Wilson Disease is a rare genetic disorder that causes excess copper to build up in the body, particularly in the liver and brain. The disease is caused by mutations in a gene called ATP7B.
If Wilson Disease is not diagnosed early and appropriately treated, the build-up of copper may cause serious damage to organs and result in severe disability and possibly death. This means that early diagnosis and prompt treatment is very important.
Although there are currently available medical treatments for Wilson Disease, patients need to take them for life and the therapeutic response is not optimal in some patients; therefore new therapeutic options are desirable.
VTX-801 is a new gene therapy treatment for Wilson Disease that is currently under investigation in GATEWAY. The GATEWAY clinical trial will explore safety, efficacy and durability of the effects of this treatment.
Clinical Trial Information
What is the GATEWAY clinical trial?
The name ‘GaTeWay’ stands for Gene Therapy for Wilson Disease.
GATEWAY is a gene therapy clinical trial (also known as a “clinical research study”) conducted in patients with Wilson Disease; it attempts to assess if the study drug called VTX-801 is safe and which is the most appropriate dose that may restore durably the elimination of excess copper through the natural route.
Who is conducting this clinical trial?
The clinical trial is sponsored by Vivet Therapeutics, a biotechnology company based in France that is developing gene therapies for rare liver disorders. Vivet is collaborating with Pfizer on the clinical supply of VTX-801, Vivet’s proprietary treatment for Wilson disease, for this Phase 1/2 clinical trial.
What should trial participants expect?
Patients will receive a single dose of VTX-801, which will occur in a hospital setting. Patients will then come to their study center at planned times over the following year so that the study team can assess how they are doing. These visits will occur more often in the early part of the trial (first year) and the number of visits will decrease thereafter . The study will continue for 4 years after the initial 1-year trial period, which will be a critical part of understanding how well VTX-801 works overtime.
What is the treatment VTX-801?
VTX-801, the gene therapy studied in this trial, contains a corrective version of the ATP7B gene, which is malfunctioning in patients affected with Wilson Disease.
The corrective version of the gene is packaged within a vector, which is a modified virus called adeno-associated virus (AAV) that has been engineered to deliver the gene into liver cells without causing any infection. The treatment is administered as a single intravenous infusion. The clinical supply of VTX-801 is manufactured by Pfizer at a U.S. production facility on behalf of Vivet Therapeutics.
View the video to learn more on how gene therapy aims to target the cause of Wilson Disease (Educational material from American Society of Gene and Cell Therapy)
Check Eligibility
If you have answered “yes” to three questions, you may be eligible to participate to GATEWAY.
If you are interested in participating, please contact the nearest participating clinical site (see maps and contact details) to check for the other enrollment criteria.
The trial will take place at several clinical sites across the United States, UK, Germany and Denmark, as indicated below. Map and site details will be updated upon receipt of local regulatory approvals to begin the trial at each new site.
Contact a US site
University of California Davis
Dr. Valentina Medici
2000 Stockton Blvd
Sacramento, CA 95817
Sandeep Dhaliwal
(916) 734‐8696
University of Michigan Health System
Dr. Frederick Askari
1500 East Medical Center Dr. UH South F5161
Ann Arbor, MI 48109
Ashley Page
(734) 998-9966
Yale University School of Medicine
Dr. Michael Schilsky
310 Cedar Street
FMB 18
New Haven, CT 06520
Daksshi Hettiarachchi
(203) 737-5037
daksshi.hettiarachchi@yale.edu
Advent Health
Dr. Regino Gonzalez-Peralta
615 East Princeton Street
Orlando, Florida
Pamela Hedrick
(407) 303-9826
Pamela.Hedrick@AdventHealth.com
University of Texas Southwestern Medical Center
Dr. William Lee
5929 Harry Hines Boulevard
POB 1
Dallas, Texas
Amy Pugh
(214) 648-4412
Wake Forest School of Medicine
Dr. Sean Rudnick
Medical Center Boulevard Winston-Salem, NC 27157
Dee Faust
+1 336.713.1442
Contact a European site
Aarhus Universitetshospital
Dr Thomas Damgaard Sandahl
Palle Juul-Jensens Boulevard 99
8200 Aarhus
Danmark
+45 5129 3791
Royal Surrey County Hospital
Pr. Aftab Ala
Egerton Road
Guilford,
Surrey GU2 7XX
United Kingdom
Pr. Aftab Ala
(+44) 1483 571 122
Universitätsklinikum Tübingen (UKT)
Pr. Ulrich Lauer
Otfried-Müller-Str. 10
72076 Tübingen
Deutschland
Prof. Ulrich Lauer
+49 7071-2983190
ulrich.lauer@med.uni-tuebingen.de
University Hospital Essen
Hufelandstrasse 55
45147 Essen
Deutschland
Prof. Hartmut Schmidt
+49 201 5990015
Frequency Asked Questions
Travel expenses and study-related testing may be covered by the Sponsor.
Yes, there is no placebo arm in this study so all participants will receive the VTX-801 experimental gene therapy.
Our gene therapy approach involves introducing a corrective gene into the liver cells in order to restore proper liver function and remove excess copper.
For a gene to enter a cell, it needs to be packaged into a delivery vehicle, otherwise known as a ‘vector’.
Viruses work well as vectors because they can easily enter our cells. Scientists can engineer appropriate versions of viruses into vectors for this purpose. These are harmless and won’t cause disease when used in people. To do this, scientists replace the DNA that is naturally present in the virus with the gene that will be used to treat the disease.
To learn more about gene therapy for Wilson Disease, see educational material from American Society of Gene & Cell Therapy.
A majority of the study visits are planned during the first year, and the total duration of the study is 5 years. During this first year, some of the visits to collect blood and urine samples, are planned at your home instead of the hospital, to make it easier for you.
We are looking for approximately 16 patients to join GATEWAY.
The GATEWAY study protocol, prepared by the Sponsor and agreed with Wilson Disease experts and competent regulatory authorities (including FDA), specifies the enrolment criteria. Based upon these criteria, the study enrolment committee, composed of investigators and co-investigators, then decide who may participate in the study.
For this first clinical trial, only adults with stable Wilson Disease may participate; however, when sufficient data has been collected in GATEWAY, other trial(s) may be organized to study other patient populations.
Currently, as VTX-801 is an investigational treatment, it is available exclusively for patients enrolled in GATEWAY.
VTX-801 will not be available outside of the context of clinical trials until the development has been completed and competent authorities have decided if it may be approved for commercialization. The whole process typically takes a few years from the start of a first clinical trial.
The clinical supply of VTX-801 is manufactured by Pfizer at a U.S. production facility on behalf of Vivet Therapeutics.
Contact: info@vivet-therapeutics.com
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