NCT04884815

A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease

An Operationally Seamless Phase 1/2/3 Study Consisting of a Safety and Dose-finding Phase 1/2 and Randomized, Open-label, Active-controlled Phase 3 to Evaluate UX701 AAV Gene Therapy in Adults With Wilson Disease

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 82 (estimated)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The primary objectives of this study are to evaluate the safety of single IV doses of UX701 in patients with Wilson disease, to select the UX701 dose with the best benefit/risk profile based on the totality of safety and efficacy data and to evaluate the effect of UX701 on copper regulation.

Detailed description

Stage 1 (Phase 1/2) is an open-label safety and dose-finding stage designed to evaluate the safety and efficacy of 4 dose levels of UX701 to establish initial safety of UX701 and select a safe and efficacious dose for further evaluation. Stage 2 (Phase 3) is a randomized, open-label, active-controlled stage to evaluate the safety and efficacy of UX701 using the dose selected in Stage 1. Stage 3 is a long-term follow-up stage designed to evaluate the safety, efficacy, and clinical benefit of UX701 for at least 5 years from the time of UX701 administration. Participants who receive UX701 will receive premedication, prophylactic oral corticosteroids and immunomodulation therapy.

Conditions

• Wilson Disease

Interventions

Type	Name	Description
GENETIC	UX701	Nonreplicating, recombinant gene transfer vector
DRUG	Standard of Care (SOC)	SOC treatment (i.e., copper chelators and/or zinc) administered according to standard regimens.

Timeline

• Start date: 2021-09-27
• Primary completion: 2029-03-01
• Completion: 2034-03-01
• First posted: 2021-05-13
• Last updated: 2026-03-13

Locations

16 sites across 5 countries: United States, Canada, Portugal, Spain, United Kingdom

Regulatory

• FDA-regulated drug study