Phase 2 Gangliosidosis Clinical Trials
2 recruitingPhase 2
What is a Phase 2 trial?
Phase 2 trials evaluate whether a treatment works for a specific condition and further assess its safety. These studies typically enroll several hundred participants and provide preliminary data on effectiveness.
Showing 1–2 of 2 trials
Recruiting
Phase 2
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
GM2 GangliosidosisNiemann-Pick Type C Disease
Azafaros A.G.21 enrolled3 locationsNCT07399704
Recruiting
Phase 1Phase 2
A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis
Lysosomal DiseasesGangliosidosisGM1
National Human Genome Research Institute (NHGRI)54 enrolled1 locationNCT03952637