Clinical Trial: Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing CNGB3 (rAAV2tYF-PR1.7-hCNGB3) in Patients With Congenital Achromatopsia Caused by Mutati

Brief Summary: This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of rAAV2tYF-PR1.7-hCNGB3 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.

Detailed Summary:

This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of rAAV2tYF-PR1.7-hCNGB3 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.

Subjects will be enrolled sequentially in four groups. Subjects in Groups 1, 2 and 3 will be at least 18 years of age and will receive a lower, middle or higher dose of study agent. Subjects in Group 4 will be at least 6 years of age 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: November 5, 2015
Date Started: February 2016
Date Completion: December 2022
Last Updated: March 7, 2017
Last Verified: March 2017