Condition
Mucopolysaccharidosis III
Total Trials
5
Recruiting
2
Active
2
Completed
1
Success Rate
50.0%-37% vs avg
Key Insights
Highlights
Success Rate
50% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 60/100
Termination Rate
20.0%
1 terminated out of 5 trials
Success Rate
50.0%
-36.5% vs benchmark
Late-Stage Pipeline
0%
0 trials in Phase 3/4
Results Transparency
100%
1 of 1 completed with results
Key Signals
1 with results50% success
Data Visualizations
Phase Distribution
4Total
P 1 (2)
P 2 (2)
Trial Status
Recruiting2
Completed1
Not Yet Recruiting1
Terminated1
Trial Success Rate
50.0%
Benchmark: 86.5%
Based on 1 completed trials
Clinical Trials (5)
Showing 5 of 5 trials
NCT02716246Phase 2Recruiting
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
NCT06333041Phase 2Not Yet Recruiting
Study of Cannabidiol in Sanfilippo Syndrome
NCT06036693Recruiting
MPS (RaDiCo Cohort) (RaDiCo-MPS)
NCT04018755Phase 1CompletedPrimary
Open-label Study of Anakinra in MPS III
NCT04088734Phase 1Terminated
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
Showing all 5 trials