Condition

Wilson's Disease clinical trials and research

• 8Clinical trials
• 11Countries

Newest Wilson's Disease clinical trials

All recruiting trials (8)

• 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

• 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

• One-a-day trientine for newly diagnosed Wilson's disease

  A new once-daily trientine formulation is being tested against d-Penicillamine as a first-line treatment for people with Wilson's disease, including both children 8 and older and adults.

  Recruiting at 5 sites Phase 3 Orphalan · Denver, Colorado and 9 other locations · NCT07465718

• ATP7B mRNA (DSL101) therapy for Wilson disease

  This treatment will test whether one or more IV infusions of ATP7B mRNA (DSL101) are safe and help control copper in adults with genetically confirmed Wilson disease.

  Recruiting Early Phase 1 DSciLab Co., Ltd. · Hefei, Anhui · NCT07240896

• Investigating heart issues in Wilson's Disease

  This study is testing how Wilson's Disease affects the heart in adults by using long-term heart monitoring to see if copper buildup causes any problems.

  Recruiting Fondation Ophtalmologique Adolphe de Rothschild · Paris · NCT05493605

• International registry for Wilson's Disease patients

  This study is gathering information from people with Wilson's Disease during their regular doctor visits to better understand the disease and how patients are managing it.

  Recruiting at 16 sites Orphalan · Leuven and 15 other locations · NCT05239858

• Examining copper levels in breast milk of pregnant women with Wilson's disease

  This study tests how treatment for Wilson's disease affects copper levels in the breast milk of pregnant women to ensure the health of both mom and baby during breastfeeding.

  Recruiting Fondation Ophtalmologique Adolphe de Rothschild · Paris · NCT05183165

• Observational study on Wilson's Disease

  This study looks at how Wilson's Disease affects people in different ways to help improve care for patients and their families.

  Recruiting First Affiliated Hospital of Fujian Medical University · Fuzhou · NCT04012658

Related topics

Quick answers

How many Wilson's Disease clinical trials are recruiting right now?

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

Where are these trials running?

In 11 countries. The most sites are in France (3 trials), China (3 trials), United States (3 trials), New Zealand (2 trials), Saudi Arabia (2 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 1/2: 2, Early Phase 1: 1, Phase 3: 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.

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