5 results
Primary objective:the primary objective of the study is to evaluate the safety and to define the maximal tolerated dose or the maximal administered dose of CC-486 as a single agent, in combination with CBDCA or ABI-007 in subjects with relapsed or…
Primary Objective:To evaluate RBC transfusion independence in the 2 treatment arms (oral azacitidine plus best supportive care versus placebo plus best supportive care) in subjects with RBC transfusion-dependent anemia and thrombocytopenia (platelet…
This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of crovalimab compared with placebo as adjunct treatment for the prevention of vaso-occlusive episodes (VOEs) in patients with sickle cell disease (SCD).
The objective of the study is to assess the long-term safety and efficacy of intravenous ATB200 co-administration with oral AT2221 in adult subjects with late-onset Pompe disease.
This study will evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of crovalimab compared with placebo for the management of acute uncomplicated vaso-occlusive episodes (VOE) in patients with sickle cell disease (SCD).