Study of Subretinally Injected SAR439483 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D

• Status: Recruiting
• Trial end date: 2022-02-01

Summary

Primary Objective:

To evaluate the safety and tolerability of ascending doses of SAR439483 administered as a unilateral subretinal injection in patients with Leber Congenital Amaurosis (LCA) caused by autosomal recessive guanylate cyclase 2D (GUCY2D) mutations (GUCY2D-LCA).

Secondary Objective:

To evaluate the efficacy of ascending doses of SAR439483 administered as a unilateral subretinal injection in patients with GUCY2D-LCA.

• Phase: Phase 1/Phase 2

Details

• Lead Sponsor: Atsena Therapeutics Inc.; Sanofi
• Treatments: Pharmaceutical Solutions; Polymyxin B; Polymyxins; Prednisolone; Prednisone; Trimethoprim