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

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 - PACUS

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

DRKS

Registry ID

DRKS00028305

Enrollment

Registered

2022-03-08

Start date

2022-04-01

Completion date

Unknown

Last updated

2025-04-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

E83.0

Interventions

Group 1: Adult patients diagnosed with Wilson disease (ICD-10: E83.0) and treated with DPA, who develop an intolerance towards DPA will be eligible. Patients will switch from DPA to Cuprior® treatment

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Inclusion criteria

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

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).

Design outcomes

Primary

Measure	Time frame
To evaluate quality of life of Wilson disease patients under Cuprior® therapy. 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.	—

Measure

Time frame

To evaluate quality of life of Wilson disease patients under Cuprior® therapy. 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

Measure	Time frame
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.	—

Countries

Germany

Contacts

Public ContactMichaela Siebert

Orphalan GmbH

michaela.siebert@orphalan.com+49 69959325022

Outcome results

None listed

Source: DRKS (via WHO ICTRP) · Data processed: Feb 4, 2026