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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
Must not have
Any history of diabetes
Positive QuantiFERON®-TB Gold tuberculosis test result
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at 1-year post treatment
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing VTX-801, a gene therapy, in adults with Wilson's Disease. The treatment involves adding new genes to help the body control copper levels. Researchers want to see if it is safe and effective over several years. VTX-801 is a mini version of the human ATP7B gene that has shown long-term correction of copper metabolism in animal studies.

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.
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.
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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have a history of diabetes.
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I tested positive for tuberculosis with the QuantiFERON test.
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I have never had HIV or HTLV.
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My kidney function is low or I have kidney disease.
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My ALT levels are high without an obvious external cause.
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I have had or currently have hepatitis B.

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

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

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

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

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Wilson's Disease is primarily treated by chelation therapy, which uses agents like penicillamine or trientine to bind and remove excess copper from the body, and zinc therapy, which blocks copper absorption in the intestines. However, gene therapy, such as the VTX-801 trial, introduces functional copies of the ATP7B gene to correct the underlying genetic defect responsible for impaired copper metabolism. This approach aims to restore normal copper processing and excretion, potentially offering a long-term solution and reducing the need for lifelong medication. For patients, this could mean improved liver function, reduced risk of liver damage, and overall better management of the disease.
A Gene Therapy Approach to Improve Copper Metabolism and Prevent Liver Damage in a Mouse Model of Wilson Disease.Long-term metabolic correction of Wilson's disease in a murine model by gene therapy.Insights gained from gene therapy in animal models of retGC1 deficiency.

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
~2 spots leftby Mar 2025