GM1 Gangliosidosis Clinical Trials

13 clinical trials for GM1 Gangliosidosis across international registries, including 4 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• 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 — RECRUITING · PHASE3
• ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program — RECRUITING
• A Natural History Study of the Gangliosidoses — RECRUITING
• FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases — RECRUITING
• Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis — ACTIVE_NOT_RECRUITING · PHASE1
• Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients — COMPLETED
• GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO) — COMPLETED
• A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis — TERMINATED · PHASE1
• Interviews and Video Capture in Patients With GM1 Gangliosidosis — COMPLETED
• Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2 — COMPLETED
• Registry Gangliosidoses — OTHER
• HSCT for High Risk Inherited Inborn Errors — COMPLETED · PHASE2
• Stem Cell Transplant for Inborn Errors of Metabolism — COMPLETED · PHASE2

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