Prospective, descriptive, non-interventional, German multicenter study to evaluate the quality of life of Wilson disease patients after switching from D-penicillamine to trientine tetrahydrochloride and to assess clinical and patient reported outcomes

Conditions: E83.0
Disorders of copper metabolism

Registration Number: DRKS00028305

Lead Sponsor: Orphalan GmbH

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting withdrawn before recruiting started

Sex: All

Target Recruitment: 60

Inclusion Criteria

Patients with provided written informed consent form (ICF) and a data protection consent.
Adult patients (= 18 years) of any gender diagnosed with Wilson disease (ICD-10 E83.0).
Patients previously treated with DPA with intolerance of the medication, which leads to a medication switch to Cuprior®. Patients should require a therapy switch from current DPA to Cuprior® treatment based on physician assessment and approval and before enrollment in the study.

Exclusion Criteria

Patients without filed prescription of Cuprior® or existing evidence of decision to switch.
Patient without recorded manifestation of DPA intolerance.
Patients with anticipated impossibility to be followed up for any reasons.
Patients switched to other Wilson disease therapies (trientine dihydrochloride, zinc salts).

Study & Design

Study Type: observational

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
To evaluate quality of life of Wilson disease patients under Cuprior® therapy.<br>After the decision to switch the therapy to Cuprior® is made by the treating physician, based on DPA intolerance, patients will be asked to participate and baseline data will be collected (V0). After therapy switch, patients will be observed for 12 months across three follow-up visits (V1 - V3). Data collection will take place via an electronic questionnaire (eCRF) filled out by the attending physician and by a questionnaire in paper form filled out by their patient.

Secondary Outcome Measures

Name	Time	Method
To evaluate treatment adherence of Wilson disease patients under Cuprior® therapy, to collect indications for treatment switch from DPA to Cuprior®, to track and report the clinical and laboratory parameters which led to the treatment switch, under Cuprior® therapy in daily practice.