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An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno Associated Virus Vector for Gene Therapy of Adults And Children With Retinal Dystrophy Associated With Defects in RPE65 (LCA)

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Trial Profile

An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno Associated Virus Vector for Gene Therapy of Adults And Children With Retinal Dystrophy Associated With Defects in RPE65 (LCA)

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 06 Nov 2021

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At a glance

  • Drugs Cevaretigene ritoparvovec (Primary)
  • Indications Leber congenital amaurosis
  • Focus Adverse reactions
  • Acronyms OPTIRPE65
  • Sponsors MeiraGTx

    • Most Recent Events

    • 07 Nov 2019 According to a MeiraGTx media release, data from this trial were presented in September 2019 at the 52nd Annual Scientific meeting of the Retina Society and in October 2019 at American academy of ophthalmology Annual Meeting
    • 10 Sep 2019 According to a MeiraGTx media release, data from this trial will be presented by trial investigator, Michel Michaelides at 52nd Annual Scientific Meeting of The Retina Society and Retina Subspecialty Day of the American Academy of Ophthalmology (AAO) 2019 Annual Meeting. The company will host a conference call and live webcast following Professor Michaelides presentation at AAO to review the data from trial.
    • 14 May 2019 Status changed from recruiting to completed according to a MeiraGTx media release.

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