Condition

Wilson Disease clinical trials and research

• 12Clinical trials
• 1NIH research projects
• 8Countries

Newest Wilson Disease clinical trials

All recruiting trials (12)

• PM577a: a one-time gene-editing therapy for Wilson disease

  This study tests PM577a, a gene-editing treatment, to see if it is safe and may help people with Wilson disease who have a p.H1069Q ATP7B mutation.

  Recruiting at 2 sites Phase 1/2 Prime Medicine, Inc. · New Haven, Connecticut and 3 other locations · NCT07748403

• Best timing for 24-hour urine copper after a brief treatment break

  This will see if a 24-hour urine copper test taken after a short, supervised pause in treatment reflects body copper levels in stable Wilson disease patients.

  Recruiting Yale University · New Haven, Connecticut · NCT07301216

• Renal Involvement in Wilson's Disease

  This study tests how Wilson's disease affects the kidneys and who might be at risk for kidney problems.

  Recruiting Fondation Ophtalmologique Adolphe de Rothschild · Paris · NCT07075393

• Skin effects of D-penicillamine in people with Wilson disease

  This project will use silicone molds and confocal imaging to see if D-penicillamine changes skin elasticity in people with Wilson disease compared with matched healthy volunteers.

  Recruiting Centre Hospitalier Universitaire de Saint Etienne · Bron and 1 other locations · NCT06945081

• Evaluating LY-M003 Injection for Wilson Disease

  This study is testing a new gene therapy called LY-M003 to see if it is safe and effective for people with Wilson Disease.

  Recruiting Early Phase 1 First Affiliated Hospital of Zhejiang University · Hangzhou, Zhejiang · NCT06650319

• Registry for Rare Diseases

  This study is creating a detailed registry to collect information on rare diseases like amyloidosis and sarcoidosis to help improve how they are diagnosed and treated.

  Recruiting Hospital Italiano de Buenos Aires · Buenos Aires, Buenos Aires · NCT06573723

• Registry for Wilson Disease in Spain

  This study is collecting information about Wilson Disease in Spain to help understand how common it is and improve how it's diagnosed and treated.

  Recruiting Asociación Española para el Estudio del Hígado · Barcelona · NCT06466291

• Daily pattern of urinary copper in people with Wilson disease

  This study will test whether urinary copper levels change during the day in people with Wilson disease and whether single spot urine samples can reflect 24-hour copper loss.

  Recruiting Hospices Civils de Lyon · Bron, Rhone · NCT06430359

• Comparing oral health in Wilson's disease patients and healthy controls

  This study looks at the dental health of people with Wilson's disease compared to healthy individuals to see how the condition affects their teeth and mouth.

  Recruiting at 2 sites Fondation Ophtalmologique Adolphe de Rothschild · Ivry-sur-Seine and 1 other locations · NCT05444127

• Registry for patients with Wilson's disease in France

  This study is collecting information from people with Wilson's disease in France to better understand the condition and improve treatment options.

  Recruiting Fondation Ophtalmologique Adolphe de Rothschild · Paris, Île-de-France Region · NCT05231876

• Identifying factors for diagnosing Wilson disease

  This study looks at the symptoms and characteristics of Wilson disease in genetically diagnosed patients to see if they can find better ways to diagnose and treat people with this condition.

  Recruiting Second Affiliated Hospital, Zhejiang University, School of Medicine · Hangzhou, Zhejiang · NCT04965545

• Observing the natural progression of Wilson Disease

  This study is tracking patients with Wilson Disease to learn more about how the condition progresses and to help improve diagnosis and treatment in the future.

  Recruiting at 4 sites Yale University · New Haven, Connecticut and 5 other locations · NCT03334292

NIH-funded research (1)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Investigating copper toxicity and its effects on the brain in a mouse model of Wilson Disease

  This study is looking at Wilson Disease, a condition that causes too much copper to build up in the body, and it's testing new treatments, like zinc salts, to see if they can help reduce harmful effects on the brain and liver while being safer than current medications.

  NIH research University of Alaska Anchorage · Anchorage, United States · NIH-10574028

Related topics

Quick answers

How many Wilson Disease clinical trials are recruiting right now?

12, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 8 countries. The most sites are in France (5 trials), United States (3 trials), China (2 trials), Argentina (1 trial), Spain (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 1/2: 1, Early Phase 1: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

Find the studies that fit you

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