Phase 1 Diamond-Blackfan Anemia Clinical Trials

2 recruitingPhase 1

What is a Phase 1 trial?

Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.

Showing 1–2 of 2 trials

Recruiting

Phase 1

Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia

RPS19 Deficient Diamond-Blackfan Anemia

Apriligen, Inc.4 enrolled2 locationsNCT07476183

Recruiting

Phase 1Phase 2

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

Diamond-Blackfan AnemiaSickle Cell Anemiabeta thalassemia major

Paul Szabolcs5 enrolled1 locationNCT03653338