Condition
Mucopolysaccharidosis Type IIIA
Total Trials
4
Recruiting
0
Active
2
Completed
1
Success Rate
100.0%+13% vs avg
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
Termination Rate
0.0%
0 terminated out of 4 trials
Success Rate
100.0%
+13.5% vs benchmark
Late-Stage Pipeline
0%
0 trials in Phase 3/4
Results Transparency
0%
0 of 1 completed with results
Key Signals
100% success
Data Visualizations
Phase Distribution
3Total
P 1 (2)
P 2 (1)
Trial Status
Active Not Recruiting2
Completed1
Unknown1
Trial Success Rate
100.0%
Benchmark: 86.5%
Based on 1 completed trials
Clinical Trials (4)
Showing 4 of 4 trials
NCT06181136Phase 1Active Not RecruitingPrimary
Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)
NCT04201405Phase 1Active Not RecruitingPrimary
Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA
NCT03612869Phase 2UnknownPrimary
Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
NCT02037880CompletedPrimary
Natural History Studies of Mucopolysaccharidosis III
Showing all 4 trials