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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
At least 18 years of age for Groups 1, 2, 3, 4, 5 and 6
Male or female subjects with documented mutations in both alleles of the CNGB3 gene
Must not have
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights
No Placebo-Only Group

Summary

This trial will test a new gene therapy for achromatopsia, a condition that causes color blindness. The goal is to see if the therapy is safe and effective.

Who is the study for?
This trial is for individuals at least 18 years old (or as young as 6 for certain groups) with achromatopsia due to CNGB3 gene mutations. They should have visual acuity not better than 20/80 in the study eye and be able to perform vision tests. Women must test negative for pregnancy. Those with significant vision differences between eyes or other eye conditions that could affect results are excluded.
What is being tested?
The trial is testing a new gene therapy called rAAV2tYF-PR1.7-hCNGB3, given through an injection into one eye of patients with color blindness caused by CNGB3 mutations. It's an open-label, non-randomized Phase 1/2 study focusing on safety first and then how well it works.
What are the potential side effects?
As this is a Phase 1/2 study primarily assessing safety, potential side effects may include typical risks associated with subretinal injections such as inflammation, infection risk, retinal detachment or damage at the injection site.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 18 years old or older.
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I have mutations in both copies of my CNGB3 gene.
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I have been diagnosed with achromatopsia.
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I am between 4 and 8 years old.
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I am at least 6 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My eye condition is due to severe nearsightedness.
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I have eye conditions that could worsen with certain treatments.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Adverse events
Secondary study objectives
Color vision
Light aversion
Visual acuity

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

9Treatment groups
Experimental Treatment
Group I: Group 7: MTD of AGTC-401Experimental Treatment1 Intervention
Subjects 4 to 8 y/o treated with a maximum tolerated dose of rAAV2tYF-PR1/7-hCNGB3 study drug determined by Groups 1-6.
Group II: Group 6: 3.2 x 10^12 vg/mL of AGTC-401Experimental Treatment1 Intervention
Subjects at least 18 y/o treated with 3.2 x 10\^12 vg/mL of rAAV2tYF-PR1/7-hCNGB3 study drug.
Group III: Group 5a: 1.1 x 10^12 vg/mL of AGTC-401Experimental Treatment1 Intervention
Subjects 4 to 8 y/o treated with 1.1 x 10\^12 vg/mL of rAAV2tYF-PR1/7-hCNGB3 study drug.
Group IV: Group 5: 1.1 x 10^12 vg/mL of AGTC-401Experimental Treatment1 Intervention
Subjects at least 18 y/o treated with 1.1 x 10\^12 vg/mL of rAAV2tYF-PR1/7-hCNGB3 study drug.
Group V: Group 4a: 3.6 x 10^11 vg/mL of AGTC-401Experimental Treatment1 Intervention
Subjects 6 to 17 y/o treated with 3.6 x 10\^11 vg/mL of rAAV2tYF-PR1/7-hCNGB3 study drug.
Group VI: Group 4: 3.6 x 10^11 vg/mL of AGTC-401Experimental Treatment1 Intervention
Subjects at least 18 y/o treated with 3.6 x 10\^11 vg/mL of rAAV2tYF-PR1/7-hCNGB3 study drug.
Group VII: Group 3: 1.2 x 10^11 vg/mL of AGTC-401Experimental Treatment1 Intervention
Subjects at least 18 y/o treated with 1.2 x 10\^11 vg/mL of rAAV2tYF-PR1/7-hCNGB3 study drug.
Group VIII: Group 2: 4.0 x 10^10 vg/mL of AGTC-401Experimental Treatment1 Intervention
Subjects at least 18 y/o treated with 4.0 x 10\^10 vg/mL of rAAV2tYF-PR1/7-hCNGB3 study drug.
Group IX: Group 1: 2.0 x 10^11 vg/mL of AGTC-401Experimental Treatment1 Intervention
Subjects at least 18 y/o treated with 2.0 x 10\^11 vg/mL of rAAV2tYF-PR1/7-hCNGB3 study drug.

Find a Location

Who is running the clinical trial?

Applied Genetic Technologies CorpLead Sponsor
10 Previous Clinical Trials
321 Total Patients Enrolled
National Eye Institute (NEI)NIH
554 Previous Clinical Trials
1,407,300 Total Patients Enrolled
1 Trials studying Color Blindness
35 Patients Enrolled for Color Blindness
Matt Feinsod, MDStudy DirectorApplied Genetics Technologies Corporation
3 Previous Clinical Trials
146 Total Patients Enrolled

Media Library

Color Blindness Research Study Groups: Group 5: 1.1 x 10^12 vg/mL of AGTC-401, Group 4: 3.6 x 10^11 vg/mL of AGTC-401, Group 5a: 1.1 x 10^12 vg/mL of AGTC-401, Group 4a: 3.6 x 10^11 vg/mL of AGTC-401, Group 1: 2.0 x 10^11 vg/mL of AGTC-401, Group 2: 4.0 x 10^10 vg/mL of AGTC-401, Group 3: 1.2 x 10^11 vg/mL of AGTC-401, Group 6: 3.2 x 10^12 vg/mL of AGTC-401, Group 7: MTD of AGTC-401
Color Blindness Clinical Trial 2023: AGTC-401 Highlights & Side Effects. Trial Name: NCT02599922 — Phase 1 & 2
~3 spots leftby Nov 2025
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