UX701 Gene Therapy for Wilson Disease
Trial Summary
What is the purpose of this trial?
This trial tests UX701, a new treatment for patients with Wilson disease. It aims to help these patients better control their copper levels, preventing harmful effects. The study will evaluate the safety and effectiveness of UX701 over time.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop your current medications. However, it requires that your Wilson disease treatment with copper chelator or zinc therapy has been stable for at least 6 months, with no changes in medication or dose during that time.
What data supports the effectiveness of the treatment UX701 for Wilson Disease?
Is UX701 gene therapy safe for humans?
How is the UX701 treatment for Wilson Disease different from other treatments?
UX701 is a gene therapy that uses a viral vector to deliver a modified version of the ATP7B gene, which helps correct copper metabolism in Wilson Disease. Unlike traditional treatments that require lifelong medication to manage symptoms, this approach aims to address the root cause of the disease by restoring normal copper processing in the liver.12456
Research Team
Medical Director
Principal Investigator
Ultragenyx Pharmaceutical Inc
Eligibility Criteria
This trial is for individuals with confirmed Wilson disease who have been avoiding high copper foods and taking copper chelators or zinc therapy consistently for at least 6 months. They should be able to follow the study's procedures, including blood and urine tests. People with significant liver inflammation, pre-existing antibodies to AAV9 capsid, severe neurological issues, a MELD score over 13, or history of liver transplant cannot participate.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Stage 1: Safety and Dose-Finding
Nonrandomized, open-label safety and dose-finding stage to evaluate the safety and efficacy of 3 dose levels of UX701
Stage 2: Randomized Treatment
Randomized, double-blind, placebo-controlled stage to evaluate the safety and efficacy of UX701 using the dose selected in Stage 1
Stage 3: Long-term Follow-up
Evaluation of long-term safety, efficacy, and clinical benefit of UX701. All participants will be followed for at least 5 years from the time of UX701 administration
Treatment Details
Interventions
- UX701
Find a Clinic Near You
Who Is Running the Clinical Trial?
Ultragenyx Pharmaceutical Inc
Lead Sponsor
Dr. Emil D. Kakkis
Ultragenyx Pharmaceutical Inc
Chief Executive Officer since 2010
MD/PhD in Biological Chemistry from UCLA
Dr. Eric Crombez
Ultragenyx Pharmaceutical Inc
Chief Medical Officer since 2023
MD from Wayne State University School of Medicine