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A Phase 3, Multicenter, Randomized, Double-Masked, Sham-Controlled 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 3, Multicenter, Randomized, Double-Masked, Sham-Controlled 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 III

Latest Information Update: 17 Feb 2026

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At a glance

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

Most Recent Events

  • 17 Feb 2026 New trial record

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