International registry for Wilson's Disease patients

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational registry collects data from Wilson's Disease patients during routine clinic visits at 6-12 month intervals. It aims to document the natural history of the disease, measure clinical aspects, and provide a comprehensive overview of patient experiences and treatment adherence. The registry will utilize standardized tools for data collection and will include a diverse patient population, including children and adults. Additionally, it seeks to enhance the generalizability of findings by comparing real-world outcomes with clinical trial data.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Patient is able to provide, and has provided, written informed consent/assent
2. Written documentation has been obtained in accordance with the relevant country and local privacy requirements, where applicable, including:

   1. For US sites: Authorization for Use and Release of Health Research Study Information
   2. For EU sites: Data Protection Consent
3. All patients diagnosed with WD including pre-symptomatic individuals and individuals with co-morbidities/diagnoses
4. Any treatments including prescribed and homeopathic/traditional therapies or naive patients on no therapy

Exclusion Criteria:

1\. Refusal of informed consent by either patient or their legally acceptable guardian

Where it's running

• University Hospital Leuven
  Leuven, Belgium Recruiting
• Hospices Civils de Lyon
  Bron, Auvergne-Rhône-Alpes, France Recruiting
• HF Adolphe de Rothschild
  Paris, Île-de-France Region, France Recruiting
• Charite-Univeritatsmedizin Berlin Hospital
  Berlin, Germany Recruiting
• Universitatsklinikum Dusseldorf
  Düsseldorf, Germany Recruiting
• Hannover Medical School (MHH)
  Hanover, Germany Recruiting

Study contacts

• Study coordinator: Carla Bennett
• Email: clinicaloperations@orphalan.com
• Phone: +44 (0)7918380893

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• 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

• 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

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.