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

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 / Drugs

UX701 Standard of Care (SOC)

View on ClinicalTrials.gov ↗

Sponsor

RARE

Ultragenyx Pharmaceutical

Rare Disease

C63/100

Study Details

Enrollment82 participants

Primary Completion—

Lead SponsorUltragenyx Pharmaceutical Inc

Data Retrieved2026-04-11 04:25:35.045783+00:00

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