Condition
Mucopolysaccharidosis IIIA
Total Trials
4
Recruiting
0
Active
1
Completed
2
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: 40/100
Termination Rate
0.0%
0 terminated out of 4 trials
Success Rate
100.0%
+13.5% vs benchmark
Late-Stage Pipeline
25%
1 trials in Phase 3/4
Results Transparency
0%
0 of 2 completed with results
Key Signals
100% success
Data Visualizations
Phase Distribution
2Total
P 1 (1)
P 3 (1)
Trial Status
Completed2
Enrolling By Invitation1
Active Not Recruiting1
Trial Success Rate
100.0%
Benchmark: 86.5%
Based on 2 completed trials
Clinical Trials (4)
Showing 4 of 4 trials
NCT04360265Phase 3Enrolling By InvitationPrimary
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
NCT06095388Phase 1Active Not RecruitingPrimary
Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA
NCT05523206CompletedPrimary
A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)
NCT02746341CompletedPrimary
Natural History Study of Patients With MPS IIIA
Showing all 4 trials