A Phase 1 Safety Study in Subjects With Leber Congenital Amaurosis (LCA) Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 Into the Retinal Pigment Epithelium (RPE) [AAV2-hRPE65v2-101]

Trial Profile

A Phase 1 Safety Study in Subjects With Leber Congenital Amaurosis (LCA) Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 Into the Retinal Pigment Epithelium (RPE) [AAV2-hRPE65v2-101]

Active, no longer recruiting
Phase of Trial: Phase I

Latest Information Update: 03 Oct 2017

At a glance

  • Drugs Voretigene neparvovec (Primary)
  • Indications Leber congenital amaurosis
  • Focus Adverse reactions
  • Acronyms LCARPE
  • Sponsors Spark Therapeutics
  • Most Recent Events

    • 10 Oct 2015 Results on 3 year durability of improvements presented at The Retina Society 48th Annual Scientific Meeting, as per Spark media release.
    • 12 Nov 2013 New trial record
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