Wilson Disease
47
11
12
21
Key Insights
Highlights
Success Rate
88% trial completion (above average)
Published Results
11 trials with published results (23%)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
6.4%
3 terminated out of 47 trials
87.5%
+1.0% vs benchmark
13%
6 trials in Phase 3/4
52%
11 of 21 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 21 completed trials
Clinical Trials (47)
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
Prescreening Study to Identify Potential Wilson Disease Participants for Gene-Editing Clinical Trial
Off Treatment Urinary Copper Excretion in Wilson Disease, Pilot Study
A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease
Wilson's Disease Treated With D-Penicillamine: Characterization of Skin Damage Secondary to Treatment by Measuring Skin Elasticity
Circadian Variation of Urinary Copper Excretion in Wilson Disease Patients
Institutional Registry of Rare Diseases
Oral Health and Wilson's Disease: SOMAWI
Description of Renal Involvement in Wilson's Disease
Real World Evidence Study in Subjects With Wilson's Disease
Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310 Injection in Patients With Wilson's Disease (WD)
Gene Therapy for Wilson Disease Evaluated by 64Cu PET/CT
Baby Detect : Genomic Newborn Screening
Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease
The Exploratory Handheld Optical Coherence Tomography Study in Wilson's Disease
Natural History of Wilson Disease
Early Check: Expanded Screening in Newborns
The Clinical Study of Botulinum Toxin Type A Injection in the Treatment of Wilson Disease
French Wilson Disease Registry
Daily Versus Alternate Day Plasma Exchange in Wilson Disease With Acute Liver Failure in Children