Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial)
Running, not enrolling · Phase 1/Phase 2
Conditions studied: Achromatopsia
In brief
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.
Key facts
- Study ID
- NCT02935517
- Run by
- Beacon Therapeutics
- People needed
- 24
- Starts
- 2017-08-03
- Expected to finish
- 2026-08-01
- Last updated by the study team
- 2022-07-22
Who can join
Age: 4 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Male or female subjects with documented mutations in both alleles of the CNGA3 gene;
- Retinal disease consistent with a clinical diagnosis of achromatopsia;
- At least 18 years of age for Groups 1, 2, 3, 4, and 5. At least 6 years of age for Group 3a, and and 4-8 years of age for Groups 4a and 6;
- Able to perform tests of visual and retinal function;
- 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;
- Acceptable laboratory parameters;
- For females of childbearing potential: A negative pregnancy test within 2 days before administration of study agent.
- Exclusion Criteria include:
- Best-corrected visual acuity difference between the two eyes of > 15 ETDRS letters (3 lines);
- Evidence of degenerative myopia in the study eye;
- Pre-existing eye conditions that would contribute to vision loss in either eye or increase the risk of subretinal injection in the study eye.
Where it is running
- VitreoRetinal Associates — Gainesville, Florida, United States
- Bascom Palmer Eye Institute — Miami, Florida, United States
- Massachusetts Eye and Ear Infirmary — Boston, Massachusetts, United States
- Cincinnati Eye Institute — Cincinnati, Ohio, United States
- Casey Eye Institute, Oregon Health and Sciences University — Portland, Oregon, United States
- Hadassah-Hebrew University Medical Center — Jerusalem, Israel
Full record on ClinicalTrials.gov
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