Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease

Wilson Disease Clinical Trial

Official title:

Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04531189
• Other study ID #: UX701-CL001
• Status: Completed
• Start date: December 11, 2020
• Est. completion date: March 25, 2022

Study information

• Verified date: April 2022
• Source: Ultragenyx Pharmaceutical Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The primary objective of the study is to determine the relevance and appropriateness of outcome assessments, including biomarkers, within the Wilson disease population to inform study design and endpoint selection for future clinical studies.

Description:

Given the limited information on the frequency and spectrum of disease manifestations and clinical course of Wilson disease, the UX701-CL001 study aims to assess the utility and feasibility of various assessments and biomarkers to inform endpoint selection for future clinical studies, better understand the relationship between biomarkers and potential clinical outcomes, and characterize the clinical presentation of Wilson disease. UX701-CL001 is a clinical survey study. Subjects will complete assessments at the study site and at home to evaluate the clinical manifestations of Wilson disease in clinical and real-world environments.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 16
• Est. completion date: March 25, 2022
• Est. primary completion date: March 25, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

Inclusion Criteria:

1. Male or female = 12 years of age at the time written informed consent is provided.

2. Confirmed diagnosis of Wilson disease.

3. Have a documented history of copper chelator (ie, penicillamine, trientine) and/or zinc therapy or be = 1 year post liver transplant with no active associated complications.

4. Willing and able to comply with all study procedures and requirements. If < 18 years of age (or as required by region), have a caregiver who is willing and able to assist with study requirements if needed.

5. Willing and able to provide written informed consent after the study has been explained and before any study-related data are collected or study-related procedures are performed. If < 18 years of age (or as required by region), willing and able to provide written assent and have a legally authorized representative who is willing and able to provide written informed consent after the study has been explained and before any study-related data are collected or study-related procedures are performed.

Exclusion Criteria:

1. History of liver disease due to a medical condition unrelated to Wilson disease.

2. Liver fibrosis stage F3 or F4.

3. Decompensated hepatic cirrhosis and/or evidence of portal hypertension.

4. Marked neurological disease requiring either nasogastric feeding or intensive inpatient medical care.

5. Female subject who is pregnant or breastfeeding or who plans to become pregnant at any time during the study.

6. Female subject of childbearing potential who has a positive urine pregnancy test on Day 1 or is unwilling to have additional pregnancy tests during the study.

7. Current or previous participation in a gene transfer study.

8. Presence or history of any disease or condition that, in the Investigator's opinion, would interfere with the subject's safety or ability to participate in the study or significantly affect interpretation of study results.

Related Conditions & MeSH terms

• Wilson Disease

Locations

Country	Name	City	State
Germany	Universitätsklinikum Heidelberg	Heidelberg	Baden-Württemberg
United States	University of Michigan Michigan Medicine Hepatology Clinic Taubman Center	Ann Arbor	Michigan
United States	Northwestern University Feinberg School of Medicine	Chicago	Illinois
United States	Jackson Memorial Hospital Miami Transplant Unit University of Miami Miller School of Medicine	Miami	Florida
United States	Yale University School of Medicine	New Haven	Connecticut
United States	Seattle Children's Hospital	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Ultragenyx Pharmaceutical Inc

Countries where clinical trial is conducted

United States,  Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Clinical manifestation of Wilson Disease under study: demographics		30 days
Primary	Clinical manifestation of Wilson Disease under study: general medical history, Wilson Disease history and treatments		30 days
Primary	Clinical manifestation of Wilson Disease under study: patient reported outcomes and clinician reported outcomes		30 days
Primary	Clinical manifestation of Wilson Disease under study: activity monitoring		30 days
Primary	Clinical manifestation of Wilson Disease under study: motor function		30 days
Primary	Clinical manifestation of Wilson Disease under study: joint pain		30 days
Primary	Clinical manifestation of Wilson Disease under study: serum copper biomarker assessments		30 days
Primary	Clinical manifestation of Wilson Disease under study: 24-hour urinary copper concentration		30 days

External Links

•   Ultragenyx Patient Advocacy/Wilson Disease Information