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

(GATEWAY Trial)

Not currently recruiting at 9 trial locations

Overseen By Sonia Valero

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Vivet Therapeutics SAS

Disqualifiers: Liver cirrhosis, Renal impairment, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 1 day

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new gene therapy called VTX-801 for individuals with Wilson's Disease, a rare condition where copper accumulates in the body, causing serious health issues. The main goal is to determine if a single dose of VTX-801 is safe and to assess its impact on the body's copper management over time, particularly after discontinuing other Wilson's Disease treatments. Suitable candidates have maintained a stable Wilson's Disease condition for at least a year and have been on recommended treatments without major issues. As a Phase 1/Phase 2 trial, this research aims to understand how VTX-801 functions in people and to measure its effectiveness in an initial, smaller group, offering participants a chance to be among the first to benefit from this innovative treatment.

Will I have to stop taking my current medications?

The trial involves withdrawing from your current Wilson's Disease therapy at some point, so you may need to stop taking those medications.

Is there any evidence suggesting that VTX-801 is likely to be safe for humans?

Research has shown that VTX-801, a gene therapy for Wilson's Disease, has been generally well-tolerated in studies so far. The therapy is administered as a one-time dose through an IV (a tube into a vein). Data from earlier studies indicated that VTX-801 is safe, with no major side effects reported. The main goal is to ensure the treatment remains safe over time, with patients monitored for up to 52 weeks after receiving the dose. It is important to note that this therapy is still under investigation, so more will be learned about its safety as additional participants receive it and more information is gathered. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for Wilson's Disease, which typically include medications like chelating agents and zinc therapy to manage copper levels, VTX-801 is a gene therapy that aims to address the root cause of the disease. Researchers are excited about VTX-801 because it works by delivering a healthy version of the ATP7B gene directly into the liver cells. This innovative approach has the potential to restore normal copper metabolism, offering a long-term solution rather than just managing symptoms. If successful, VTX-801 could significantly reduce the need for lifelong medication and improve the quality of life for patients.

What evidence suggests that VTX-801 might be an effective treatment for Wilson's Disease?

Research has shown that VTX-801, a type of gene therapy, could help treat Wilson's Disease. In animal studies, VTX-801 greatly reduced copper buildup in the liver, a major issue for those with Wilson's Disease, and improved the body's ability to manage copper. This is crucial because Wilson's Disease causes harmful copper accumulation. These early findings are promising and suggest that VTX-801 might effectively address the disease's main cause in humans. ^{2 3 6 7 8}

Are You a Good Fit for This Trial?

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 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.

I have been treated for Wilson's disease following international guidelines without signs of treatment failure.

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.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single ascending dose of VTX-801 intravenously

1 day

Follow-up

Participants are monitored for safety, tolerability, and pharmacological activity of VTX-801

5 years

Regular visits up to 1 year post-treatment

Long-term follow-up

Participants continue to be monitored for long-term safety and effectiveness

4 years

What Are the Treatments Tested in This Trial?

Interventions

• VTX-801

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.

How Is the Trial Designed?

Group I: VTX-801 Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Vivet Therapeutics SAS

Lead Sponsor

Trials

1

Recruited

4+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04537377

NCT04537377 | A Phase I/II Study of VTX-801 in Adult ... The objectives of this clinical trial are to assess, for up to 5 years, the safety, tolerability and pharmacological activity of a single ascending doses of VTX ...

2. withpower.com withpower.com/trial/phase-2-hepatolenticular-degeneration-7-2021-d3350

Gene Therapy (VTX-801) for Wilson's Disease Research in mice with Wilson's Disease shows that VTX-801, a gene therapy, significantly reduces copper buildup in the liver and improves copper metabolism, ...

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/26409215/

Long-term metabolic correction of Wilson's disease in a ... Our data demonstrate that AAV8-AAT-ATP7B-mediated gene therapy provides long-term correction of copper metabolism in a clinically relevant animal model of WD.

4. sciencedirect.com sciencedirect.com/science/article/pii/S2352320424000373

Research progress in stem cell therapy for Wilson disease This article discusses the research progress of stem cell therapy for WD from various sources, as well as the challenges and future prospects

5. vivet-therapeutics.com vivet-therapeutics.com/vivet-therapeutics-presents-interim-data-on-its-phase-1-2-gateway-trial-for-the-treatment-of-wilson-disease-at-easl-congress-2024/

Vivet Therapeutics presents interim data on its Phase 1/2 ... Its most advanced therapy is VTX-801, a novel gene therapy for Wilson's Disease, with key clinical read-outs expected by the end of 2024.

6. vivet-therapeutics.com vivet-therapeutics.com/vivet-therapeutics-doses-first-patient-in-cohort-2-in-phase-1-2-gateway-clinical-trial-for-treatment-of-wilson-disease/

Vivet Therapeutics Doses First Patient in Cohort 2 in Phase ... Its most advanced therapy is VTX-801, a novel gene therapy for Wilson's Disease, with key clinical read-outs expected by the end of 2024. Vivet ...

7. pfizer.com pfizer.com/news/press-release/press-release-detail/vtx-801-receives-us-fda-fast-track-designation-treatment

VTX-801 Receives U.S. FDA Fast Track Designation ... VTX-801 is a novel investigational gene therapy for Wilson Disease ... safety and tolerability of VTX-801 at 52 weeks after a single infusion.

8. wilsondisease.org wilsondisease.org/wp-content/uploads/2024/10/GTx-Updates-10-18-24.pdf

Update: Gene Therapies for Wilson Disease Ultragenyx Data gathered so far from the two doses that were tested shows that while the gene therapy was safe, it did not show a significant enough effect ...