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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]

Status: Active, no longer recruiting
Phase of Trial: Phase I

Latest Information Update: 23 Nov 2018

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At a glance

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

    • 23 Nov 2018 According to a Novartis media release, the EC decision for approval of Luxturna, is based on a positive CHMP opinion opinion, that looked at the data from this phase 1 clinical trial (NCT00516477), its follow-up trial (NCT01208389) and a phase 3 trial (NCT00999609).
    • 23 Nov 2018 According to a Spark Therapeutics media release,European commission has granted a marketing authorization for Luxturna (voretigene neparvovec), for the treatment of adult and pediatric patients with vision loss due to inherited retinal dystrophy, this authorization will be valid in all 28 member states of the EU, as well as Iceland, Liechtenstein and Norway.
    • 21 Sep 2018 According to a Novartis media release, a marketing authorization decision from the European Commission is anticipated within approximately two months of the positive CHMP opinion. If approved, the authorization will be valid in all 28 member states of the EU, as well as Iceland, Liechtenstein and Norway.
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