Clinical Trial: Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: 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 CN

Brief 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.

Detailed 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.

Subjects will be enrolled sequentially in four groups. Subjects in Groups 1, 2 and 3 will be between 18 and 55 years of age, inclusive, and will receive a lower, middle or higher dose of study agent. Subjects in Group 4 will be between 6 and 55 years of age, inclusive, and will receive the maximum tolerated dose identified in Groups 1, 2 and 3.

Safety will be monitored by evaluation of ocular and non ocular adverse events and hematology and clinical chemistry parameters. Efficacy parameters will include visual acuity, light discomfort testing, color vision, static visual field, ERG, adaptive optics retinal imaging and OCT.


Sponsor: Applied Genetic Technologies Corp

Current Primary Outcome: Adverse events [ Time Frame: 1 year ]

Proportion of participants experiencing grade 3 or greater adverse events


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Visual acuity [ Time Frame: 1 year ]
    Changes in best corrected visual acuity compared to pre-treatment
  • Light aversion [ Time Frame: 1 year ]
    Changes in light discomfort testing compared to pre-treatment
  • Color vision [ Time Frame: 1 year ]
    Changes in color vision testing compared to pre-treatment


Original Secondary Outcome: Same as current

Information By: Applied Genetic Technologies Corp

Dates:
Date Received: October 13, 2016
Date Started: March 2017
Date Completion: June 2023
Last Updated: March 7, 2017
Last Verified: March 2017