What side effects are associated with this type of intervention?

Gene therapy for Wilson's Disease, such as VTX-801, involves introducing genetic material to correct or compensate for defective genes. Common side effects of gene therapy can include immune reactions, where the body attacks the introduced viral vectors, leading to inflammation and other immune responses. There may also be risks of insertional mutagenesis, where the introduced genes disrupt normal genes, potentially causing cancer. In the context of Wilson's Disease, specific side effects observed in animal models include liver inflammation and fibrosis. Long-term effects and safety profiles are still under investigation, making it crucial for patients to discuss potential risks and benefits with their healthcare providers.

What prior FDA approvals exist?

Currently, there are no specific mentions of FDA-approved gene therapies for Wilson's Disease in the provided context. However, gene therapy approaches, such as the one being studied in the VTX-801 trial, aim to introduce genetic material to correct or compensate for defective genes responsible for the disease. These therapies are still in clinical trial phases, assessing their safety, tolerability, and pharmacological activity. Traditional treatments for Wilson's Disease typically involve chelating agents like penicillamine and trientine, which help remove excess copper from the body, and zinc acetate, which prevents copper absorption.

What other types of research exist?

A promising alternative to VTX-801 for Wilson's Disease is the use of synthetic AAV vector, AAVAnc80, carrying the miniATP7B gene. This approach has shown effectiveness in preventing liver damage, restoring copper homeostasis, and improving survival in preclinical studies with mice. Another potential alternative could be the development of small molecule drugs or other gene therapies that target similar pathways to correct copper metabolism.

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Gene Therapy

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

Phase 1 & 2

Waitlist Available

Research Sponsored by Vivet Therapeutics SAS

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Male or female aged 18 and 65 years inclusive

Confirmed diagnosis of WD

Must not have

Any history of diabetes

Positive QuantiFERON®-TB Gold tuberculosis test result

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at 1-year post treatment

Awards & highlights

No Placebo-Only Group

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects are not explicitly listed but may include reactions related to the infusion process, changes in liver and kidney function tests, and other unforeseen complications due to the nature of gene therapy.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 18 and 65 years old.

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I have been diagnosed with Wilson's disease.

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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

You may be eligible for the trial if you check “No” for criteria below:

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I have a history of diabetes.

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I tested positive for tuberculosis with the QuantiFERON test.

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I have never had HIV or HTLV.

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My kidney function is low or I have kidney disease.

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My ALT levels are high without an obvious external cause.

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I have had or currently have hepatitis B.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at 1-year post treatment

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at 1-year post treatment

This trial's timeline: 3 weeks for screening, Varies for treatment, and at 1-year post treatment for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Safety and tolerability profile (including treatment-emergent adverse events (TEAE))

Secondary study objectives

24-hour urinary Cu

Free serum Cu

Serum ceruloplasmin activity (enzymatic assay)

+2 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: VTX-801Experimental Treatment1 Intervention

0 / 9Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Wilson's Disease is primarily treated by chelation therapy, which uses agents like penicillamine or trientine to bind and remove excess copper from the body, and zinc therapy, which blocks copper absorption in the intestines. However, gene therapy, such as the VTX-801 trial, introduces functional copies of the ATP7B gene to correct the underlying genetic defect responsible for impaired copper metabolism. This approach aims to restore normal copper processing and excretion, potentially offering a long-term solution and reducing the need for lifelong medication. For patients, this could mean improved liver function, reduced risk of liver damage, and overall better management of the disease.

A Gene Therapy Approach to Improve Copper Metabolism and Prevent Liver Damage in a Mouse Model of Wilson Disease.Long-term metabolic correction of Wilson's disease in a murine model by gene therapy.Insights gained from gene therapy in animal models of retGC1 deficiency.