An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Tolerability of GS010 (rAAV2/2-ND4) in Patients With Leber Hereditary Optic Neuropathy Due to Mutations in the Mitochondrial NADH Dehydrogenase 4 Gene

Trial Profile

An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Tolerability of GS010 (rAAV2/2-ND4) in Patients With Leber Hereditary Optic Neuropathy Due to Mutations in the Mitochondrial NADH Dehydrogenase 4 Gene

Completed
Phase of Trial: Phase I/II

Latest Information Update: 14 Jun 2017

At a glance

  • Drugs GS 010 (Primary)
  • Indications Leber's hereditary optic atrophy
  • Focus Adverse reactions
  • Sponsors GenSight Biologics
  • Most Recent Events

    • 14 Jun 2017 Long-term results after 96 weeks of follow-up published in GenSight Biologics media release.
    • 28 Apr 2017 Results presented at the 69th Annual Meeting of the American Academy of Neurology.
    • 22 Feb 2017 Planned End Date changed from 1 Feb 2019 to 1 Jun 2020.
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