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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year

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

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

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.

0 / 5Eligibility criteria met

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

546 Previous Clinical Trials

1,401,896 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 4: 3.6 x 10^11 vg/mL of AGTC-401, Group 4a: 3.6 x 10^11 vg/mL of AGTC-401, Group 5: 1.1 x 10^12 vg/mL of AGTC-401, Group 5a: 1.1 x 10^12 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is enrollment into this trial open to participants at present?

"As per the information on clinicaltrials.gov, this trial is not currently accepting applicants even though it was last updated in July 20th 2022. Nonetheless, there are seven other medical trials that are actively seeking volunteers right now."

Answered by AI

How many locations are engaging in this research project?

"Patients for this research endeavour are being enrolled at the Cincinnati Eye Institute in Ohio, Baylor College of Medicine in Texas, and University of Wisconsin's McPherson Eye Research Institute. Additionally, 11 other sites are participating throughout the United States."

Answered by AI