International Wilson's Disease Patient Registry (iWilson Registry)

April 29, 2024 updated by: Orphalan
Longitudinal, observational, non-interventional, standard of care Registry. Data will be collected from the routinely scheduled WD clinic visits at approximately 6-12 month intervals. At enrolment, in addition to data from the clinic visit, retrospective data will be collected from the diagnostic evaluation and any relevant past medical history and a summary of WD medication history.

Study Overview

Status

Recruiting

Conditions

Detailed Description

This is a retrospective/prospective observational registry without a control group. A standardised collection tool will be utilised to capture and compare data. The registry will use common data elements, guided by a literature review, and expert input from the multidisciplinary advisory board. The SF-12 Health Survey will be requested from patients aged 18 and over at entry to the Registry to provide a snapshot of baseline quality of life assessment in patients with WD. This will enhance generalisability (external validity) and comparability with clinical trial data.

The objectives of the registry are:

  1. Natural history of diagnosis and treated WD patients by documentation of observed clinical practice and patient experience
  2. Measure, and compare epidemiological and clinical aspects of Wilson Disease (WD) in the participating countries, thereby encouraging the identification of new standards for the management of WD
  3. To provide data for epidemiological research and identify groups of patients potentially eligible for multi-centre trials
  4. Describe clinical pathways, by documenting standardised examinations and tests used to monitor WD patients including tools to evaluate adherence and compliance to therapies using real-world outcomes.
  5. To be more inclusive (heterogeneous patient population; paediatric, adults, elderly) using much broader inclusion criteria and fewer exclusion criteria than commonly used in randomised trials.
  6. Longer follow-up compared to RCTs beneficial detecting delayed or long-term benefits or harm and surveillance for rare events of the disease itself or therapies

Patients will be seen as per their usual standard of care visits. Data points of interest for the Registry will be collected. These may include but not limited to: Medical history, Wilson's Disease Diagnosis and Medical History, Genetics, Liver Assessments, Fibrosis Stage, Liver Transplant, Neurological Assessments, Psychological Assessments, Clinical Global Impression of Change Scale (CGIC), Laboratory measurements incl. Urinary Copper Excretion and Free Copper (NCC), Physical Examination, Adverse Reactions and Serious Adverse Reactions documented by physicians

Study Type

Observational

Enrollment (Estimated)

500

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Belgium
      • Leuven, Belgium
        • Recruiting
        • University Hospital Leuven
        • Principal Investigator:
          • David Cassiman, Dr.
  • France
    • Auvergne-Rhone-Alpes
      • Bron, Auvergne-Rhone-Alpes, France, 69677
        • Recruiting
        • Hospices Civils de Lyon
        • Principal Investigator:
          • Eduardo Couchonnal Bedoya
    • Île-de-France
      • Paris, Île-de-France, France
        • Recruiting
        • HF Adolphe de Rothschild
        • Principal Investigator:
          • Aurelia Poujois
  • Germany
      • Berlin, Germany
        • Recruiting
        • Charite-Univeritatsmedizin Berlin Hospital
        • Principal Investigator:
          • Frank Tacke, Dr.
      • Düsseldorf, Germany
        • Recruiting
        • Universitätsklinikum Düsseldorf
        • Principal Investigator:
          • Christian Hartmann, Dr.
      • Hannover, Germany
        • Recruiting
        • Hannover Medical School (MHH)
        • Principal Investigator:
          • Heiner Wedemeyer, Dr.
      • Leipzig, Germany
        • Recruiting
        • Universitätsklinikum Leipzig
        • Principal Investigator:
          • Thomas Berg, Dr.
  • Poland
      • Warsaw, Poland
        • Recruiting
        • Institute of Psychiatry and Neurology
        • Principal Investigator:
          • Anna Czlonkowska, Dr.
      • Warsaw, Poland
        • Recruiting
        • The Children's Memorial Health Institute
        • Principal Investigator:
          • Piotrem Socha, Prof.
  • Saudi Arabia
    • Ar Riyāḑ
      • Riyāḑ, Ar Riyāḑ, Saudi Arabia, 11564
        • Recruiting
        • King Faisal Specialist Hospital in Riyadh
        • Principal Investigator:
          • Saad Alghamdi
  • Spain
      • Barcelona, Spain
        • Recruiting
        • University Hospital Clínic de Barcelona, C. de Villarroel
        • Principal Investigator:
          • Zoe Marino, Dr.
      • Sevilla, Spain
        • Recruiting
        • Hospital Universitario Virgen del Rocío
        • Principal Investigator:
          • Javier Ampuero, Dr.
      • Valencia, Spain
        • Recruiting
        • Hospital Universitario y Politecnico La Fe
        • Principal Investigator:
          • Marina Berenguer, Dr.
    • Canary Islands
      • Las Palmas De Gran Canaria, Canary Islands, Spain
        • Recruiting
        • Hospital Universitario GC Dr Negrín
        • Principal Investigator:
          • Quinones, Dr.
  • United Kingdom
      • Leeds, United Kingdom
        • Recruiting
        • Leeds Teaching Hospitals NHS Trust
        • Principal Investigator:
          • Joanna Moore, Dr.
      • London, United Kingdom
        • Recruiting
        • Royal Free Hospital
        • Principal Investigator:
          • Emmanouil Tsochatzis, Professor

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

The Registry aims to be as inclusive as possible. All patients with WD (adult and paediatric ≥12) who provide informed consent/assent can be included. They will be approached during regular follow up visits for ongoing care of their Wilson's Disease by their primary physician.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Describe the natural history of treated WD
Time Frame: 5 years
Describe the natural history of treated WD and to explore the clinical phenotype, geographic, ethnic and gender influences on the course of the condition.
5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Orphalan

Collaborators

Ergomed

Investigators

  • Study Chair: Pramod Mistry, PhD, Yale University Hospital, Conneticut

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 29, 2022

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

December 1, 2027

Study Registration Dates

First Submitted

February 4, 2022

First Submitted That Met QC Criteria

February 4, 2022

First Posted (Actual)

February 15, 2022

Study Record Updates

Last Update Posted (Actual)

April 30, 2024

Last Update Submitted That Met QC Criteria

April 29, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ORPH-131-005

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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