Wilson Disease
49
12
17
21
Key Insights
Highlights
Success Rate
88% trial completion (above average)
Published Results
11 trials with published results (22%)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
6.1%
3 terminated out of 49 trials
87.5%
+0.9% vs benchmark
12%
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: 86.6%
Based on 21 completed trials
Clinical Trials (49)
Description of Renal Involvement in Wilson's Disease
A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD)
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
Subclinical Myocardial Dysfunction in Children With Wilson's Disease
An Exploratory Study to Evaluate the Tolerability and Safety of MWAV201 in Subjects With Wilson Disease
Early Check: Expanded Screening in Newborns
Natural History of 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
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