Brief Title
Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease
Official Title
Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease
Brief 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.
Detailed 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.
Study Type
Observational
Primary Outcome
Clinical manifestation of Wilson Disease under study: demographics
Condition
Wilson Disease
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Estimated Enrollment
35
Start Date
December 11, 2020
Completion Date
March 2021
Primary Completion Date
March 2021
Eligibility Criteria
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.
Gender
All
Ages
12 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Ultragenyx Medical Director, 1-415-483-8800, [email protected]
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT04531189
Organization ID
UX701-CL001
Responsible Party
Sponsor
Study Sponsor
Ultragenyx Pharmaceutical Inc
Study Sponsor
Ultragenyx Medical Director, Study Director, Ultragenyx Pharmaceutical
Verification Date
January 2021