bluebird bio

This is a non randomized, open label, multi site, single dose, Phase 1/2 study in up to 15 adults with β thalassemia major who receive at least 100 mL/kg/year of packed red blood cells (pRBCs) or ≥8 transfusions of pRBCs per year. The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin® BB305 Drug Product (autologous CD34+ hematopoietic stem cells transduced with LentiGlobin® BB305 lentiviral vector encoding the human βA T87Q globin gene and stored in cryopreservative solution in the final immediate container for the intended medical use).

 

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More information: clinicaltrials.gov

mast therapeutics

The purpose of this study is to evaluate whether ANX-188 can reduce the duration of vaso-occlusive crisis (VOC) in subjects with sickle cell disease. The study will also evaluate whether ANX-188 can reduce the frequency of rehospitalization of subjects due to a recurrence of VOC. Additionally, this study will compare the development of acute chest syndrome during VOC in subjects who receive ANX-188 to those who do not receive ANX-188.

 

More information: clinicaltrials.gov

cvbf eu logo

Deferiprone (DFP) is the most extensively studied oral iron chelator to date. It has been authorised in Europe in 1999 for the treatment of iron overload in patients with beta-thalassaemia major when DFO is contraindicated or inadequate. Despite a wide experience of DFP there are limited experimental data available on DFP in children and no pharmacokinetic data in children under 6 years of age. On the basis of the existing data in adults and adolescent, in the DEEP-1 trial a pharmacokinetic bridging model will be developed to support the dose selection in children aged less than 6 years. The study will consist of two phases, namely an experimental phase, during which patients will receive a single dose and a modeling phase, during which PK data obtained after single dose in patients < 6 years of age will be analysed in conjunction with historical PK data in adults and older children and adolescents. The model-based analysis of the data obtained after single dose will enable the assessment of the dosing regimen required for the purpose of accurate pharmacokinetic bridging. The ratio between the predicted systemic exposure parameters (AUC and Cmax) in the target population and reference group will be used as basis for recommendation of the dose in the target population.

 

More information: clinicaltrials.gov

novartis

In this study, non-invasive R2- and T2*-MRI techniques will be applied to the liver and the heart, respectively, to complement the primary variable (serum ferritin) assessed in patients with various transfusion-dependent anaemias. The main objective of this study is to assess the prevalence and severity of cardiac and liver siderosis in patients with transfusional siderosis. This study will also aim to establish possible correlations between cardiac and liver iron levels with clinical effects in patients with different transfusion-dependent anaemias. Patients will be eligible for enrollment irrespective of receiving chelation therapy or not (and irrespective of the chelating agent used).

 

More information: clinicaltrials.gov