OTC Deficiency
5
1
3
1
Key Insights
Highlights
Success Rate
50% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 75/100
20.0%
1 terminated out of 5 trials
50.0%
-36.5% vs benchmark
20%
1 trials in Phase 3/4
0%
0 of 1 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 1 completed trials
Clinical Trials (5)
Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous OTC Genotype
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
Study for Adolescents and Adults With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810
A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD
Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism