A Phase 1/2 Dose Escalation Study of Subretinally Injected SAR439483 Administered in Patients with Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D

This Phase 1/2 study will evaluate the safety, tolerability and efficacy of ascending doses of AAV5-GUCY2D gene therapy (SAR439483) administered as a unilateral subretinal injection in patients with Leber Congenital Amaurosis (LCA) caused by autosomal recessive GUCY2D mutations (GUCY2D-LCA). The study will be separated into two phases, the dose escalation phase and a dose expansion phase. In the dose escalation phase, approximately 9 adult patients with two confirmed GUCY2D mutations will be enrolled in Cohorts 1-3 (3 per cohort), and cohorts will be treated with ascending doses of the study drug (i.e. cohort 1 will receive 1 low dose, cohort 2 will receive 1 mid-dose, cohort 3 will receive 1 high dose). Cohorts 4 and 5 will be enrolled during the dose expansion phase and will have approximately 3 adult and 3 pediatric participants in each respective cohort. The cohorts for the dose expansion phase will be treated with the maximum tolerated dose or maximum administered dose (whichever has the more optimal risk-benefit profile) as identified in the dose escalation phase. Evaluation of a safe and effective dose of the study drug will enable optional dose identification and further characterization of the safety profile in the target population in a pivotal study.

You may be eligible for this study if you meet the following criteria:

• Conditions:
  TBD
• Age: - 99 Years
• Gender: All