Condition
MPS IIIA
Total Trials
3
Recruiting
1
Active
1
Completed
0
Key Insights
Highlights
No notable highlights available yet
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 80/100
Termination Rate
33.3%
1 terminated out of 3 trials
Success Rate
0.0%
-86.5% vs benchmark
Late-Stage Pipeline
33%
1 trials in Phase 3/4
Results Transparency
0%
1 of 0 completed with results
Key Signals
1 with results0
Data Visualizations
Phase Distribution
3Total
P 1 (1)
P 2 (1)
P 3 (1)
Trial Status
Recruiting1
Enrolling By Invitation1
Terminated1
Clinical Trials (3)
Showing 3 of 3 trials
NCT02716246Phase 2RecruitingPrimary
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
NCT04360265Phase 3Enrolling By Invitation
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
NCT04088734Phase 1TerminatedPrimary
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
Showing all 3 trials