Active not recruitingPHASE1, PHASE2INTERVENTIONAL

A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease

This research study is looking at a new treatment called VTX-801 for adults with Wilson's Disease. VTX-801 is a type of gene therapy, which means it aims to address the root cause of the disease. The study will last up to five years and involves giving the treatment directly into a vein. Researchers will carefully check the safety of VTX-801, how well people tolerate it, and what effects it has on the body. A key part of the study is seeing if patients can gradually reduce their usual Wilson's Disease medications after receiving VTX-801. This is a "Phase I/II" study, meaning it's an early step to understand the treatment better in a small group of people.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Vivet Therapeutics SAS

Enrolment target

Start

03 Sep 2021

Estimated completion

18 Jun 2029

Wilson's Disease

What is this study about?

Wilson's Disease is a rare genetic condition where too much copper builds up in the body, primarily in the liver and brain. If left untreated, this can cause serious health problems. Current treatments focus on removing excess copper and preventing its build-up, and people usually need to take medication for life.

This study, called a "Phase I/II" study, is investigating a new treatment called VTX-801. VTX-801 is a "gene therapy," which is a type of medicine that tries to correct the problem by introducing a healthy copy of the gene that is faulty in people with Wilson's Disease. The aim is to help the body process copper properly again. This means it could potentially be a long-lasting treatment.

The main goals of this study are to find out if VTX-801 is safe for adults with Wilson's Disease and how well their bodies handle it. Researchers also want to see what effects the treatment has on the body and whether patients might eventually be able to reduce or even stop their regular Wilson's Disease medication. The study will follow participants for up to five years to carefully observe these long-term effects.

Key takeaways

Tests a new gene therapy (VTX-801) for Wilson's Disease.
Aims to check safety, tolerability, and effectiveness.
Could potentially reduce the need for current medications.
Involves treatments given into a vein.
Requires commitment for up to five years of monitoring.
Only for adults aged 18-65 with stable Wilson's Disease.

Who may be eligible?

To join this study, you would need to be an adult between 18 and 65 years old with an official diagnosis of Wilson's Disease. You should also be currently receiving treatment for your Wilson's Disease according to standard medical advice and your condition needs to have been stable for at least one year. This means your symptoms haven't changed much, and tests that check copper levels are steady.

There are also some reasons why you might not be able to join. For example, if you have certain liver problems, such as signs of serious liver damage or abnormal results in some blood tests (like those for liver function or blood clotting), you wouldn't be eligible. The study also cannot include individuals with significant kidney problems, or certain infections like HIV or hepatitis. People with a history of diabetes or high body mass index (BMI) over 35 kg/m2 would also be unable to participate.

If you are pregnant or breastfeeding, or if you have any other serious health conditions or medications that might interfere with the study, you would not be able to take part. The research team will carefully review all your medical information to make sure joining the study is safe for you.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you between 18 and 65 years old?
Have you been officially diagnosed with Wilson's Disease?
Have you been receiving standard treatment for your Wilson's Disease, and has your condition been stable for at least one year?
Do you have good overall liver and kidney function according to your doctor?
Are you free from certain infections like HIV or hepatitis?
Are you not pregnant or breastfeeding?

Answer every question to see your result.

What does participation involve?

If you join this study, you would receive the study medicine, VTX-801, which is given through a drip into your vein (intravenous infusion). The study plans to test different doses of VTX-801. After receiving the treatment, researchers will closely monitor you to see how your body reacts and if your current Wilson's Disease treatment can be gradually reduced or even stopped.

Your participation would involve regular visits to the study clinic for up to five years. During these visits, the research team would conduct various assessments. These might include physical examinations, blood and urine tests, and other procedures to check your health, how well the treatment is working, and to monitor for any side effects. These assessments help the doctors understand the safety and effects of VTX-801 over time. The exact number and frequency of visits would be detailed by the study team.

Potential risks and benefits

Participating in a clinical trial like this could potentially offer benefits, such as access to a new treatment that might improve your condition or reduce your need for lifelong medication. However, as VTX-801 is an experimental treatment, there are also potential risks involved, and side effects are possible, some of which might not yet be known. The research team will explain all known risks to you. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (10)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

UC Davis Medical Center
Verified postcode
Sacramento, United States
Yale University School of Medecine
Verified postcode
New Haven, United States
Advent Health
Verified postcode
Orlando, United States
University of Michigan Health System
Verified postcode
Ann Arbor, United States
Wake Forest School of Medicine
Verified postcode
Winston-Salem, United States
University of Texas Southwestern Medical Center
Verified postcode
Dallas, United States
Aarhus University Hospital
Verified postcode
Aarhus, Denmark
University Hospital Essen
Verified postcode
Essen, Germany
Universitätsklinikum Tübingen (UKT)
Verified postcode
Tübingen, Germany
Royal Surrey County Hospital
Verified postcode
Guildford, United Kingdom

Common questions

What is Wilson's Disease?

Wilson's Disease is a genetic condition where your body collects too much copper, which can harm organs like your liver and brain.

What is a 'gene therapy'?

Gene therapy is a new type of treatment that tries to fix the root cause of a disease by adding or changing genes in your body.

What does 'Phase I/II' study mean?

This means it's an early-stage study designed to first check the safety of a new treatment and then see if it has a positive effect in a small group of people.

How long would I be in the study?

You would be part of the study for up to five years, involving regular check-ups and monitoring.

Will I still take my regular Wilson's Disease medicine?

The study aims to see if you can gradually reduce or stop your current Wilson's Disease medication after receiving the study treatment.