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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]

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

Latest Information Update: 23 Nov 2018

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At a glance

  • Drugs Voretigene neparvovec (Primary)
  • Indications Leber congenital amaurosis
  • Focus Registrational; Therapeutic Use
  • 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 a phase 1 clinical trial (NCT00516477), its follow-up trial (NCT01208389) and this 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.
    • 29 Oct 2018 Results presented in the Spark Technology media release.
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