Urea Cycle Disorder
20
2
3
11
Key Insights
Highlights
Success Rate
73% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 59/100
20.0%
4 terminated out of 20 trials
73.3%
-13.2% vs benchmark
10%
2 trials in Phase 3/4
55%
6 of 11 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 11 completed trials
Clinical Trials (20)
Dysregulated Urea-synthesis at Terminal Uremia
Liver Disease in Urea Cycle Disorders
Hepatic Histopathology in Urea Cycle Disorders
MyRareDiet A Novel Diet Tracking Tool
A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders
Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)
Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)
HepaStem Long-Term Safety Registry
Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM)
Systemic Biomarkers of Brain Injury From Hyperammonemia
Th1, Th2, Th17 Phenotype in Urea Cycle Disorders
Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLD
Chronic Liver Disease in Urea Cycle Disorders
UNLOCKED: A Phase 2, Open-label Trial With KB195 in Subjects With a Urea Cycle Disorder
Manipulating the Gut Microbiome Study
A Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCD
Safety and Tolerability of SYNB1020-CP-001
A Safety and Efficacy Study of Infusions of HepaStem in Urea Cycle Disorders Pediatric Patients
Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism
Neurologic Injuries in Adults With Urea Cycle Disorders