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Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
Condition: Leber Congenital Amaurosis · LCA · LCA1 · Sponsor: Atsena Therapeutics Inc.
About this study
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.
This description comes directly from the study's public registry record.
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Locations (2)
| Casey Eye Institute - Oregon Health & Science University | Portland, Oregon, United States | — |
| Scheie Eye Institute, University of Pennsylvania | Philadelphia, Pennsylvania, United States | — |
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Source record: clinicaltrials.gov/study/NCT03920007