Sandhoff Disease Clinical Trials

17 clinical trials for Sandhoff Disease across international registries, including 3 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

A Natural History Study of the Gangliosidoses — RECRUITING
Longitudinal Study of Neurodegenerative Disorders — RECRUITING
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells — RECRUITING · PHASE1
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol) — NOT_YET_RECRUITING · PHASE2
A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2 — TERMINATED · PHASE3
Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease — ACTIVE_NOT_RECRUITING
GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO) — COMPLETED
A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease — TERMINATED · PHASE1
N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) — COMPLETED · PHASE2
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders — TERMINATED · PHASE1
Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2 — COMPLETED
Synergistic Enteral Regimen for Treatment of the Gangliosidoses — TERMINATED · PHASE4
Registry Gangliosidoses — OTHER
Stem Cell Transplant for Inborn Errors of Metabolism — COMPLETED · PHASE2
Gene Therapy for Tay-Sachs Disease — COMPLETED
Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease) — COMPLETED · PHASE1
Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses — COMPLETED · PHASE3

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