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

NCT04884815 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 82

Last updated 2026-03-13

No results posted yet for this study

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.

Conditions

  • Wilson Disease

Interventions

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.

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceutical Inc

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-09-27
Primary Completion
2029-03-31
Completion
2034-03-31
FDA Drug
Yes

Countries

  • United States
  • Canada
  • Portugal
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04884815 on ClinicalTrials.gov