Phase 3 Sickle Cell Disease Trials
5 Phase 3 trials for Sickle Cell Disease. Phase 3 trials are the final stage of testing before a treatment can be submitted for FDA approval.
What is a Phase 3 trial?
Phase 3 clinical trials study a treatment in large groups (hundreds to thousands of people) to confirm its effectiveness, monitor side effects, and compare it to standard treatments. These trials are the last major step before the FDA considers approving a new treatment.
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood...
RElated Haplo-DonoR Haematopoietic stEm Cell Transplantation for Adults With Severe Sickle Cell Disease
The purpose of this clinical trial is to evaluate the clinical and cost effectiveness of Haploidentical Stem Cell Transplantation (SCT) for adults with severe sickle cell disease...
Oral Ketamine for Treatment of Vaso-Occlusive Pain
The purpose of this study is to learn more about the feasibility of oral ketamine for the treatment of painful sickle-cell crises in children and adolescents as a supplement to...
The Efficacy and Safety of Rilzabrutinib in Patients Aged 10 to 65 Years With Sickle-cell Disease
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate...
Promoting Utilization and Safety of Hydroxyurea Using Precision in Africa
Sickle cell anemia (SCA) is among the world's most common and devastating blood disorders, affecting more than 300,000 newborns per year. Most infants with SCA are born in the...