82 Participants Needed

UX701 Gene Therapy for Wilson Disease

Recruiting at 23 trial locations
HC
PC
Overseen ByPatients Contact: Trial Recruitment
Age: 18+
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: Ultragenyx Pharmaceutical Inc
Must be taking: Copper chelators, Zinc
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

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?

Research in animal models, like mice and rats, shows that gene therapy can help correct copper metabolism and reduce liver damage in Wilson Disease. This suggests that similar gene therapy approaches, like UX701, might be effective in treating the condition.12345

Is UX701 gene therapy safe for humans?

The safety data for UX701 gene therapy in humans is not directly available, but studies in mice with Wilson Disease show that similar gene therapy approaches can reduce liver damage and improve copper metabolism without significant toxicity.14567

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

MD

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

I have been on a stable Wilson disease treatment for at least 6 months.
I am willing and able to follow all study requirements, including frequent tests and long-term follow-up.
I have been diagnosed with Wilson disease.
See 1 more

Exclusion Criteria

Participation in another gene transfer study or use of another gene transfer product before or during study participation
Your MELD score is higher than 13.
History of copper chelator or zinc therapy noncompliance, in the Investigator's judgment, within 6 months prior to Screening
See 9 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

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

Varies

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

Varies

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

5 years

Treatment Details

Interventions

  • UX701
Trial OverviewThe study is testing UX701 gene transfer against a placebo in patients with Wilson disease. It aims to assess the safety of single IV doses of UX701 and its effectiveness on regulating copper levels in the body. The best dose will be chosen based on safety and efficacy data collected during the trial.
Participant Groups
6Treatment groups
Experimental Treatment
Group I: Stage 2: UX701 at Selected DoseExperimental Treatment1 Intervention
Participants randomized to UX701 receive a single, peripheral IV infusion of UX701 at the selected dose.
Group II: Stage 2: Standard of Care (SOC) to UX701Experimental Treatment2 Interventions
Participants randomized to SOC will continue their baseline SOC medications for 52 weeks, followed by a single, peripheral IV infusion of UX701 at the selected dose. Following UX701 administration, participants will be evaluated for modification of their SOC medications.
Group III: Stage 1: UX701 Dose Level 4Experimental Treatment1 Intervention
Participants receive a single, peripheral IV infusion of UX701 at dose level 4.
Group IV: Stage 1: UX701 Dose Level 3Experimental Treatment1 Intervention
Participants receive a single, peripheral IV infusion of UX701 at dose level 3.
Group V: Stage 1: UX701 Dose Level 2Experimental Treatment1 Intervention
Participants receive a single, peripheral IV infusion of UX701 at dose level 2.
Group VI: Stage 1: UX701 Dose Level 1Experimental Treatment1 Intervention
Participants receive a single, peripheral intravenous (IV) infusion of UX701 at dose level 1.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Ultragenyx Pharmaceutical Inc

Lead Sponsor

Trials
94
Recruited
104,000+

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

References

Long-term metabolic correction of Wilson's disease in a murine model by gene therapy. [2019]
Chances and shortcomins of adenovirus-mediated ATP7B gene transfer in Wilson disease: proof of principle demonstrated in a pilot study with LEC rats. [2019]
3.United Arab Emiratespubmed.ncbi.nlm.nih.gov
Perspectives for gene therapy of Wilson disease. [2019]
A Gene Therapy Approach to Improve Copper Metabolism and Prevent Liver Damage in a Mouse Model of Wilson Disease. [2020]
Long-Term Correction of Copper Metabolism in Wilson's Disease Mice with AAV8 Vector Delivering Truncated ATP7B. [2020]
Early gestational gene transfer with targeted ATP7B expression in the liver improves phenotype in a murine model of Wilson's disease. [2021]
Pathophysiology and clinical features of Wilson disease. [2022]