Tay-Sachs Disease
12
3
3
5
Key Insights
Highlights
Success Rate
63% trial completion
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 52/100
25.0%
3 terminated out of 12 trials
62.5%
-24.0% vs benchmark
17%
2 trials in Phase 3/4
80%
4 of 5 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 5 completed trials
Clinical Trials (12)
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
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)
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
Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)
Diagnostic and Screening Study of Genetic Disorders