalassemia, Diamond-Blackfan Anemia, DBA | 1 May 2003 | 1 Feb 2018 |
| Safety and Efficacy of Deferasirox in Patients With Transfusion Dependent Iron Overload - a Non-comparative Extension Study | Novartis Pharmaceuticals | iron overload, iron chelation therapy, B-thalassemia | 1 Feb 2003 | 1 Jan 2008 |
| L-Glutamine Therapy for Sickle Cell Anemia | Los Angeles Biomedical Research Institute | sickle cell disease, sickle cell anemia, L-glutamine, Sickle Cell Anemia (homozygous and heterozygous), Sickle ß0-Thalassemia | 1 Jan 2003 | 1 Nov 2009 |
| Efficacy Study in Removing Excess Iron From the Heart | ApoPharma | Iron Overload, Thalassemia, Haemosiderosis, Cardiac, Deferiprone, Chelation | 1 Dec 2002 | 1 Oct 2004 |
| A Pilot Study of HSCT for Patients With High-Risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regimen | University of Pittsburgh | Sickle Cell Disease, Thalassemia, Hemoglobinopathies | 1 Jun 2002 | 1 Dec 2012 |
| Stem Cell Transplant for Hemoglobinopathy | Masonic Cancer Center, University of Minnesota | high risk hemoglobinopathy, stem cell transplant, donor lymphocyte infusion, transfusion dependent, stem cell donor, cord blood, marrow, transfusion dependent non-malignant hematologic disorders | 1 Jun 2002 | 1 Jun 2016 |
| Study With Deferiprone and/or Desferrioxamine in Iron Overloaded Patients | Lipomed | Deferiprone, L1, Desferrioxamine, Hemochromatosis, Iron overload, Thalassemia | 1 Jan 2002 | |
| Stem Cell Transplant in Sickle Cell Disease and Thalassemia | Columbia University | stem cell transplant, sickle cell disease, thalassemia, moderately ablative, cord blood transplant, matched family donor | 1 Dec 2001 | 1 Jan 2014 |
| Intensive Combined Chelation Therapy for Iron-Induced Cardiac Disease in Patients With Thalassemia Major | Ospedale Microcitemico | | 1 Dec 2001 | 1 Jun 2006 |
| Oral Magnesium Pidolate, Hemoglobin SC Disease, MG Pidolate | Baylor College of Medicine | Hemoglobin SC disease, sickle cell-hemoglobin | 1 Jan 2001 | 1 May 2006 |
| Phase II Study of Azacitidine and Phenylbutyrate in Patients With Thalassemia Major | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | genetic diseases and dysmorphic syndromes, hematologic disorders, rare disease, thalassemia major | 1 Nov 2000 | |
| Efficacy Study of the Use of Sequential DFP-DFO Versus DFP | Azienda Ospedaliera V. Cervello | thalassemia major, chelation treatment, secondary hemochromatosis | 1 Sep 2000 | 1 Jan 2008 |
| Arginine Therapy in Sickle Cell Disease-VOC Clinical Trial | Children's Hospital & Research Center Oakland | sickle cell disease, vaso-occlusive pain episodes, arginine, nitric oxide | 1 Sep 2000 | 1 Jun 2007 |
| Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies | Baylor College of Medicine | | 1 Aug 2000 | |
| Bone Marrow Transplant From Donor Using Less Toxic Conditioning for Patient With High Risk Hemoglobinopathies | Baylor College of Medicine | | 1 Aug 2000 | |
| Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN) | National Heart, Lung, and Blood Institute (NHLBI) | chelator, iron, transfusion, anemia, thalassemia | 1 Jul 2000 | 1 Jul 2006 |
| 5-Azacytidine and Phenylbutyrate to Treat Severe Thalassemia | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Fetal Hemoglobin, 5-Azacytidine, Phenylbutyrate, Thalassemia, Hemoglobin, Anemia, Blood, Thalassemia Major, Beta Thalassemia | 1 Jun 2000 | 1 Jun 2003 |
| Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders | Fairview University Medical Center | chronic congenital neutropenia, chronic neutropenia, congenital pure red cell aplasia, disease-related problem/condition, genetic diseases and dysmorphic syndromes, hematologic disorders, neutropenia, pure red cell aplasia, rare disease, sickle cell anemia, thalassemia major | 1 Jun 2000 | |
| A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients With Sickling Disorders | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Fetal Hemoglobin, Hematopoietic Growth Factor, Peripheral Blood CD34 Cells, Vasoocclusive Crisis, Sickle Cell Anemia, Sickle Cell Disease, Sickle Cell Disorder | 1 Mar 2000 | 1 Oct 2000 |
| Mobilization and Handling of Stem Cells for Transplant From Healthy Volunteers With Sickle Cell Trait | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Donor Apheresis, Sickle Cell Trait, Red Cell Alloimmunization | 1 Jan 2000 | 1 Jul 2002 |
| Hydroxyurea to Treat Beta-Thalassemia (Cooley's Anemia) | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Gamma Gene, Hemoglobin Switching, Erythropoiesis, HbE Hemoglobin Chain Synthesis Imbalance, Cooley's Anemia, Beta-Thalassemia Intermedia | 1 Dec 1999 | 1 Feb 2002 |
| Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia | Boston University | genetic diseases and dysmorphic syndromes, hematologic disorders, rare disease, thalassemia intermedia | 1 Mar 1999 | |
| Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia | National Heart, Lung, and Blood Institute (NHLBI) | | 1 Jan 1999 | 1 Aug 2006 |
| Combination Iron Chelation Therapy | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | chelation therapy, combination chemotherapy, iron poisoning, thalassemia, chelating agent, deferoxamine, drug screening /evaluation, therapy evaluation | 1 Dec 1998 | 1 Nov 2002 |
| Diagnostic Pilot Study of Dual Energy Absorptiometry in the Detection of Osteopenia or Osteoporosis in Patients With Thalassemia Major | National Center for Research Resources (NCRR) | disease-related problem/condition, genetic diseases and dysmorphic syndromes, hematologic disorders, osteoporosis, rare disease, thalassemia major | 1 Jun 1998 | 1 Aug 2000 |
| Phase II Randomized Trial of Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers | FDA Office of Orphan Products Development | dermatologic disorders, genetic diseases and dysmorphic syndromes, hematologic disorders, rare disease, sickle cell anemia, skin ulcers, thalassemia major | 1 Sep 1997 | |
| Stem Cell Transplantation (SCT) for Genetic Diseases | National Center for Research Resources (NCRR) | Fanconi's anemia, amegakaryocytic thrombocytopenia, aplastic anemia, congenital pure red cell aplasia, genetic diseases and dysmorphic syndromes, hematologic disorders, inborn errors of metabolism, metachromatic leukodystrophy, pure red cell aplasia, rare disease, sphingolipidoses, thalassemia major | 1 Jan 1995 | |
| Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis | Memorial Sloan-Kettering Cancer Center | ERYTHROPOIESIS, Genetic Disorders, Sickle Cell Anemia, Thalassemia, Diamond Blackfan Anemia | 1 Jan 1994 | 1 Aug 2008 |
| Chelation Therapy of Iron Overload With Pyridoxal Isonicotinoyl Hydrazone | National Heart, Lung, and Blood Institute (NHLBI) | | 1 Jun 1989 | |
| Deferoxamine for the Treatment of Hemochromatosis | National Heart, Lung, and Blood Institute (NHLBI) | Desferal, Thalassemia, Liver Iron Concentration, Endocrine Evaluation, Diabetes Mellitus, Cardiac Disease, Acquired Anemia | 1 Apr 1985 | |
| Evaluation of Subcutaneous Desferrioxamine as Treatment for Transfusional Hemochromatosis | National Heart, Lung, and Blood Institute (NHLBI) | | 1 Jan 1978 | |
| Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants | Joanne Kurtzberg | Haploidentical Donor, T-cell depleted Stem Cells, Allogeneic Transplant, Umbilical Cord Blood Donor, High Risk Malignancies, Metabolic Disorders, Immune Deficiency, Acute Lympoblastic Leukemia, Acute Myelogenous Leukemia, Myelodysplastic Syndrome, ALL, AML, MDS, CGD, SCID, Adrenoleukodystrophy, Metachromaticleukodystrophy, Krabbe, PMD, Hunter's, Hurler's, Severe Aplastic Anemia, Lymphoma, Sickle Cell Disease, Thalassemia | | |
| A Dose Escalation Study of Intravenous L-citrulline in Steady-state Sickle Cell Disease | Suvankar Majumdar | | | |
