Prescreening Study to Identify Potential Participants for a Clinical Study in Wilson Disease (WD) Due to the ATP7B p.H1069Q or p.R778L Mutations
Status: Recruiting
Location: See all (7) locations...
Study Type: Observational
SUMMARY
The aim of this study is to inform and improve future clinical trials in Wilson Disease (WD) by better understanding how patients with WD are living with and managing the disease, and by identifying key factors that shape their decisions to participate in clinical research.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:
• Confirmed Wilson Disease (WD) as determined by the following criteria:
‣ An established clinical diagnosis of WD
⁃ Genetic analysis confirming the presence of biallelic pathogenic variants at ATP7B, at least one of which is EITHER p.H1069Q OR p.R778L OR Participants without a confirmed genetic diagnosis may enroll only with explicit approval from the Medical Monitor
Locations
United States
California
University of California Los Angeles Medical Center
RECRUITING
Los Angeles
University of California Davis Health
RECRUITING
Sacramento
Connecticut
Yale New Haven Hospital
RECRUITING
New Haven
Illinois
Northwestern University
RECRUITING
Chicago
Massachusetts
Massachusetts General Hospital
RECRUITING
Boston
Michigan
University of Michigan Medicine
RECRUITING
Ann Arbor
Texas
American Research Corporation
RECRUITING
San Antonio
Time Frame
Start Date: 2025-12-29
Estimated Completion Date: 2027-12
Participants
Target number of participants: 30
Treatments
Participants with at least 1 allele with the ATP7B p.H1069Q or p.R778L mutation
Related Therapeutic Areas
Sponsors
Leads: Prime Medicine, Inc.