Clinical Studies and Trials
Medicine is advanced through human volunteers. Some studies test new therapies and others gather information.
Ultragenyx’s Wilson Disease Gene Therapy Program Update
Ultragenyx is pleased to announce that the first patient has been dosed in the CYPRUS2+ study, a one-time intravenous (IV) infusion of UX701, an investigational gene therapy for the treatment of Wilson disease. Ultragenyx is sponsoring this global study to determine if UX701 gene therapy is a safe and effective treatment for adults with Wilson disease. This adeno-associated virus (AAV) gene therapy has the potential to restore ATP7B function, which may improve copper metabolism and distribution.
GATEWAY IS NOW ACTIVELY ENROLLING AT 3 US CLINICAL SITES!
#GATEWAY is a Vivet Therapeutics clinical trial for #WilsonDisease, taking place at several clinical sites across the United States and Europe.
Learn more about this currently recruiting clinical trial and our #Gene Therapy, VTX-801:
Wilson Disease Patient Registry Study
The Wilson Disease Association (WDA) is not aware of any other multicenter, multinational Wilson Disease registry with prospective data collection in the world. The results of this research will benefit current and future WD patients globally.
Learn more about this study:
The Wilson Disease copper balance study
The aim of this clinical study in people with Wilson Disease is to find out whether Alexion’s investigational product is able to help restore copper balance by promoting the removal of more copper from the body than is taken in through food and drink. This clinical study is currently recruiting, and if you would like to learn more, please contact Celerion Lincoln, Nebraska, at email: WilsonStudyInfo@celerion.com or call 866-445-7033. This clinical study is sponsored by Alexion Pharmaceuticals, Inc.
Ultragenyx Research Opportunities for Patients
Multiple Ways to Participate in Wilson Disease Research Studies
Who We Are
Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company working to develop new products for the treatment of rare and ultra-rare diseases. We are committed to both developing new treatments and helping advance the understanding of rare diseases.
How You Can Help
We are currently developing an investigational gene therapy (UX701) for Wilson disease. We are committed to partnering with patients, caregivers, and family members.
There are multiple ways you can help. Please consider whether one or more studies below may be a good fit for you or your loved one. All studies below are institutional review board (IRB)-approved.
If you are interested in learning more about how to get involved, please email PatientAdvocacy@Ultragenyx.com.
Alexion Clinical Trial
Alexion is now recruiting for copper and Molybdenum Balance in Participants with Wilson disease treated with ALXN 1840.
Click here for more information, or click here for a brochure about the trial.
WILSON DISEASE ASSOCIATION
Contact Us
- Toll Free: 866-961-0533
- Phone: 414-961-0533
Email: info@wilsonsdisease.org