Prescreening Study to Identify Potential Wilson Disease Participants for Gene-Editing Clinical Trial
Running, not enrolling
Conditions studied: Wilson Disease
In brief
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.
Key facts
- Study ID
- NCT07226622
- Run by
- Prime Medicine, Inc.
- People needed
- 30
- Starts
- 2025-12-29
- Expected to finish
- 2027-12-01
- Last updated by the study team
- 2026-05-12
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- Prior history of gene therapy, liver transplantation, hepatocyte (cellular) transplantation, or active listing for liver transplantation
- For individuals with known ATP7B genotype: individual does not have at least 1 ATP7B allele with either the p.H1069Q or p.R778L mutation.
- Significant neurological conditions within the prior 12 months which may impact participant safety or participation in the study, including ability to complete study requirements or procedures as outlined in the clinical study protocol.
- In patients with psychiatric involvement, current or fluctuant clinical instability with new or changing diagnoses or substantial medication regimen changes in the past 12 months that could limit their participation, in the opinion of the Investigator.
- History of cirrhotic decompensation within the past year.
- Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the participant or would preclude the participant from successful study completion, including participant unable or unwilling to comply with the protocol requirements.
- Current participation in an investigational study for the treatment of WD.
- Prior or active malignancy or myeloproliferative disorder (excluding Stage 1 or lower, fully treated/excised malignant and pre-malignant disease of the skin, cervix or colon. Additionally, any other malignant and pre-malignant disease that the Investigator in consultation with the treating oncologist and study Medical Monitor deem has been fully treated/excised for > 5 years).
Where it is running
- University of California Los Angeles Medical Center — Los Angeles, California, United States
- University of California Davis Health — Sacramento, California, United States
- Yale New Haven Hospital — New Haven, Connecticut, United States
- Northwestern University — Chicago, Illinois, United States
- Massachusetts General Hospital — Boston, Massachusetts, United States
- University of Michigan Medicine — Ann Arbor, Michigan, United States
- American Research Corporation — San Antonio, Texas, United States
Full record on ClinicalTrials.gov
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