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Adeno-Associated Viral Vector Serotype 3B Encoding Shortened Human Atp7B

This article provides an overview of a clinical trial investigating the use of VTX-801, a gene therapy drug containing an Adeno-Associated Viral Vector Serotype 3B Encoding Shortened Human ATP7B, for the treatment of Wilson’s Disease. The trial aims to assess the safety, tolerability, and potential effectiveness of this innovative approach in adult patients with Wilson’s Disease.

Table of Contents

What is VTX-801?

VTX-801 is a new gene therapy medication being developed to treat Wilson’s Disease. It is currently being tested in a Phase I/II clinical trial to assess its safety and effectiveness in adult patients.[1]

The full name of this medication is ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE 3B ENCODING SHORTENED HUMAN ATP7B. This long name describes how the medication is made and how it works. Let’s break it down:

  • Adeno-associated viral vector: This is a harmless virus that has been modified to carry genetic material into cells.
  • Serotype 3B: This refers to a specific type of the virus that has been chosen for this therapy.
  • Encoding shortened human ATP7B: The virus carries a shortened version of the ATP7B gene, which is the gene that doesn’t work properly in people with Wilson’s Disease.

Wilson’s Disease

Wilson’s Disease is a rare genetic disorder that causes copper to build up in the body, particularly in the liver and brain. This can lead to serious health problems if left untreated. Currently, patients with Wilson’s Disease need to take medication daily for their entire lives to manage the condition.[1]

How VTX-801 Works

VTX-801 is a type of gene therapy. It works by delivering a working copy of the ATP7B gene to the patient’s liver cells. This gene helps the body remove excess copper properly. The therapy uses a modified virus to carry the gene into the cells.[1]

Specifically, VTX-801 contains:

  • A non-replicating recombinant adeno-associated viral (AAV) vector: This is the modified virus that carries the gene.
  • A shortened form of the human ATP7B gene (ATP7B-minigene): This is a compact version of the gene that still functions properly.
  • Instructions for making a shortened but functional form of the ATP7B protein (miniATP7B): This protein helps remove excess copper from the body.

The therapy is given as a single dose through an intravenous (IV) infusion, which means it’s delivered directly into the bloodstream.[1]

Clinical Trial Details

The clinical trial for VTX-801 is a Phase I/II study, which means it’s an early-stage trial focused on safety and initial effectiveness. Here are some key details about the trial:[1]

  • It’s a multicenter trial, meaning it’s being conducted at multiple hospitals or research centers.
  • It’s non-randomized and open-label, which means all participants receive the treatment and both patients and doctors know what treatment is being given.
  • The trial uses an adaptive design, allowing for modifications based on early results.
  • Patients will be followed for 5 years after receiving the treatment.
  • The trial uses a single dose-escalation approach, meaning different groups of patients may receive different doses of the medication to find the best dose.

Eligibility Criteria

To participate in the trial, patients must meet certain criteria. Some of the key inclusion criteria are:[1]

  • Age between 18 and 65 years old
  • Confirmed diagnosis of Wilson’s Disease
  • Currently receiving treatment for Wilson’s Disease according to international recommendations
  • Stable Wilson’s Disease for at least 1 year

There are also several exclusion criteria, which are conditions that would prevent a person from participating in the trial. These include certain liver problems, other health conditions, and pregnancy.

Safety and Effectiveness

The main goal of this trial is to assess the safety and tolerability of VTX-801. Researchers will be looking at:[1]

  • Side effects that occur during treatment
  • Changes in laboratory test results
  • Changes in vital signs and heart activity (ECG)
  • Changes in brain and abdominal MRI scans

The trial will also look at how well the treatment works. This includes measuring:

  • Copper levels in urine over 24 hours
  • Activity of a protein called ceruloplasmin in the blood
  • Whether patients respond to the treatment
  • How the immune system responds to the treatment

It’s important to note that as this is an early-stage trial, the full effectiveness and any potential long-term side effects of VTX-801 are not yet known. More research will be needed to fully understand how well this treatment works and how safe it is for long-term use.

Aspect Details
Study Type Phase I/II, Multicenter, Non-randomized, Open Label, Adaptive Design
Drug Name VTX-801 (Adeno-Associated Viral Vector Serotype 3B Encoding Shortened Human ATP7B)
Condition Wilson’s Disease
Primary Objective Assess safety and tolerability of single ascending doses of VTX-801
Secondary Objectives Explore pharmacodynamics and efficacy, assess immune responses, support dose selection
Key Inclusion Criteria Adults 18-65 years, confirmed Wilson’s Disease diagnosis, stable for ≥1 year
Key Exclusion Criteria Liver cirrhosis, HIV, hepatitis B/C, severe renal impairment, pregnancy
Administration Single intravenous dose
Follow-up Duration 5 years

FAQ

  • What is VTX-801 and how does it work? VTX-801 is a gene therapy product designed to treat Wilson's Disease. It uses a modified virus (adeno-associated viral vector) to deliver a shortened but functional version of the ATP7B gene to patients' cells. This gene helps the body properly process copper, which is the main issue in Wilson's Disease.
  • Who is eligible to participate in this clinical trial? The trial is open to adults aged 18-65 with a confirmed diagnosis of Wilson's Disease. Participants must have been on stable treatment for at least one year and show no signs of inadequate treatment or significant changes in their condition.
  • How is VTX-801 administered in the trial? VTX-801 is administered as a single dose through intravenous (IV) infusion. The trial will test different dose levels to determine the most appropriate dosage.
  • What are the main objectives of this clinical trial? The primary objective is to assess the safety and tolerability of VTX-801 in adult patients with Wilson's Disease. Secondary objectives include exploring the drug's effectiveness, evaluating immune responses to the treatment, and gathering data to support dose selection for future studies.
  • How long will participants be followed in this study? The trial includes a 5-year follow-up period to monitor the long-term effects and safety of the VTX-801 treatment in patients with Wilson's Disease.

Ongoing Clinical Trials on Adeno-Associated Viral Vector Serotype 3B Encoding Shortened Human Atp7B

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

    Not recruiting

    2 1 1
    Investigated drugs:
    Denmark Germany

Glossary

  • Wilson's Disease: A rare genetic disorder that causes copper to accumulate in the liver, brain, and other vital organs. Without treatment, it can cause severe organ damage and life-threatening complications.
  • Gene Therapy: A technique that uses genes to treat or prevent disease. In this case, it involves introducing a functional version of the ATP7B gene to help the body process copper correctly.
  • Adeno-Associated Viral Vector: A modified virus used to deliver genetic material into cells. It's considered safe because it doesn't cause disease in humans and can effectively transport genes to target cells.
  • ATP7B: The gene responsible for producing a protein that helps regulate copper levels in the body. Mutations in this gene cause Wilson's Disease.
  • Intravenous (IV): A method of administering medication directly into a vein using a needle or tube.
  • Pharmacodynamics: The study of how a drug affects the body, including its mechanism of action and the relationship between drug concentration and effect.
  • ALT (Alanine Aminotransferase): An enzyme found primarily in the liver. Elevated levels can indicate liver damage or disease.
  • ULN (Upper Limit of Normal): The highest value of a physiological measurement considered normal in healthy people.
  • Ceruloplasmin: A protein that carries copper in the blood and helps regulate iron metabolism.
  • Treatment-Emergent Adverse Events (TEAE): Any unfavorable and unintended sign, symptom, or disease that appears or worsens after starting a medical treatment.

References

  1. http://clinicaltrials.eu/trial/study-on-gene-therapy-with-vtx-801-and-copper-64cu-chloride-for-adults-with-wilsons-disease/