Sandhoff Disease
16
3
4
7
Key Insights
Highlights
Success Rate
64% trial completion
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 52/100
25.0%
4 terminated out of 16 trials
63.6%
-22.9% vs benchmark
19%
3 trials in Phase 3/4
71%
5 of 7 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 7 completed trials
Clinical Trials (16)
A Natural History Study of the Gangliosidoses
Longitudinal Study of Neurodegenerative Disorders
A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2
Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease
GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease
N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
Synergistic Enteral Regimen for Treatment of the Gangliosidoses
Registry Gangliosidoses
Stem Cell Transplant for Inborn Errors of Metabolism
Gene Therapy for Tay-Sachs Disease
Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)
Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses