GM2 Gangliosidosis Clinical Trials

12 clinical trials for GM2 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
• A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease — RECRUITING · PHASE2
• A Natural History Study of the Gangliosidoses — RECRUITING
• The Myelin Disorders Biorepository Project — RECRUITING
• Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease — COMPLETED · PHASE2
• A Natural History of Late Onset Tay-Sachs Disease — COMPLETED
• LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies — COMPLETED
• Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease — ACTIVE_NOT_RECRUITING
• Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) — TERMINATED · PHASE3
• N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) — COMPLETED · PHASE2
• Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2 — COMPLETED
• Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A multinational, multi-center, open-label, rater-blinded Phase II study — OTHER

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