Trial Profile

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, no longer recruiting

Phase of Trial: Phase I/II

Latest Information Update: 22 May 2025

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At a glance

11 May 2025 Planned number of patients changed from 94 to 82.
11 May 2025 Planned End Date changed from 1 Nov 2031 to 1 Mar 2034.
11 May 2025 Planned primary completion date changed from 1 Aug 2026 to 1 Mar 2029.

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