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

(GATEWAY Trial)

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

4 Participants Needed

What You Need to Know Before You Apply

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.

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.

How does the gene therapy treatment VTX-801 for Wilson's Disease differ from other treatments?

VTX-801 is a gene therapy that uses a virus to deliver a smaller version of the ATP7B gene to correct copper metabolism in Wilson's Disease, unlike current treatments that rely on lifelong use of copper-chelating drugs and zinc salts, which do not restore normal copper metabolism. ^{1 2 3 4 5}

What data supports the effectiveness of the treatment 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, suggesting it could help manage the disease in humans. ^{1 4 5 6 7}

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. United Arab Emirates pubmed.ncbi.nlm.nih.gov

Perspectives for gene therapy of Wilson disease. [2019]

2. United States pubmed.ncbi.nlm.nih.gov

A Gene Therapy Approach to Improve Copper Metabolism and Prevent Liver Damage in a Mouse Model of Wilson Disease. [2020]

3. Netherlands pubmed.ncbi.nlm.nih.gov

Long-term metabolic correction of Wilson's disease in a murine model by gene therapy. [2019]

4. United States pubmed.ncbi.nlm.nih.gov

High value of 64Cu as a tool to evaluate the restoration of physiological copper excretion after gene therapy in Wilson's disease. [2022]

5. Netherlands pubmed.ncbi.nlm.nih.gov

Bis-choline tetrathiomolybdate in patients with Wilson's disease: an open-label, multicentre, phase 2 study. [2018]

6. United States pubmed.ncbi.nlm.nih.gov

Long-Term Correction of Copper Metabolism in Wilson's Disease Mice with AAV8 Vector Delivering Truncated ATP7B. [2020]

7. England pubmed.ncbi.nlm.nih.gov

WTX101 - an investigational drug for the treatment of Wilson disease. [2018]