- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04531189
Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease
April 28, 2022 updated by: Ultragenyx Pharmaceutical Inc
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.
Study Overview
Status
Completed
Conditions
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
Enrollment (Actual)
16
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Baden-Württemberg
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Heidelberg, Baden-Württemberg, Germany, 69120
- UniversitatsKlinikum Heidelberg
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-
-
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Connecticut
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New Haven, Connecticut, United States, 06510
- Yale University School Of Medicine
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Florida
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Miami, Florida, United States, 33136
- Jackson Memorial Hospital Miami Transplant Unit University of Miami Miller School of Medicine
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Illinois
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Chicago, Illinois, United States, 60611
- Northwestern University Feinberg School of Medicine
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Michigan
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Ann Arbor, Michigan, United States, 48109
- University of Michigan Michigan Medicine Hepatology Clinic Taubman Center
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Washington
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Seattle, Washington, United States, 98105
- Seattle Children's Hospital
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-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
12 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Sampling Method
Non-Probability Sample
Study Population
Subjects with a confirmed diagnosis of Wilson Disease will be enrolled from outpatient clinics and metabolic genetic centers in the United States.
Subjects may participate regardless of their current treatment.
Subjects who have had a liver transplant for treatment of Wilson Disease are also invited to participate.
Description
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Prospective
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Clinical manifestation of Wilson Disease under study: demographics
Time Frame: 30 days
|
30 days
|
Clinical manifestation of Wilson Disease under study: general medical history, Wilson Disease history and treatments
Time Frame: 30 days
|
30 days
|
Clinical manifestation of Wilson Disease under study: patient reported outcomes and clinician reported outcomes
Time Frame: 30 days
|
30 days
|
Clinical manifestation of Wilson Disease under study: activity monitoring
Time Frame: 30 days
|
30 days
|
Clinical manifestation of Wilson Disease under study: motor function
Time Frame: 30 days
|
30 days
|
Clinical manifestation of Wilson Disease under study: joint pain
Time Frame: 30 days
|
30 days
|
Clinical manifestation of Wilson Disease under study: serum copper biomarker assessments
Time Frame: 30 days
|
30 days
|
Clinical manifestation of Wilson Disease under study: 24-hour urinary copper concentration
Time Frame: 30 days
|
30 days
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Ultragenyx Medical Director, Ultragenyx Pharmaceutical
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Helpful Links
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
December 11, 2020
Primary Completion (Actual)
March 25, 2022
Study Completion (Actual)
March 25, 2022
Study Registration Dates
First Submitted
July 27, 2020
First Submitted That Met QC Criteria
August 24, 2020
First Posted (Actual)
August 28, 2020
Study Record Updates
Last Update Posted (Actual)
April 29, 2022
Last Update Submitted That Met QC Criteria
April 28, 2022
Last Verified
April 1, 2022
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Digestive System Diseases
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Liver Diseases
- Genetic Diseases, Inborn
- Basal Ganglia Diseases
- Movement Disorders
- Neurodegenerative Diseases
- Metabolism, Inborn Errors
- Heredodegenerative Disorders, Nervous System
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Metal Metabolism, Inborn Errors
- Hepatolenticular Degeneration
Other Study ID Numbers
- UX701-CL001
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
No
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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