Not recruitingRare disease

Study on Gene Therapy with VTX-801 and Copper (64Cu) Chloride for Adults with Wilson's Disease

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying Wilson's Disease, a rare genetic disorder that affects how the body processes copper. The study involves a new treatment called VTX-801, which is a type of gene therapy. Gene therapy is a method that uses genes to treat or prevent diseases. In this case, VTX-801 is designed to help the body manage copper levels better by using a special viral vector to deliver a shortened form of the human gene ATP7B, which is important for copper regulation.

The purpose of the study is to assess the safety and tolerability of VTX-801 when given to adult patients with Wilson's Disease. The study will involve a single dose of the treatment, administered through an intravenous infusion, which means it is given directly into a vein. Participants will be monitored over a period of five years to observe how they respond to the treatment and to ensure it is safe. The study will also involve the use of 64Cu, a form of copper, to help track how the body processes copper during the trial.

Throughout the study, various health checks will be conducted, including clinical examinations and tests like ECG (a test that records the electrical activity of the heart) and MRI (a type of scan that uses magnetic fields to create detailed images of the body). These checks will help researchers understand the effects of the treatment on the participants' health. The study aims to provide valuable information that could lead to better treatment options for people with Wilson's Disease in the future.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment
The trial begins with an initial assessment to confirm eligibility. This includes verifying age between 18 and 65 years, a confirmed diagnosis of Wilson's Disease, and stable condition for at least one year.
Laboratory tests are conducted to assess copper metabolism, including 24-hour urinary copper and free serum copper levels, as well as liver enzymes, hemoglobin, and white blood cell count.
Step 2
Treatment administration
The treatment involves a single dose of VTX-801, administered intravenously. This is a gene therapy designed to address Wilson's Disease.
The solution for infusion is given through a vein, allowing the treatment to enter the bloodstream directly.
Step 3
Monitoring and follow-up
Following the administration of VTX-801, monitoring occurs to assess safety and tolerability. This includes checking for any treatment-emergent adverse events, conducting clinical examinations, and reviewing changes in laboratory parameters.
Vital signs, ECG, and MRI scans of the brain and abdomen are performed to ensure the patient's well-being.
Step 4
Long-term follow-up
The study includes a 5-year follow-up period to evaluate the long-term effects of the treatment.
During this time, assessments of free serum copper, total serum copper, 24-hour urinary copper, and serum ceruloplasmin activity are conducted. The patient's response to VTX-801 and any immune response to the treatment are also monitored.

Who can join the trial?

6 criteria

Must be a male or female between 18 and 65 years old.
Must have a confirmed diagnosis of Wilson's Disease, a condition that affects how the body handles copper.
Must have been treated for Wilson's Disease according to international guidelines, with no signs of inadequate treatment.
Must have a stable condition for at least 1 year, meaning:
No major changes in neurological exams or mood disorder status.
Stable lab results related to copper metabolism, including 24-hour urinary copper, free serum copper, and other tests like liver enzymes, hemoglobin, and white blood cell count.

Who cannot join the trial?

8 criteria

Patients who are not adults. This means anyone under 18 years old cannot participate.
Patients who are pregnant or breastfeeding. This is to ensure the safety of both the mother and the baby.
Patients with a history of severe allergic reactions to medications. This is to prevent any serious health risks during the trial.
Patients with other serious health conditions that might interfere with the study. This includes conditions that could make it unsafe for the patient to participate.
Patients who are currently participating in another clinical trial. This is to avoid any interference with the results of either study.
Patients who have used certain medications recently that could affect the study results. This is to ensure accurate and reliable data.
Patients who have a history of substance abuse. This is to ensure the safety and reliability of the study results.
Patients who are unable to follow the study procedures or attend the required visits. This is important for the consistency and success of the trial.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

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Aarhus University Hospital
Aarhus, Denmark
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Universitaetsklinikum Essen AöR
Essen, Germany
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Universitaetsklinikum Tuebingen AöR
Tuebingen, Germany

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Investigated drugs

VTX-801 is an investigational therapy being studied for its potential to treat Wilson's Disease. It is administered as a single dose through an intravenous (IV) infusion. The study aims to evaluate the safety and tolerability of VTX-801 in adult patients with Wilson's Disease, especially when background Wilson's Disease therapy is withdrawn.

What is already known about the treatment

VTX-801 – This medication is administered intravenously, meaning it is given directly into a vein. It is currently being studied in clinical trials, specifically in a Phase I/II study, to evaluate its safety and tolerability in patients with Wilson's Disease. The main therapeutic indication for VTX-801 is Wilson's Disease, a genetic disorder that causes excessive copper accumulation in the body. At the molecular level, VTX-801 is designed to deliver a functional copy of the ATP7B gene, which is responsible for regulating copper levels in the body. It falls under the pharmacological classification of gene therapy, aiming to correct the underlying genetic defect in Wilson's Disease.

Investigated diseases

Wilson's Disease – Wilson's Disease is a rare genetic disorder that causes excessive copper accumulation in the body, particularly affecting the liver and brain. This buildup occurs due to the body's inability to properly eliminate copper, leading to its toxic accumulation. Initially, symptoms may include fatigue, abdominal pain, and jaundice, as the liver becomes damaged. As the disease progresses, neurological symptoms such as tremors, difficulty speaking, and coordination problems may develop. The condition can also affect mental health, causing mood swings and cognitive difficulties. Over time, if untreated, the damage to organs can become more pronounced.