Trials / NCT03920007
Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
Underway, not enrolling Phase 1 / Phase 2 Gene therapy
- Sponsor
- Atsena Therapeutics Inc.
- Treatment
- ATSN-101; ATSN-101 Diluent Solution; Prednisone; Triamcinalone Acetonide; 1% Prednisolone; Trimethoprim/polymyxin B
- Gene
- GUCY2D
- Conditions
- Leber Congenital Amaurosis, LCA, LCA1
- Ages
- 6 Years to no upper limit
- Enrollment
- 15 participants
- Started
- 2019-10-22
- Main results expected
- 2023-05-19
- Results posted
- Yes
- Record last updated
- 2026-08-03
What the study is testing
Primary Objective:
To evaluate the safety and tolerability of ascending doses of ATSN-101 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 ATSN-101 administered as a unilateral subretinal injection in patients with GUCY2D-LCA.
Where it runs
United States (2)
- Casey Eye Institute - Oregon Health & Science University, Portland, Oregon
- Scheie Eye Institute, University of Pennsylvania, Philadelphia, Pennsylvania