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Virus Therapy
AAV Gene Therapy for Color Blindness
Phase 1 & 2
Waitlist Available
Research Sponsored by Applied Genetic Technologies Corp
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Male or female subjects with documented mutations in both alleles of the CNGA3 gene
Male or female subjects with documented mutations in both alleles of the CNGA3 gene;
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights
Study Summary
This trial will test a new treatment for achromatopsia, a condition that causes decreased color vision. The study will measure the safety and effectiveness of the new treatment.
Who is the study for?
This trial is for males and females with a specific type of color blindness called CNGA3 achromatopsia. Participants must have mutations in both alleles of the CNGA3 gene, be at least 18 years old (or as young as 6 for certain groups), and have visual acuity not better than 20/80. Women who can bear children must test negative for pregnancy.Check my eligibility
What is being tested?
The trial tests AGTC-402, a new gene therapy given through an injection into one eye to treat achromatopsia caused by CNGA3 mutations. It's an open-label study, meaning everyone knows they're getting the treatment, focusing on safety first and then how well it works.See study design
What are the potential side effects?
While specific side effects are not listed here, typical risks may include reactions at the injection site, potential worsening of vision or eye discomfort, and general risks associated with subretinal injections.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have mutations in both copies of my CNGA3 gene.
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I have mutations in both copies of my CNGA3 gene.
Select...
I have been diagnosed with achromatopsia.
Select...
I am 18 years old or older.
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I am a woman who can have children and have a recent negative pregnancy test.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 1 year
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Adverse events
Secondary outcome measures
Color vision
Light aversion
Visual acuity
Trial Design
8Treatment groups
Experimental Treatment
Group I: Group 6: MTD of AGTC-402Experimental Treatment1 Intervention
Subjects 4 to 8 y/o treated with a maximum tolerated dose of rAAV2tYF-PR1/7-hCNGA3 study drug determined by Groups 1-5.
Group II: Group 5: 3.2 x 10^12 vg/mL of AGTC-402Experimental Treatment1 Intervention
Subjects at least 18 y/o treated with 3.2 x 10^12 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Group III: Group 4a: 1.1 x 10^12 vg/mL of AGTC-402Experimental Treatment1 Intervention
Subjects 4 to 8 y/o treated with 1.1 x 10^12 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Group IV: Group 4: 1.1 x 10^12 vg/mL of AGTC-402Experimental Treatment1 Intervention
Subjects at least 18 y/o treated with 1.1 x 10^12 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Group V: Group 3a: 3.6 x 10^11 vg/mL of AGTC-402Experimental Treatment1 Intervention
Subjects 6 to 17 y/o treated with 3.6 x 10^11 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Group VI: Group 3: 3.6 x 10^11 vg/mL of AGTC-402Experimental Treatment1 Intervention
Subjects at least 18 y/o treated with 3.6 x 10^11 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Group VII: Group 2: 1.2 x 10^11 vg/mL of AGTC-402Experimental Treatment1 Intervention
Subjects at least 18 y/o treated with 1.2 x 10^11 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Group VIII: Group 1: 4.0 x 10^10 vg/mL of AGTC-402Experimental Treatment1 Intervention
Subjects at least 18 y/o treated with 4.0 x 10^10 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
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Who is running the clinical trial?
Applied Genetic Technologies CorpLead Sponsor
10 Previous Clinical Trials
329 Total Patients Enrolled
Matt Feinsod, MDStudy DirectorApplied Genetics Technologies Corporation
3 Previous Clinical Trials
154 Total Patients Enrolled
David Jacobs, MD, MBAStudy DirectorApplied Genetics Technologies Corporation
1 Previous Clinical Trials
32 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am a woman who can have children and have a recent negative pregnancy test.I can undergo tests for my eyesight and retina.I am at least 18 years old, or I am 6-8 years old if in specific groups.I have mutations in both copies of my CNGA3 gene.Your vision in the eye being studied is not better than 20/80 on an eye chart.I have mutations in both copies of my CNGA3 gene.The vision in one eye is much worse than the other by more than 3 lines on an eye chart.I have been diagnosed with achromatopsia.My eye condition is due to severe nearsightedness.I have eye conditions that could worsen with certain treatments.I am 18 years old or older.Your vision in the eye being studied is not better than a certain level, even with glasses or contacts.I am at least 6 years old for Group 3a, or between 4-8 years old for Groups 4a and 6.Your lab test results are within normal range.I am a woman who can have children and have a recent negative pregnancy test.
Research Study Groups:
This trial has the following groups:- Group 1: Group 1: 4.0 x 10^10 vg/mL of AGTC-402
- Group 2: Group 2: 1.2 x 10^11 vg/mL of AGTC-402
- Group 3: Group 3: 3.6 x 10^11 vg/mL of AGTC-402
- Group 4: Group 3a: 3.6 x 10^11 vg/mL of AGTC-402
- Group 5: Group 4: 1.1 x 10^12 vg/mL of AGTC-402
- Group 6: Group 4a: 1.1 x 10^12 vg/mL of AGTC-402
- Group 7: Group 5: 3.2 x 10^12 vg/mL of AGTC-402
- Group 8: Group 6: MTD of AGTC-402
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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