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Studies and Advocacy Initiatives

#GATEWAY is a Vivet Therapeutics clinical trial for #WilsonDisease

#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:

www.gatewaytrialwilsondisease.com

Ultragenyx’s Wilson Disease Gene Therapy Program is NOW ENROLLING!

August, 2021
CYPRUS2+ is a randomized, double blind, placebo controlled, seamless Phase 1/2/3 study to assess safety, tolerability, and efficacy of a one-time intravenous (IV) infusion of UX701, an investigational AAV-Mediated Gene Therapy, for the treatment of Wilson Disease. Enrollment will include adults living with Wilson Disease currently being well managed on standard of care. Click here for more information.

The Wilson Disease copper balance study

August, 2021
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: This email address is being protected from spambots. You need JavaScript enabled to view it. or call 866-445-7033. This clinical study is sponsored by Alexion Pharmaceuticals, Inc.

Ultragenyx research opportunities for patients

August, 2021
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. Click here for more information.

Alexion Clinical Trial

June 22, 2021
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 on this trial.


Ricarda Tomlin, BS, CCRP
Manager, Clinical Research
Transplant Surgery, Yale School of Medicine

If you are interested in participating in a clinical trial, you have several options: (1) talk to your doctor or contact a Wilson Disease specialist near you, (2) network with other patients and the patient association, (3) search the internet.

The first two options are straightforward, but the third should be approached with some caution. As with all internet searches, it is important to make sure information comes from a reliable source. Here are some details about the most reliable and comprehensive places to find a list of clinical trials:

In 1997, the US Congress enacted a law requiring the creation of a public database for all clinical trials. This database, www.ClinicalTrials.gov, is intended to give patients, the general public, health care providers and researchers insight into what research is being done and access to results. A similar database exists in Europe (EU-CTR, www.clinicaltrialsregister.eu) and the World Health Organization launched a search portal (www.who.int/ictrp) that combines information from 17 different registries on one platform.

If you are interested in learning more, you can go to any of these websites and type “Wilson Disease” into the search box to generate a comprehensive list of clinical trials. There are filters to limit your search for trials open to recruitment (i.e., trials that currently accept new enrollees) or by drug name (if you are looking for a specific trial).

Once you are in the record for a specific study, you will find an outline of the study, planned outcome measures, information on who is eligible to participate, and a list of locations currently participating in the study with contact information. Participation in research is always voluntary and you should take your time to find out enough information and get your questions answered before you sign up for a study.

Clinical research works best when it is done in a transparent manner, when all interested patients have access to research participation, and when all outcomes, not just positive findings, are made public making these non-commercial public databases a valuable tool to patients and researchers alike.

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