OTC Deficiency
5
1
3
1
Key Insights
Highlights
Success Rate
50% trial completion
Treatment Research
7 treatments under study, led by Arct810
Research Leaders
5 sponsors active, led by Arcturus Therapeutics, Inc.
Related Conditions
Often studied with 12 related conditions
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 75/100
20.0%
1 terminated out of 5 trials
50.0%
-36.6% 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: 86.6%
Based on 1 completed trials
Clinical Trials (5)
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous OTC Genotype
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
Research Network
Common Treatments
- Arct8102
- Survey1
- Cmpcps0011
- Avalotcagene Ontaparvovec1
- Prednisolone1
Active Sponsors
- Arcturus Therapeutics, Inc.2
- University Children's Hospital, Zurich1
- Ultragenyx Pharmaceutical Inc1
- Camp4 Therapeutics Corp.1
- CAMP4 Therapeutics Corporation1
Top Investigators
University Children's Hospital, Zurich
Erasmus Medical Center
Q-Pharm Pty Ltd
Related Conditions
- Otcd2
- Ornithine Transcarbamylase Deficiency2
- Inborn Errors Of Metabolism1
- Aminoacidopathy1
- Phenylketonurias1