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Gene Therapy

Gene Therapy (VTX-801) for Wilson's Disease (GATEWAY Trial)

Phase 1 & 2
Recruiting
Research Sponsored by Vivet Therapeutics SAS
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Male or female aged 18 and 65 years inclusive
Confirmed diagnosis of WD
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at 1-year post treatment
Awards & highlights

GATEWAY Trial Summary

This trial will test if VTX-801, a gene therapy, is safe and effective in treating Wilson's Disease.

Who is the study for?
Adults aged 18-65 with confirmed Wilson's Disease, who have been stable on treatment for at least a year. They must not have severe kidney issues, signs of liver failure, certain blood abnormalities, or active infections like HIV or hepatitis. Pregnant or breastfeeding individuals and those with a BMI ≥ 35 kg/m2 cannot participate.Check my eligibility
What is being tested?
The trial is testing VTX-801, a gene therapy given through an IV to adults with Wilson's Disease. It will be studied over five years to see how safe it is and how well it works when patients stop their usual treatments.See study design
What are the potential side effects?
Potential side effects are not explicitly listed but may include reactions related to the infusion process, changes in liver and kidney function tests, and other unforeseen complications due to the nature of gene therapy.

GATEWAY Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am between 18 and 65 years old.
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I have been diagnosed with Wilson's disease.
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My Wilson's disease has been stable for at least a year, with no major changes in my neurological state or mood, and stable copper levels.
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I have been treated for Wilson's disease following international guidelines without signs of treatment failure.

GATEWAY Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at 1-year post treatment
This trial's timeline: 3 weeks for screening, Varies for treatment, and at 1-year post treatment for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Safety and tolerability profile (including treatment-emergent adverse events (TEAE))
Secondary outcome measures
24-hour urinary Cu
Free serum Cu
Serum ceruloplasmin activity (enzymatic assay)
+2 more

GATEWAY Trial Design

1Treatment groups
Experimental Treatment
Group I: VTX-801Experimental Treatment1 Intervention

Find a Location

Who is running the clinical trial?

Vivet Therapeutics SASLead Sponsor

Media Library

Wilson's Disease Clinical Trial 2023: VTX-801 Highlights & Side Effects. Trial Name: NCT04537377 — Phase 1 & 2
VTX-801 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04537377 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any vacancies available for individuals to participate in this clinical trial?

"As reported on clinicaltrials.gov, this medical study is still in the process of recruiting participants. The trial was first launched on September 3rd 2021 and underwent a revision as recently as November 4th 2022."

Answered by AI

Does this trial welcome elderly individuals aged 85 or older?

"This clinical trial is interested in participants aged 18 and up, but no older than 65."

Answered by AI

Are there any particular demographics that are more conducive to taking part in this medical study?

"This trial is recruiting 16 adults aged 18-65 with a documented diagnosis of Wilson's Disease who have been receiving adequate treatment for over a year. In addition to age, gender and illness criteria, participants must present no noteworthy changes in their neurological or psychological status since being diagnosed as well as stable laboratory results pertaining to copper metabolism."

Answered by AI

Are there numerous locations in Canada executing this research project?

"The clinical trial is being conducted at University of Texas Southwestern Medical Center in Dallas, Yale University School of Medecine in New Haven, and the University of Michigan Health System in Ann Arbor. Additionally, there are 5 other sites providing access to this medication."

Answered by AI

How many test subjects are enrolled in this clinical experiment?

"Affirmative. The clinical trial, which was first listed on September 3rd 2021, is actively bringing in participants and can be found on the website of clinicaltrials.gov. 16 individuals need to be registered from 5 separate sites for this experiment to begin."

Answered by AI
~4 spots leftby Mar 2025