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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]

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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: 13 Mar 2024

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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

    • 15 Oct 2020 According to a Novartis media release, based on the data of this study, the Health Canada has approved Luxturna (voretigene neparvovec), a one-time gene therapy for the treatment of adult and pediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.
    • 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.
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