Principal Investigator
Enrollment Status
Clinical Trial ID
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.
Phase
N/A
Funding Agency/Sponsor
Industry
Disease
Other
Enrollment 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.