Urea Cycle Disorders
26
4
4
19
Key Insights
Highlights
Success Rate
90% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
7.7%
2 terminated out of 26 trials
90.5%
+4.0% vs benchmark
15%
4 trials in Phase 3/4
42%
8 of 19 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 19 completed trials
Clinical Trials (26)
A Clinical Study of Glycerol Phenylbutyrate in Chinese Patients With Urea Cycle Disorders
Ureagenesis Analysis in Healthy Subjects and in Urea Cycle Disorder Patients
At Home Ammonia Monitoring of Inborn Errors of Ammonia Metabolism
To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)
Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)
Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders
Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders
Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)
Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders
Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders
A Study of Glyceryl Tri-(4-phenylbutyrate) Administered Orally as a Single Dose, and Twice Daily for Seven Consecutive Days to Subjects With Hepatic Impairment With Cirrhosis and to a Control Group
Longitudinal Study of Urea Cycle Disorders
PCORI Urea Cycle Disorder Study
Safety Study of HepaStem for the Treatment of Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN)
Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)
Nitric Oxide Supplementation in Argininosuccinic Aciduria
Clinical Food Study to Evaluate the Effect of KB195 on Gut Nitrogen Metabolism in Patients With Urea Cycle Disorders
Long-term Safety Follow-up Study of Patients Having Received HepaStem (SAF001)
Pilot Study: Urea Cycle Disorders Practice Patterns and Outcomes Assessment
Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder