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

Completed

Conditions studied: Wilson Disease

In brief

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.

Key facts

Study ID

NCT04531189

Run by

Ultragenyx Pharmaceutical Inc

People needed

16

Starts

2020-12-11

Expected to finish

2022-03-25

Last updated by the study team

2022-04-29

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male or female ≥ 12 years of age at the time written informed consent is provided.
• Confirmed diagnosis of Wilson disease.
• 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.
• 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.
• 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.

You may not qualify if…

• History of liver disease due to a medical condition unrelated to Wilson disease.
• Liver fibrosis stage F3 or F4.
• Decompensated hepatic cirrhosis and/or evidence of portal hypertension.
• Marked neurological disease requiring either nasogastric feeding or intensive inpatient medical care.
• Female subject who is pregnant or breastfeeding or who plans to become pregnant at any time during the study.
• 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.
• Current or previous participation in a gene transfer study.
• 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.

Where it is running

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

Full record on ClinicalTrials.gov

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