Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial)
A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of AGTC 402, a Recombinant Adeno-associated Virus Vector Expressing CNGA3, in Patients With Congenital Achromatopsia Caused by Mutations in the CNGA3 Gene
1 other identifier
interventional
24
2 countries
6
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_1
Started Aug 2017
Longer than P75 for phase_1
6 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 13, 2016
CompletedFirst Posted
Study publicly available on registry
October 17, 2016
CompletedStudy Start
First participant enrolled
August 3, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2026
ExpectedJuly 22, 2022
July 1, 2022
5 years
October 13, 2016
July 20, 2022
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Adverse events
Proportion of participants experiencing grade 3 or greater adverse events
1 year
Secondary Outcomes (3)
Visual acuity
1 year
Light aversion
1 year
Color vision
1 year
Study Arms (8)
Group 1: 4.0 x 10^10 vg/mL of AGTC-402
EXPERIMENTALSubjects at least 18 y/o treated with 4.0 x 10\^10 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Group 2: 1.2 x 10^11 vg/mL of AGTC-402
EXPERIMENTALSubjects at least 18 y/o treated with 1.2 x 10\^11 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Group 3: 3.6 x 10^11 vg/mL of AGTC-402
EXPERIMENTALSubjects at least 18 y/o treated with 3.6 x 10\^11 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Group 3a: 3.6 x 10^11 vg/mL of AGTC-402
EXPERIMENTALSubjects 6 to 17 y/o treated with 3.6 x 10\^11 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Group 4: 1.1 x 10^12 vg/mL of AGTC-402
EXPERIMENTALSubjects at least 18 y/o treated with 1.1 x 10\^12 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Group 4a: 1.1 x 10^12 vg/mL of AGTC-402
EXPERIMENTALSubjects 4 to 8 y/o treated with 1.1 x 10\^12 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Group 5: 3.2 x 10^12 vg/mL of AGTC-402
EXPERIMENTALSubjects at least 18 y/o treated with 3.2 x 10\^12 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Group 6: MTD of AGTC-402
EXPERIMENTALSubjects 4 to 8 y/o treated with a maximum tolerated dose of rAAV2tYF-PR1/7-hCNGA3 study drug determined by Groups 1-5.
Interventions
AGTC-402 is a non-replicating, rep/cap-deleted, recombinant adeno-associated virus vector that expresses the CNGA3 gene.
Eligibility Criteria
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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (6)
VitreoRetinal Associates
Gainesville, Florida, 32607, United States
Bascom Palmer Eye Institute
Miami, Florida, 33136, United States
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, 02114, United States
Cincinnati Eye Institute
Cincinnati, Ohio, 45242, United States
Casey Eye Institute, Oregon Health and Sciences University
Portland, Oregon, 97239, United States
Hadassah-Hebrew University Medical Center
Jerusalem, 91120, Israel
Related Publications (1)
Banin E, Gootwine E, Obolensky A, Ezra-Elia R, Ejzenberg A, Zelinger L, Honig H, Rosov A, Yamin E, Sharon D, Averbukh E, Hauswirth WW, Ofri R. Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia. Mol Ther. 2015 Sep;23(9):1423-33. doi: 10.1038/mt.2015.114. Epub 2015 Jun 19.
PMID: 26087757BACKGROUND
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
David Jacobs, MD, MBA
Applied Genetics Technologies Corporation
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 13, 2016
First Posted
October 17, 2016
Study Start
August 3, 2017
Primary Completion
August 1, 2022
Study Completion (Estimated)
August 1, 2026
Last Updated
July 22, 2022
Record last verified: 2022-07
Data Sharing
- IPD Sharing
- Will not share