drugset / Trial / NCT02599922
Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial)
Phase 1/2
Active not recruiting
32 enrolled
Beacon Therapeutics
National Eye Institute (NEI) · collab
Non-randomizedParallel-groupOpen-labelTreatment
Summary
This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-401 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGB3 gene. The primary study endpoint will be safety and the secondary study endpoint will be efficacy.
Timeline
- Start
- 2016-04-11
- Primary completion
- 2022-07
- Completion
- 2026-07
Publications
- Background Komaromy AM, Alexander JJ, Rowlan JS, Garcia MM, Chiodo VA, Kaya A, Tanaka JC, Acland GM, Hauswirth WW, Aguirre GD. Gene therapy rescues cone function in congenital achromatopsia. Hum Mol Genet. 2010 Jul 1;19(13):2581-93. doi: 10.1093/hmg/ddq136. Epub 2010 Apr 8.
- Davis JL. The Blunt End: Surgical Challenges of Gene Therapy for Inherited Retinal Diseases. Am J Ophthalmol. 2018 Dec;196:xxv-xxix. doi: 10.1016/j.ajo.2018.08.038. Epub 2018 Sep 5.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | rAAV2tYF-PR1.7-hCNGB3 | Gene therapy (AAV / viral vector) | 4e+10 vg | Other |
| Subject | rAAV2tYF-PR1.7-hCNGB3 | Gene therapy (AAV / viral vector) | 1.2e+11 vg | Other |
| Subject | rAAV2tYF-PR1.7-hCNGB3 | Gene therapy (AAV / viral vector) | 2e+11 vg | Other |
| Subject | rAAV2tYF-PR1.7-hCNGB3 | Gene therapy (AAV / viral vector) | 3.6e+11 vg | Other |
| Subject | rAAV2tYF-PR1.7-hCNGB3 | Gene therapy (AAV / viral vector) | 1.1e+12 vg | Other |
| Subject | rAAV2tYF-PR1.7-hCNGB3 | Gene therapy (AAV / viral vector) | 3.2e+12 vg | Other |