Last updated on February 2018

Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)


Brief description of study

A clinical trial of AAV2/5 vector for patients with Defects in RPE65

Detailed Study Description

A dose escalation and dose expansion (Phase I/II) trial of adults and children with retinal dystrophy associated with defects in RPE65. ATIMP will be administered to one eye only in a single sub-retinal procedure

Clinical Study Identifier: NCT02781480

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Recruitment Status: Open


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