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Adeno-Associated Viral Vector Serotype 9 Encoding Human Atp7B

This article discusses the clinical trials of UX701, an innovative gene therapy using Adeno-Associated Viral Vector Serotype 9 Encoding Human ATP7B for the treatment of Wilson Disease. The study aims to evaluate the safety and efficacy of this groundbreaking approach in managing copper metabolism in adult patients with Wilson Disease. We’ll explore the trial design, eligibility criteria, and potential outcomes of this promising treatment.

Table of Contents

What is UX701?

UX701 is an experimental gene therapy being developed for the treatment of Wilson Disease. It is also known as ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE 9 ENCODING HUMAN ATP7B or AAV9ATP7B-MBD456.[1] This therapy is designed to introduce a functional copy of the ATP7B gene into the body, which is responsible for copper metabolism.

How does UX701 work?

UX701 uses a modified virus called adeno-associated virus serotype 9 (AAV9) to deliver a healthy copy of the ATP7B gene to liver cells. The ATP7B gene provides instructions for making a protein that helps regulate copper levels in the body. In people with Wilson Disease, this gene is faulty, leading to copper accumulation. By introducing a working copy of the gene, UX701 aims to restore normal copper metabolism.[1]

What is Wilson Disease?

Wilson Disease is a rare genetic disorder that affects copper metabolism. People with this condition cannot properly remove excess copper from their body, leading to its accumulation in various organs, particularly the liver and brain. This can cause a wide range of symptoms, including liver disease, neurological problems, and psychiatric issues.[1]

Clinical Trial Details

A clinical trial is currently underway to evaluate the safety and effectiveness of UX701 for Wilson Disease. This study is divided into several stages:[1]

  • Stage 1 (Phase 1/2): This initial stage aims to assess the safety of UX701 and determine the best dose for further testing.
  • Stage 2 (Phase 3): This stage will compare UX701 to a placebo to evaluate its effectiveness in treating Wilson Disease.
  • Stage 3: This final stage will provide long-term follow-up to assess the ongoing safety and benefits of UX701.

Eligibility Criteria

To participate in this study, patients must meet certain criteria, including:[1]

  • Be 18 years or older
  • Have a confirmed diagnosis of Wilson Disease based on genetic testing
  • Be on stable copper chelator and/or zinc therapy for at least 12 months
  • Have stable urinary copper levels
  • Be willing to follow a low-copper diet

There are also several exclusion criteria, such as a history of liver transplant, advanced liver disease, or certain other medical conditions.

Potential Benefits

The potential benefits of UX701 therapy include:[1]

  • Improved copper regulation in the body
  • Reduced need for standard-of-care medications
  • Improved liver function and overall health
  • Better quality of life for patients with Wilson Disease

Safety Considerations

As with any experimental treatment, there are potential risks and side effects. The clinical trial is designed to carefully monitor participants for any adverse events. Some safety measures include:[1]

  • Regular monitoring of liver function and copper levels
  • Use of prophylactic oral corticosteroids to prevent potential liver-related side effects
  • Long-term follow-up to assess ongoing safety

It’s important to note that UX701 is still in the experimental stage, and its long-term safety and effectiveness are not yet fully known. Patients considering participation in this clinical trial should discuss the potential risks and benefits with their healthcare provider.

Aspect Details
Study Drug UX701 (Adeno-Associated Viral Vector Serotype 9 Encoding Human ATP7B)
Condition Wilson Disease
Study Design Multicenter, seamless, adaptive Phase 1/2/3 clinical study
Primary Objectives Evaluate safety, select optimal dose, assess effect on copper regulation, determine reduction in standard-of-care medication
Key Eligibility Criteria Adults ≥18 years, confirmed Wilson Disease diagnosis, stable on current therapy for ≥12 months
Study Duration 52 weeks initial follow-up, followed by long-term follow-up up to 4 years
Primary Endpoints Change in 24-hour urinary copper concentration, percent reduction in standard-of-care medication
Secondary Endpoints Changes in ceruloplasmin activity, patient-reported outcomes, liver copper concentration

FAQ

  • What is UX701 and how does it work? UX701 is a gene therapy that uses an adeno-associated viral vector (AAV9) to deliver a functional copy of the ATP7B gene to patients with Wilson Disease. This gene is responsible for copper metabolism, and the therapy aims to improve the body's ability to regulate copper levels.
  • Who is eligible for the UX701 clinical trial? The trial is open to adults (18 years and older) with a confirmed diagnosis of Wilson Disease based on genetic testing. Participants must have been on stable copper chelator and/or zinc therapy for at least 12 months and have maintained a copper-restricted diet.
  • How is the clinical trial structured? The trial is divided into multiple stages: Stage 1 (Phase 1/2) is a non-randomized, open-label study to evaluate safety and find the optimal dose. Stage 2 (Phase 3) is a randomized, double-blind, placebo-controlled study to assess the efficacy of the selected dose. Stage 3 involves long-term follow-up of participants.
  • What are the main goals of the UX701 clinical trial? The primary objectives are to evaluate the safety of UX701, assess its effect on copper regulation (measured by 24-hour urinary copper concentration), and determine if it can reduce the need for standard-of-care medication. The trial also aims to study the therapy's impact on biomarkers of copper metabolism, liver function, and overall health.
  • How long does the clinical trial last? The initial stages of the trial last 52 weeks, followed by a long-term follow-up period. Stage 3 of the trial includes a 4-year long-term follow-up to assess the ongoing safety and efficacy of UX701.

Ongoing Clinical Trials on Adeno-Associated Viral Vector Serotype 9 Encoding Human Atp7B

  • Study on the Safety and Effects of UX701 Gene Therapy for Adults with Wilson Disease

    Recruiting

    2 1 1 1
    Investigated drugs:
    Denmark France Germany Italy Portugal Spain

Glossary

  • Wilson 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.
  • Adeno-Associated Viral Vector (AAV): A small virus that has been modified to deliver genetic material into cells without causing disease. In gene therapy, AAVs are used to introduce functional genes to replace or supplement faulty ones.
  • ATP7B Gene: The gene responsible for producing a protein that helps regulate copper levels in the body. Mutations in this gene cause Wilson Disease.
  • Copper Chelator: A medication that binds to copper in the body, allowing it to be removed through urine. Examples include penicillamine and trientine.
  • Standard of Care (SOC) Medication: The currently accepted treatment for Wilson Disease, which typically includes copper chelators and/or zinc therapy.
  • 24-hour Urinary Copper Concentration: A test that measures the amount of copper excreted in urine over a 24-hour period. It's used to assess copper metabolism and monitor treatment effectiveness in Wilson Disease.
  • Ceruloplasmin: A protein that carries copper in the blood. Levels of ceruloplasmin are often low in people with Wilson Disease.
  • Genome Copies (GC): A measure of the number of viral particles or gene copies in a gene therapy dose, often expressed as GC/kg (genome copies per kilogram of body weight).

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-ux701-gene-therapy-for-adults-with-wilson-disease/