Clinical Trials

Avascular necrosis (AVN) is a serious complication of sickle cell disease, especially in pediatric patients where the prevalence is between 3% and 8% and are more frequent in patients with multiple vaso-occlusive crisis (VOC). The prevalence of AVN is usually made by a study of the hip through radiography, whereas other possible sites of ischemic infarcts are evaluated only in case of specific symptoms. In addition, bone infarcts may be the trigger for additional VOC.

The objective of this study is to evaluate the pharmacokinetics of deferiprone and its 3-O-glucuronide metabolite following administration of a single 1500 mg dose of Ferriprox in patients with sickle cell disease.

The pathophysiology of sickle cell disease (SCD) manifestations, are complex with interactions of intracellular hemoglobin, membrane and endothelial activation but the hallmark remains recurrent and painful vaso-occlusive episodes (VOC). These painful episodes are thought to result from ischemia caused when small blood vessels are occluded by misshapen, inflexible erythrocytes. Painful episodes are the most common cause of hospitalization, morbidity, and impairment for SCD patients. There is no therapy that completely prevents or directly aborts painful events for all patients.

The purpose of this study is to assess the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of Aes-103 (active ingredient 5-hydroxymethyl-2-furfural \[5-HMF\]) compared with placebo in subjects with stable sickle cell disease (SCD). Safety will be measured by monitoring adverse events (AEs), electrocardiograms (ECGs), vital signs, and laboratory values. Pharmacokinetics of Aes-103 will be measured over time in plasma, red blood cell hemolysate and binding of Aes-103 to hemoglobin.

Sickle cell disease (SCD) is the most common genetic disease in France. Its consequences on patient's life-course and quality of life need to be precisely identified among French patients and their family to be able to improve patients care according to their specific needs.

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.

This Phase 1/Phase 2 study will evaluate GMI-1070, a pan-selectin inhibitor, in adults with stable sickle cell disease. The study will assess safety, pharmacokinetics, and microvascular effects of intravenous GMI-1070 in the outpatient setting.

The purpose of the study is to assess the safety and tolerability of AG-946 in healthy volunteers after oral administration of single ascending doses (SAD) and multiple ascending doses (MAD) of AG-946 over 14 or up to 28 days of dosing, and to identify a range of doses that are safe and pharmacologically active in participants with sickle cell disease. The SAD and MAD parts of the study will be randomized and double-blinded, and will assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of AG-946 as well as the effect of food (SAD only) on the pharmacokinetics (PK) of AG-946.

In this study, patient blood samples from NMA transplants will be provided by Pittsburgh, and samples from myeloablative transplants will be provided by Atlanta (comparative controls). Samples would be obtained pre- and post-BMT from the recipient at a total of 7 timepoints, and from the donor at one timepoint.

Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients with Hemoglobinopathies