A Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (LCA) Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 to the Retinal Pigment Epithelium (RPE) [AAV2-hRPE65v2-301]

Trial Profile

A Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (LCA) Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 to the Retinal Pigment Epithelium (RPE) [AAV2-hRPE65v2-301]

Active, no longer recruiting
Phase of Trial: Phase III

Latest Information Update: 10 Nov 2017

At a glance

  • Drugs Voretigene neparvovec (Primary)
  • Indications Leber congenital amaurosis
  • Focus Registrational; Therapeutic Use
  • Sponsors Spark Therapeutics
  • Most Recent Events

    • 10 Nov 2017 According to a Spark Therapeutics media release, data from this trial were presented at the American Academy of Ophthalmology (AAO) Retina Subspecialty Day 2017.
    • 10 Nov 2017 Three-year follow-up data published in the Spark Therapeutics Media Release
    • 07 Nov 2017 According to a Spark Therapeutics media release, the Marketing Authorization Application (MAA) for LUXTURNA has been validated by European Medicines Agency (EMA).
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