A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing RPE65 (rAAV2-CB-hRPE65) in Patients With Leber Congenital Amaurosis Type 2

Trial Profile

A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing RPE65 (rAAV2-CB-hRPE65) in Patients With Leber Congenital Amaurosis Type 2

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

Latest Information Update: 29 Jul 2017

At a glance

  • Drugs Voretigene neparvovec (Primary)
  • Indications Leber congenital amaurosis
  • Focus Adverse reactions
  • Sponsors Applied Genetic Technologies Corporation
  • Most Recent Events

    • 26 Jun 2017 Planned End Date changed from 1 Dec 2027 to 1 Dec 2017.
    • 22 Apr 2016 Results published online in the journal Ophthalmology and will appear in the April print issue of the journal, according to an Applied Genetic Technologies Corporation media release.
    • 22 Apr 2016 Results published in an Applied Genetic Technologies Corporation media release.
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