drugset / Trial / NCT03920007
Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
NaSingle-groupOpen-labelTreatment
Summary
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.
Timeline
- Start
- 2019-09-12
- Primary completion
- 2023-05-19
- Completion
- 2027-05-19
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | ATSN-101 | Unknown | — | Other |
| Background | Prednisolone | Other / unclassified | 1 % | Other |
| Background | Prednisone | Other / unclassified | — | Oral |
| Background | Triamcinalone Acetonide | Unknown | — | Other |
| Background | Trimethoprim/polymyxin B | Unknown | — | Topical |