Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial)

Phase 1/2 Active not recruiting 24 enrolled Beacon Therapeutics

Non-randomizedSingle-groupOpen-labelTreatment

Summary

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.

Timeline

Start: 2017-08-03
Primary completion: 2022-08
Completion: 2026-08

Publications

Background Banin E, Gootwine E, Obolensky A, Ezra-Elia R, Ejzenberg A, Zelinger L, Honig H, Rosov A, Yamin E, Sharon D, Averbukh E, Hauswirth WW, Ofri R. Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia. Mol Ther. 2015 Sep;23(9):1423-33. doi: 10.1038/mt.2015.114. Epub 2015 Jun 19.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	AGTC-402	Gene therapy (AAV / viral vector)	4e+10 unknown	Other
Subject	AGTC-402	Gene therapy (AAV / viral vector)	1.2e+11 unknown	Other
Subject	AGTC-402	Gene therapy (AAV / viral vector)	3.6e+11 unknown	Other
Subject	AGTC-402	Gene therapy (AAV / viral vector)	1.1e+12 unknown	Other
Subject	AGTC-402	Gene therapy (AAV / viral vector)	3.2e+12 unknown	Other

Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial)

Summary

Timeline

Publications

Drugs

Indications