Phase 3 Genetic Disease Clinical Trials
4 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 1–4 of 4 trials
Recruiting
Phase 3
Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
Muscular Dystrophy, DuchenneDMDNeuromuscular Diseases (NMD)+10 more
Dyne Therapeutics90 enrolled1 locationNCT07608432
Recruiting
Phase 3
A Phase 3 Study of NTLA-2001 in ATTRv-PN
Nervous system diseasePeripheral nervous system diseaseAmyloidosis+17 more
Intellia Therapeutics60 enrolled14 locationsNCT06672237
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