Condition
Mucopolysaccharidosis Type 7
Total Trials
3
Recruiting
0
Active
0
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
Low Risk
Score: 25/100
Termination Rate
0.0%
0 terminated out of 3 trials
Success Rate
100.0%
+13.5% 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 results100% success
Data Visualizations
Phase Distribution
1Total
P 1 (1)
Trial Status
Enrolling By Invitation1
Completed1
Unknown1
Trial Success Rate
100.0%
Benchmark: 86.5%
Based on 1 completed trials
Clinical Trials (3)
Showing 3 of 3 trials
NCT03655223Enrolling By Invitation
Early Check: Expanded Screening in Newborns
NCT01856218Phase 1CompletedPrimary
An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
NCT02097251UnknownPrimary
An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
Showing all 3 trials