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

Description

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.

Study Start Date

May, 01 2017

Estimated Completion Date

June 2023

Interventions

  • Biological: AGTC-402

Study ID

Applied Genetic Technologies Corp -- AGTC-CNGA3-002

Status

Recruiting

Trial ID

NCT02935517

Study Type

Interventional

Trial Phase

Phase 1/Phase 2

Enrollment Quota

24

Sponsor

Applied Genetic Technologies Corp

Inclusion Criteria

    1. Retinal disease consistent with a diagnosis of achromatopsia and documented mutations in both alleles of the CNGA3 gene 2. At least 18 years of age for Groups 1, 2 and 3 and at least 6 years of age for Group 4 3. Able to perform tests of visual and retinal function 4. Visual acuity in the study eye not better than 55 ETDRS letters (Snellen equivalent 20/80) based on the average of two examinations at the baseline visit 5. Acceptable laboratory parameters 6. For females of childbearing potential: A negative pregnancy test within 2 days before administration of study agent.

Exclusion Criteria

    1. Presence of myopia which, in the opinion of Investigator, may create increased surgical risk for the subject in the study eye 2. Evidence of degenerative myopia in the study eye 3. Pre-existing eye conditions that would contribute to vision loss in either eye or increase the risk of subretinal injection in the study eye.

Gender

All

Ages

6 Years and older

Accepts Healthy Volunteers

No

Study Locations and Contact Information (9)

Study Location Distance Name Phone Email
Massachusetts Eye and Ear Infirmary - Boston, Massachusetts 2.8 miles None None None
University of Pennsylvania - Philadelphia, Pennsylvania 270.8 miles None None None
VitreoRetinal Associates - Gainesville, Florida 1,083.3 miles None None None
Bascom Palmer Eye Institute - Miami, Florida 1,259.8 miles None None None
Casey Eye Institute Oregon Health and Sciences University - Portland, Oregon 2,538.3 miles None None None
Casey Eye Institute Oregon Health and Sciences University - Portland, Oregon 2,538.3 miles None None None
Casey Eye Institute Oregon Health and Sciences University - Portland, Oregon 2,538.3 miles None None None
Casey Eye Institute Oregon Health and Sciences University - Portland, Oregon 2,538.3 miles None None None
Casey Eye Institute Oregon Health and Sciences University - Portland, Oregon 2,538.3 miles None None None

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