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

(GATEWAY Trial)

Recruiting at 9 trial locations

Overseen By Sonia Valero

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Vivet Therapeutics SAS

No Placebo Group

Trial Summary

What is the purpose of this trial?

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.

Eligibility Criteria

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.

Inclusion Criteria

I am between 18 and 65 years old.

I have been diagnosed with Wilson's disease.

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

Pregnancy or breastfeeding

I have not had signs of worsening liver cirrhosis or bleeding in the stomach or intestines in the last 6 months.

I don't have any health conditions or treatments that could affect this study.

Treatment Details

Interventions

• VTX-801 (Gene Therapy)

Trial Overview 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.

Participant Groups

Group I: VTX-801 Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Vivet Therapeutics SAS

Lead Sponsor

Trials

1

Recruited

4+