Condition
PAH Deficiency
Total Trials
4
Recruiting
0
Active
0
Completed
0
Key Insights
Highlights
No notable highlights available yet
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 90/100
Termination Rate
50.0%
2 terminated out of 4 trials
Success Rate
0.0%
-86.5% vs benchmark
Late-Stage Pipeline
25%
1 trials in Phase 3/4
Results Transparency
0%
0 of 0 completed with results
Key Signals
0
Data Visualizations
Phase Distribution
3Total
P 1 (2)
P 4 (1)
Trial Status
Terminated2
Enrolling By Invitation1
Unknown1
Clinical Trials (4)
Showing 4 of 4 trials
NCT05222178Phase 1Terminated
Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency
NCT03952156Phase 1Terminated
Gene Therapy Clinical Study in Adult PKU
NCT04348708Enrolling By Invitation
Long-Term Follow Up Study of Subjects Previously Administered HMI 102
NCT04227080Phase 4Unknown
BH4 Responsiveness in PAH Deficiency PKU Patients
Showing all 4 trials