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 - UX701-CL301

Status: Active, no longer recruiting

Phase of Trial: Phase I/II

Latest Information Update: 28 Nov 2025

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At a glance

Drugs Rivunatpagene miziparvovec (Primary) ; Eculizumab; Prednisolone; Rituximab; Tacrolimus
Indications Hepatolenticular degeneration
Focus Adverse reactions; First in man; Registrational; Therapeutic Use
Acronyms Cyprus2+
Sponsors Ultragenyx Pharmaceutical

04 Nov 2025 According to an Ultragenyx Pharmaceutical media release, cohort 4 enrollment complete, data expected in the first half of 2026.
19 Sep 2025 Status changed from recruiting to active, no longer recruiting.
11 May 2025 Planned number of patients changed from 94 to 82.

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