UX701 Gene Therapy for Wilson Disease

Recruiting at 22 trial locations

Overseen By Patients Contact: Trial Recruitment

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Ultragenyx Pharmaceutical Inc

No Placebo Group

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.

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

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.

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

Treatment Details

Interventions

• UX701 (Gene Therapy)

Trial Overview The 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

Group I: Stage 3: Placebo or UX701 Experimental Treatment 2 Interventions

Participants randomized in Stage 2 to UX701 will receive a single, peripheral IV infusion of normal saline (placebo). Participants randomized in Stage 2 to placebo will be eligible for a single, peripheral IV infusion of UX701 at the selected dose.

Group II: Stage 2: UX701 or Placebo Experimental Treatment 2 Interventions

Participants randomized 2:1 to receive UX701 or Placebo. Participants randomized to UX701 receive a single, peripheral IV infusion of UX701 at a dose selected in Stage 1. Participants randomized to placebo will receive a single, peripheral IV infusion of normal saline (placebo).

Group III: Stage 1: UX701 Dose Level 3 Experimental Treatment 1 Intervention

Participants receive a single, peripheral IV infusion of UX701 at dose level 3.

Group IV: Stage 1: UX701 Dose Level 2 Experimental Treatment 1 Intervention

Participants receive a single, peripheral IV infusion of UX701 at dose level 2.

Group V: Stage 1: UX701 Dose Level 1 Experimental Treatment 1 Intervention

Participants receive a single, peripheral intravenous (IV) infusion of UX701 at dose level 1.

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