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A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Seamless, Adaptive, Safety, Dose-Finding, and Phase 3 Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson Disease

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Trial Profile

A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Seamless, Adaptive, Safety, Dose-Finding, and Phase 3 Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson Disease

Status: Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 13 Oct 2024

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At a glance

  • Drugs UX 701 (Primary) ; Prednisolone
  • Indications Hepatolenticular degeneration
  • Focus Adverse reactions; First in man; Registrational; Therapeutic Use
  • Acronyms Cyprus2+
  • Sponsors Ultragenyx Pharmaceutical

    • Most Recent Events

    • 03 Oct 2024 According to an Ultragenyx Pharmaceutical media release, the company will be submitting a protocol amendment for the additional cohort at a moderately increased dose and with an optimized immunomodulation regimen to optimize delivery efficiency and efficacy for the AAV vector.
    • 03 Oct 2024 According to an Ultragenyx Pharmaceutical media release, in Stage 1, 15 patients were enrolled into three sequential dosing cohorts and followed for at least 24 weeks.
    • 03 Oct 2024 According to an Ultragenyx Pharmaceutical media release, the company plans to enroll an additional cohort in Stage 1 at a moderately increased dose and with an optimized immunomodulation regimen to enhance the efficiency and efficacy of the gene therapy, with the objective of having the majority of patients come off of standard-of-care treatment before selecting a dose for the randomized placebo-controlled stage of the study.

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