Principal Investigator
Enrollment Status
Clinical Trial ID
Clinical Trial 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.
Phase
Phase 1/Phase 2
Funding Agency/Sponsor
Industry
Disease
Retina
Enrollment Eligibility
Inclusion Criteria include:
1. Male or female subjects with documented mutations in both alleles of the CNGB3 gene;
2. Retinal disease consistent with a clinical diagnosis of achromatopsia;
3. At least 18 years of age for Groups 1, 2, 3, 4, 5 and 6. At least 6 years of age for
Group 4a, and 4-8 years of age for Groups 5a and 7;
4. Able to perform tests of visual and retinal function;
5. 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;
6. Acceptable laboratory parameters;
7. For females of childbearing potential: A negative pregnancy test within 2 days before
administration of study agent.
Exclusion Criteria include:
1. Best-corrected visual acuity difference between the two eyes of > 15 ETDRS letters (3
lines);
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.