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The objective of this study is to assess the effect of alkali administration on bicarbonate and potassium levels in patients with Sickle Cell Disease (SCD) and depressed serum bicarbonate levels. The study is a prospective non-blinded evaluation of tolerability and efficacy of alkali repletion with 4 weeks of observation and two sequential 4 week courses of escalating oral sodium bicarbonate treatment.

More information: clinicaltrials.gov, ITHANET Clinical Trials

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Patients who have sickle cell VOC are usually treated with opioids, such as morphine. However, this current way of treating them has not improved the health, medical outcomes, or rates of hospitalizations. In addition, since VOC can happen very frequently over a long period of time, giving opioids over and over again can cause both short-term and long-term problems. Nitrous oxide (N2O) is a way of treating pain that may provide a better alternative to repeatedly giving opioids over long periods of time. N2O has been shown to provide up to 3 hours of pain relief in inpatient patients with VOC whose pain did not improve with morphine infusions, and is used extensively in France, where almost half of 85 pediatric emergency departments use nitrous oxide to treat children with VOC whose pain did not get better with standard treatment with morphine. However, pain relief which N2O provides in the acute setting has not been well described. Therefore, the purpose of our study is to describe how well N2O can relieve the pain in patients with SCD who present to the emergency department and are experiencing a VOC.

More information: clinicaltrials.gov, ITHANET Clinical Trials

sangamo

Sangamo BioSciences, Inc. announced that the California Institute for Regenerative Medicine (CIRM) has granted the Company a $6.4 million Strategic Partnership Award to develop a potentially curative ZFP Therapeutic for beta-thalassemia based on the application of its zinc finger nuclease (ZFN) gene-editing technology in hematopoietic stem cells (HSCs).

The four-year grant provides matching funds for preclinical work that will support an Investigational New Drug application and a phase 1 clinical trial for a ZFP Therapeutic. The goal of the Therapeutic is to utilize genome-editing technology to raise levels of fetal hemoglobin in red blood cells to a sufficient degree to have a positive clinical effect.

More information: Sangamo Biosciences press release, CIRM funding

uh-case-medical-center

The investigators propose to evaluate exhaled carbon monoxide (CO) in patients with sickle hemoglobin types: hemoglobin SS (HbSS), hemoglobin SC (HbSC), and hemoglobin S (HbS)-beta thalassemia during routine clinic visits, and longitudinally. The investigators goal is to know whether exhaled carbon monoxide (etCO) differs amongst subjects with different sickle cell syndrome genotypes, and whether it is a stable marker of hemolytic rate, as reflected in routine labs obtained for clinical care (including total hemoglobin, reticulocyte count, lactate dehydrogenase, and, when sampled, total and direct bilirubin). The investigators hope to establish whether this inexpensive and non-invasive test faithfully reflects hemolytic parameters in sickle cell syndromes.

More information: clinicaltrials.gov, ITHANET Clinical Trials

vanderbilt-ingram cancer

Allogeneic blood or marrow transplantation (alloBMT) is a curative therapy for a variety of hematologic disorders, including sickle cell disease and thalassemia. Even when it is clear that alloBMT can give to these patients an improvement in their disease, myeloablative transplants have important toxicities and mortalities associated. The lack of suitable donors continues to be a limit to access to transplantation. Substantial progress has been made recently in the development of pre-treatment regimens that facilitate the sustained engraftment of donor marrow with reduced toxicity. Most of these regimens incorporate highly immunosuppressive drugs, which allow the reduction or elimination of myeloablative agents or total body irradiation without endangering the sustained engraftment of HLA-identical allogeneic stem cells. Preliminary results of non-myeloablative allogeneic stem cell transplantation suggest that the procedure can be performed in patients who are ineligible for myeloablative alloBMT, and that sustained remissions of several hematologic malignancies can be obtained.

 

More information: clinicaltrials.gov, ITHANET Clinical Trials