Phase 3 Genetic Disease Clinical Trials
2 recruitingPhase 3
What is a Phase 3 trial?
Phase 3 trials compare the new treatment against the current standard of care in large groups, often hundreds to thousands of participants. Successful Phase 3 results are typically required for regulatory approval.
Showing 1–2 of 2 trials
Recruiting
Phase 3
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Beta-ThalassemiaSickle Cell DiseaseHematologic Diseases+4 more
Vertex Pharmaceuticals Incorporated26 enrolled6 locationsNCT05477563
Recruiting
Phase 3
A Study to Learn About the Safety and Efficacy of the Drug Oleogel-S10 in Japanese Patients With Epidermolysis Bullosa
Skin DiseasesEpidermolysis BullosaConnective Tissue Disease+7 more
Chiesi Farmaceutici S.p.A.6 enrolled6 locationsNCT06917690