A Follow-On Study to Evaluate the Safety of Re-Administration of Adeno-Associated Viral Vector Containing the Gene for Human RPE65 [AAV2-hRPE65v2] to the Contralateral Eye in Subjects With Leber Congenital Amaurosis (LCA) Previously Enrolled in a Phase 1 Study

Trial Profile

A Follow-On Study to Evaluate the Safety of Re-Administration of Adeno-Associated Viral Vector Containing the Gene for Human RPE65 [AAV2-hRPE65v2] to the Contralateral Eye in Subjects With Leber Congenital Amaurosis (LCA) Previously Enrolled in a Phase 1 Study

Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 03 Apr 2017

At a glance

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

    • 09 Jan 2017 An updated four-year long term data (n=8) published in the Spark Therapeutics Media Release.
    • 07 Jul 2016 New trial record
    • 01 Jul 2016 Spark plans to present efficacy analyses on the four-year follow-up data from the relevant cohort of the this trial (n=8) in 4Q 2016, according to a company media release.
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