Clinical Studies, Trials and Surveys

Medicine is advanced through human volunteers. Some studies test new therapies and others gather information.

The Wilson Disease Association posts information here about clinical trials, studies and surveys that you can participate in. Please use this information to decide what is best for you.

Posts are screened for legitimacy.

Please remember, when you share your personal information or opinions in surveys, they benefit somebody and should be taken seriously. Use caution if asked to compete surveys by unknown parties that solicited you.

Posts are here to advance research in the treatment of Wilson disease. Many patients are interested in the same. The identities of participants posted here are kept anonymous from pharmaceutical companies.

Please review our terms of use.

Wilson Disease Patient Registry Study

The Registry is a Wilson Disease Association-sponsored study.

Ultragenyx Wilson Disease Gene Therapy Trial Update for WDA

November 13, 2023|0 Comments

As requested, we are providing an update on Stage 1 of our Cyprus2+ study, assessing safety and efficacy of UX701, an investigational gene therapy for the treatment of Wilson disease.
The status update below is based on the first dosing group of 5 patients as of October 8, 2023:

  • Four out of 5 patients have had reductions in urinary copper and are tapering off of chelators and/or zinc therapy, including 2 of 3 earlier treated patients in the group that are now completely off standard therapy
  • Duration of time on the study for this group ranged from 16 to 82 weeks
  • Tapering of chelators and/or zinc therapy began at 12 weeks following a single intravenous infusion of UX701
  • While none of the patients have experienced treatment-related adverse events, we do not have sufficient data to reach any conclusions regarding the safety of UX701 or whether adverse events will occur as the study continues.

Additional information:

  • The study is expected to complete dosing of all 15 patients in Stage 1 at the end of 2023
  • Additional information on safety and efficacy is expected to be shared in the first half of 2024
  • Ultragenyx is sponsoring this global study of UX701, an investigational gene therapy for which safety and effectiveness has not been established. This type of research takes time, so it will be a while before we know whether and when UX701 will receive regulatory approval.

Ultragenyx would like to express our gratitude to those individuals who are participating in our study. These experiences and insights help advance our understanding of Wilson disease and have the potential to move research forward for future generations. As requested, we will continue to share updates as new information becomes available. For questions, please email PatientAdvocacy@ultragenyx.com.

Vivet Therapeutics’ Wilson Disease Gene Therapy Clinical Trial Update

September 20, 2023|0 Comments

On behalf of the Vivet Therapeutics Wilson disease study team, we are happy to share updates about the ongoing and enrolling GATEWAY clinical trial.

GATEWAY is an international gene therapy clinical trial for people living with Wilson disease assessing the safety, efficacy and durability (how long-lasting) of study medication VTX-801.

What is VTX-801? In people living with Wilson disease, the ATP7B gene – normally active in the liver – is malfunctioning. VTX-801 is an investigational gene therapy containing a corrective version of the ATP7B gene; this gene is packaged within a vector, which is the empty (non-infectious) shell of a virus engineered to deliver the corrective gene into liver cells via the blood. The treatment is administered as a single intravenous infusion.

Where is this study taking place? There are now ten participating sites open across the United States and Europe: 6 sites in the U.S. (California, Connecticut, Florida, Michigan, Texas and North Carolina) and 4 sites in Europe (Denmark, Germany and the UK). For eligible patients, assistance with travel and study-related expenses will be available.

Who is sponsoring the study? Vivet Therapeutics is sponsoring the GATEWAY clinical trial. Vivet is dedicated to the development of innovative therapies for people affected by rare liver disorders such as Wilson disease.

Visit www.gatewaytrialwilsondisease.com/ for more information about eligibility and clinical trial sites.

Ultragenyx’s Wilson Disease Gene Therapy Program Update

January 17, 2023|0 Comments

On behalf of the Ultragenyx Wilson disease study team, we would like to share a recent update on our CYPRUS2+ study.

Ultragenyx has changed Stage 1 of our CYPRUS2+ study to an open-label, single arm design. This means that all patients enrolled in the study will receive UX701, an investigational gene therapy for the treatment of Wilson disease. Stage 1 of the study no longer utilizes placebo, which will allow us to better understand the biological response to UX701 in real-time, as well as optimize the safe reduction of current treatment for WD.

The changes to the CYPRUS2+ protocol for Stage 1 may take place at different times depending on site locations. For sites located outside of the U.S., they must await approval by their country’s regulatory agency. Whether or when our investigational gene therapy will be approved by regulatory agencies as a treatment for Wilson disease is not known. The results from additional studies are needed to decide whether this treatment is safe and effective.

You are invited to email PatientAdvocacy@Ultragenyx.com for more information or read the full update located at: https://clinicaltrials.gov/ct2/show/NCT04884815?term=ux701&draw=2&rank=1.

GATEWAY IS NOW ACTIVELY ENROLLING AT 3 US CLINICAL SITES!

September 23, 2022|0 Comments

It now has a dedicated website for the patient community and also a dedicated call center for US patients with a patient liaison you can speak with. website address is: https://www.gatewaytrialwilsondisease.com/

#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

February 2022 Update

Donations

Your support allows the WDA to maintain this website, produce educational materials, support research, and hold meetings for people living with WD, their families, and the healthcare community.

Membership

Your support allows the WDA to maintain this website, produce educational materials, support research, and hold meetings for people living with WD, their families, and the healthcare community.

Peer to Peer Fundraiser

Starting a fundraiser is easy to do, and it’s fun. We’ll even give you all the tools you’ll need to make a difference. With your help we can ensure critical steps of our 5 year plan get completed. This fundraiser requires no parties, walks or selling anything. You just send it out to people that you know. Contact Judi Keller at judi.keller@wilsonsdisease.org if you require further assistance with setting this up.

We Are Wilsons

Share Your Story
We will be collecting patient stories and photographs to post on our website, Facebook and soon, Instagram. The Wilson Warrior stories have proven to be inspirational and we think we need a way to collect any story.

Final Gift Plan

Consider WDA in a “Will” or Final Gift Plan
The Wilson Disease Association is 35 years old and is represented by board members from across the country and Canada. We have volunteers and partners globally and spearhead research that impacts our patients directly but affects a broader population beyond our patients. The long term importance of what the Wilson Disease Association does is limited only to the imagination because it affects generations of families around the world. Please consider the Wilson Disease Association in your will. Giving in this way is not restricted to the wealthy or a gender.

Corporate Matches

Let us help you find employers and corporate donors to match your donation.
As a benefit to employees, many companies increase the impact of their employees’ gifts to WDA by providing matching contributions. Most corporations provide a 1:1 match; while some companies will double or even triple the matching amount to further encourage and leverage their employees’ philanthropy.

Facebook Picture Post

Share a picture of yourself on Facebook
Help us spread the word by sharing a picture of yourself holding a sign with a message that we can post on Facebook and Instagram.

Facebook Fundraiser

Start your own fundraiser on Facebook
We’ll help you get started and give you tips to reach your goal.

Shop at AmazonSmile

You Shop. Amazon Gives.
Shop Online and Help the WDA. When you shop online at www.igive.com, designate the Wilson’s Disease Association as your charity of choice and a percentage of each sale (up to 12.5%) will be donated. Retailers include CD Now, J Crew, Avon, and many others. Log on for more information!

WILSON DISEASE ASSOCIATION

Contact Us