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A Phase 1/2/3, Multi-center, Two-part Clinical Trial to Evaluate the Safety and Efficacy of Gene Therapy for Leber's Hereditary Optic Neuropathy (LHON) Associated With ND4 Mutation

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Trial Profile

A Phase 1/2/3, Multi-center, Two-part Clinical Trial to Evaluate the Safety and Efficacy of Gene Therapy for Leber's Hereditary Optic Neuropathy (LHON) Associated With ND4 Mutation

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

Latest Information Update: 23 Feb 2023

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At a glance

  • Drugs Esonadogene imvoparvovec (Primary)
  • Indications Leber's hereditary optic atrophy
  • Focus Adverse reactions; Registrational; Therapeutic Use
  • Acronyms GOLD
  • Sponsors Neurophth Therapeutics

    • Most Recent Events

    • 21 Feb 2023 Status changed from recruiting to active, no longer recruiting.
    • 21 Feb 2023 According to Neuropth media release, the company announced the last patient enrollment in phase III clinical trial of this study for the treatment of Leber hereditary optic neuropathy (LHON) in China, with this enrollment it marks the completion of patient enrollment od the phase I/II/III of this study.
    • 08 Nov 2022 Planned End Date changed from 30 Apr 2027 to 29 Feb 2028.

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