Condition
Tay Sachs Disease
Total Trials
6
Recruiting
0
Active
1
Completed
4
Success Rate
80.0%-7% vs avg
Key Insights
Highlights
Success Rate
80% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 59/100
Termination Rate
16.7%
1 terminated out of 6 trials
Success Rate
80.0%
-6.5% vs benchmark
Late-Stage Pipeline
0%
0 trials in Phase 3/4
Results Transparency
50%
2 of 4 completed with results
Key Signals
2 with results80% success
Data Visualizations
Phase Distribution
4Total
Not Applicable (1)
P 1 (1)
P 2 (2)
Trial Status
Completed4
Terminated1
Active Not Recruiting1
Trial Success Rate
80.0%
Benchmark: 86.5%
Based on 4 completed trials
Clinical Trials (6)
Showing 6 of 6 trials
NCT06614569Active Not Recruiting
Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease
NCT01372228Phase 1Terminated
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
NCT00383448Phase 2Completed
HSCT for High Risk Inherited Inborn Errors
NCT00176904Phase 2Completed
Stem Cell Transplant for Inborn Errors of Metabolism
NCT01999257Not ApplicableCompletedPrimary
Efficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent.
NCT01869270CompletedPrimary
Gene Therapy for Tay-Sachs Disease
Showing all 6 trials