RecruitingPhase 1 / Phase 2NCT07748403
Metabolic
Wilson Disease
Also known as Hepatolenticular degeneration, ATP7B deficiency
Wilson disease is caused by mutations in ATP7B, which codes for a copper-transporting protein in the liver. Without it, copper accumulates in the liver, brain, and other organs.
ORPHA:905 ↗Gene ATP7BPrevalence 1-9 per 100,000 (Orphanet)Onset Adolescent, Adult, ChildhoodGenetic (autosomal recessive)
20
studies recruiting now
as of 7 Sept 2026
84
studies registered in total
as of 7 Sept 2026
10
countries with a recruiting site
as of 7 Sept 2026
5 Aug 2026
most recent study posted
among recruiting studies
Recruiting trials
RecruitingNCT05239858
International Wilson's Disease Patient Registry (iWilson Registry)
RecruitingNCT03334292
Natural History of Wilson Disease
RecruitingNCT07301216
Off Treatment Urinary Copper Excretion in Wilson Disease, Pilot Study
RecruitingEarly phase 1NCT06650319
A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 20 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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About Wilson Disease
Wilson disease is caused by mutations in ATP7B, which codes for a copper-transporting protein in the liver. Without it, copper accumulates in the liver, brain, and other organs. It presents as liver disease, neurological symptoms (tremor, dysarthria, psychiatric changes), and Kayser-Fleischer rings in the cornea. Wilson disease is one of the few genetic conditions that can be effectively treated with copper chelation therapy.
Common clinical features
HepatomegalyAnemiaThrombocytopeniaClumsinessArthralgiaBone painWeight lossPathologic fracture
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
5 approved treatments and 7 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Approved: Penicillamine (Cuprimine)Approved: Zinc Acetate (Galzin)Approved: TrientineApproved: Trientine Hydrochloride (Cufence)Approved: Trientine Tetrahydrochloride (Cuprior)
Phase 3Tiomolibdate Choline
Phase 3Zinc Acetate Anhydrous (Zinc acetate anhydrous component of nano-dtpa capsule zinc acetate)
Phase 2Nimatpagene Pariparvovec
Phase 1/2Rivunatpagene Miziparvovec
Phase 1 (early)Succimer (Chemet)
Phase 1 (early)Zinc Gluconate
Phase 1 (early)2,3-Dimercapto-1-Propanesulfonic Acid
Before you apply
Things trial teams commonly ask about for Wilson Disease. Not eligibility rules; those are set by each study.
- Current standard treatment (penicillamine, trientine, zinc) history is typically required at enrollment
- Liver function and degree of neurological involvement determine which trials apply
- Some trials target patients intolerant to existing copper chelators
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).