Phase 3 Niemann-Pick Type C 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

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)

Niemann-Pick Type C Disease

Azafaros A.G.72 enrolled34 locationsNCT07082725

Recruiting

Phase 3

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

GM1 GangliosidosisGM2 GangliosidosisNiemann-Pick Type C Disease

Azafaros A.G.147 enrolled37 locationsNCT07054515