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A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of AGTC 402, a Recombinant Adeno-associated Virus Vector Expressing CNGA3, in Patients With Congenital Achromatopsia Caused by Mutations in the CNGA3 Gene
This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-402, administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGA3 gene. The primary study endpoint will be safety and the secondary study endpoint will be efficacy.
Details
| Lead sponsor | Beacon Therapeutics |
|---|---|
| Phase | Phase 1/Phase 2 |
| Status | ACTIVE_NOT_RECRUITING |
| Enrolment | 24 |
| Start date | 2017-08-03 |
| Completion | 2026-08 |
Conditions
- Achromatopsia
Interventions
- AGTC-402
Primary outcomes
- Adverse events — 1 year
Proportion of participants experiencing grade 3 or greater adverse events
Countries
United States, Israel