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Programs and Research
Studies and Advocacy Initiatives

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.

Ultragenyx research opportunities for patients

May 19, 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.

Phase 1/2 Gene therapy study of VTX-801 in Adult Patients with Wilson Disease

Opening for enrollment: A new Phase 1/2, multi-center, non-randomized, open-label study to evaluate the safety, tolerability and pharmacological activity of a single dose VTX-801. This study is sponsored by Vivet Therapeutics.

The investigational product (VTX-801) is a gene therapy for the treatment of Wilson Disease. Gene therapy for Wilson Disease involves expression of a functional version of ATP7B (the protein that is not properly functional or is absent in patients with Wilson Disease) in a patient’s liver cells. This is accomplished using a viral vector that carries the coding sequence for this protein (the VTX-801 investigational product) and transports it through the bloodstream to the liver cells. The viral vector is designed to not reproduce itself inside a patients’ liver. The aim of this study is to determine whether VTX-801 is safe and to find the best dose to use for correction of the metabolic defect in copper metabolism in Wilson disease. VTX-801 has been tested in animals and primates, but this study is the first time VTX-801 is being administered to humans.

The study is being conducted at several sites across the US, currently including Yale University School of Medicine, the University of Michigan Health System and the UC Davis Medical Center, and in Europe. A list of sites and contact information can be found here: https://clinicaltrials.gov/ct2/show/NCT04537377

If you have Wilson Disease and your disease has been stable for the last year on medical treatment, you may be eligible. (Screening tests will be performed to further determine eligibility).

Study participation is for 5 years. The first year of the study requires 3 inpatient (hospital) stays and 34 visits (24 of which can be done at your home). Year2 requires 4 visits, Years 3-5 two visits per year. Travel expenses and study related testing will be covered by the study.

For further information about this study, please go to https://clinicaltrials.gov/ct2/show/NCT04537377 for up to date contact information and a list of actively enrolling sites.


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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