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]
Active, no longer recruiting
Phase of Trial: Phase III
Latest Information Update: 23 Feb 2018
At a glance
- Drugs Voretigene neparvovec (Primary)
- Indications Leber congenital amaurosis
- Focus Registrational; Therapeutic Use
- Sponsors Spark Therapeutics
- 23 Feb 2018 According to a Spark Therapeutics media release, data from this trial will be presented at the American Association for Pediatric Ophthalmology and Strabismus (AAPOS) Annual Meeting 2018.
- 19 Dec 2017 According to a U.S. Food and Drug Administration media release, The U.S. Food and Drug Administration today approved Luxturna (voretigene neparvovec-rzyl), a new gene therapy, to treat children and adult patients with an inherited form of vision loss that may result in blindness.Data from this study was used in NDA submission.
- 10 Nov 2017 According to a Spark Therapeutics media release, data from this trial were presented at the American Academy of Ophthalmology (AAO) Retina Subspecialty Day 2017.
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Table of Contents
- At a glance
- Trial Overview
- Trial Details
- Trial Centres
- Trial History