Clinical trials for GM2 gangliosidosis
4 recruiting or active trials synced from ClinicalTrials.gov.
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Clinical trial records are synced from ClinicalTrials.gov through automated extraction.
Report missing data- RECRUITINGPhase 2NCT07399704A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C DiseaseAzafaros A.G.
- ACTIVE NOT RECRUITINGNCT06614569Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff DiseaseTerence Flotte · PI: Terence Flotte, MD
- ACTIVE NOT RECRUITINGPhase 2NCT05758922Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients with GM2 Gangliosidosis or Niemann-Pick Type C DiseaseAzafaros A.G.
- RECRUITINGNCT05007990Caregiving Networks Across Disease Context and the Life CourseNational Human Genome Research Institute (NHGRI) · PI: Laura M Koehly, Ph.D.