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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

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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: 09 Oct 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

Most Recent Events

  • 19 Sep 2025 Status changed from recruiting to active, no longer recruiting.
  • 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.

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