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Hereditary Red Blood Cell Disorder (Disorder)
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Hereditary Red Blood Cell Disorder (Disorder) trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Hereditary Red Blood Cell Disorder (Disorder) trials you may qualify forThe aim of this study to evaluate the safety and efficacy of a nonmyeloablative conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSC…
A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or w…
Gene therapy is a new possible treatment for the anemia of DBAS. Gene therapy will soon be available for patients with RPS19-mutated DBAS. This involves inserti…
The purpose of this study is to maintain a comprehensive registry of patients with the rare inherited bone marrow failure syndrome Diamond Blackfan anemia (DBA)…
This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignan…
The purpose of this research study is to look at genes and determine how they interact with each other to find changes that could explain why some people's immu…
Background: Sickle cell disease (SCD) is a disorder that causes episodes of acute pain and progressive organ damage. Ways to manage SCD have evolved slowly. Tr…
The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) versus placebo in adolescent and adult sic…