Recruiting Studies
Advanced SearchData: ClinicalTrials.gov · Source of record for eligibility and locations
Top Cities for Wilson Disease Clinical Trials
Wilson Disease clinical trials are recruiting across 8 cities. Here are the cities with the most active studies:
Wilson Disease Trial Phases
3 research organizations are sponsoring wilson disease trials.
About Wilson Disease
Wilson disease is a rare inherited disorder that causes copper to accumulate in the liver, brain, and other organs. It can cause liver disease, neurological problems, and psychiatric symptoms. Treatment involves copper-chelating agents and zinc to prevent copper absorption.
Clinical trials are advancing new treatments for wilson disease. Currently, 4 studies are recruiting a combined 410 participants across the United States. Research is being conducted by 3 organizations including Yale University, Prime Medicine, Inc., Orphalan.
2026 Wilson Disease Research Landscape
As of October 2026, the wilson disease clinical trial landscape includes 4 actively recruiting studies across 8 cities in the United States. These studies are collectively seeking 410 participants, with an average enrollment target of 103 per study.
The research spans multiple phases of development: 1 study is in PHASE1, 1 study is in PHASE2, 1 study is in PHASE3. The presence of Phase 3 trials indicates that some wilson disease treatments are in late-stage testing and may be approaching regulatory review.
Research is being led by 3 different organizations, including Yale University, Prime Medicine, Inc., Orphalan.
Geographically, wilson disease trials are most concentrated in New Haven, Connecticut (4 trials); Orlando, Florida (1 trial); Houston, Texas (1 trial); Seattle, Washington (1 trial); Chicago, Illinois (1 trial) and 3 other cities.
Featured Wilson Disease Studies
Highlighted recruiting studies for wilson disease, selected by enrollment size and research scope.
Natural History of Wilson Disease
The purpose of the registry/repository is to provide a mechanism to store data and specimens to support the conduct of future research about Wilson disease (WD). The overall aim is to determine the optimal testing for diagnosis and parameters for monitoring treatment of WD that will aid product utilization and development.
A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD)
The purpose of this study is to evaluate the safety, tolerability, biological activity, and initial efficacy of PM577a, an investigational Prime Editing therapy, in adults and adolescents with Wilson disease (WD). Wilson disease is caused by changes (mutations) in the ATP7B gene that prevent the body from removing excess copper normally. PM577a is designed to precisely correct one of the most com...
Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.
The goal of this clinical trial is to learn if a new trientine tetrahydrochloride (TETA 4HCl) formulation administered once a day compared to d-Penicillamine (DPA) as a first line treatment for people living with Wilson's disease (WD) is effective and safe. The study is enrolling children aged 8 years and older weighing at least 55 lb (25 kg) and adults with a recent diagnosis of WD. People recent...
Frequently Asked Questions About Wilson Disease Clinical Trials
Are there wilson disease clinical trials near me?
Yes, there are 4 wilson disease clinical trials currently recruiting across 8+ cities in the United States, including New Haven, Connecticut; Orlando, Florida; Houston, Texas. Browse the studies above to find one at a location convenient for you.
How do I join a wilson disease clinical trial?
To join a wilson disease clinical trial: 1) Browse the available studies on this page, 2) Click on a study that interests you, 3) Check the study locations to find a site near you, 4) Review the eligibility criteria, and 5) Contact the study site or complete the eligibility form. The process is free and you can withdraw at any time.
Are wilson disease clinical trials free?
Yes, participation in wilson disease clinical trials is free. Study-related treatments, medical tests, and doctor visits are provided at no cost to participants. Many studies also offer compensation for your time and travel expenses.
What types of wilson disease treatments are being studied?
Current wilson disease clinical trials are testing a range of approaches across PHASE1 (1 study), PHASE2 (1 study), PHASE3 (1 study). These include new drugs, combination therapies, medical devices, and other interventions sponsored by 3 research organizations.
Is it safe to participate in wilson disease clinical trials?
Clinical trials are carefully regulated by the FDA and institutional review boards (IRBs). All trials must follow strict safety protocols, and participants receive close medical monitoring throughout the study. You can withdraw from a trial at any time without penalty.
Data updated October 15, 2026 from ClinicalTrials.gov
About This Data
Data: ClinicalTrials.gov · Source of record for eligibility and locations
Clinical trial information on this page is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health (NIH) and National Library of Medicine (NLM). Study data is refreshed every hour to ensure accuracy.
Medical Disclaimer: The information provided on this page is for informational purposes only and does not constitute medical advice. Always consult with a qualified healthcare provider before making decisions about clinical trial participation or changes to your treatment plan.
Page reviewed by the HelloStudys Research Team · Last updated October 15, 2026 · Data from ClinicalTrials.gov