Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial)
Achromatopsia
About this trial
This is an interventional treatment trial for Achromatopsia focused on measuring Color vision deficiency, Color vision defects, Vision disorders, Sensation disorders, Neurologic manifestations, Nervous system diseases, Eye diseases, Signs and symptoms
Eligibility Criteria
Inclusion Criteria:
- 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.
Sites / Locations
- VitreoRetinal Associates
- Bascom Palmer Eye Institute
- Massachusetts Eye and Ear Infirmary
- Cincinnati Eye Institute
- Casey Eye Institute, Oregon Health and Sciences University
- Hadassah-Hebrew University Medical Center
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Arm 6
Arm 7
Arm 8
Experimental
Experimental
Experimental
Experimental
Experimental
Experimental
Experimental
Experimental
Group 1: 4.0 x 10^10 vg/mL of AGTC-402
Group 2: 1.2 x 10^11 vg/mL of AGTC-402
Group 3: 3.6 x 10^11 vg/mL of AGTC-402
Group 3a: 3.6 x 10^11 vg/mL of AGTC-402
Group 4: 1.1 x 10^12 vg/mL of AGTC-402
Group 4a: 1.1 x 10^12 vg/mL of AGTC-402
Group 5: 3.2 x 10^12 vg/mL of AGTC-402
Group 6: MTD of AGTC-402
Subjects at least 18 y/o treated with 4.0 x 10^10 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Subjects at least 18 y/o treated with 1.2 x 10^11 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Subjects at least 18 y/o treated with 3.6 x 10^11 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Subjects 6 to 17 y/o treated with 3.6 x 10^11 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Subjects at least 18 y/o treated with 1.1 x 10^12 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Subjects 4 to 8 y/o treated with 1.1 x 10^12 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Subjects at least 18 y/o treated with 3.2 x 10^12 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Subjects 4 to 8 y/o treated with a maximum tolerated dose of rAAV2tYF-PR1/7-hCNGA3 study drug determined by Groups 1-5.