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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 below

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I have mutations in both copies of my CNGA3 gene.

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I have mutations in both copies of my CNGA3 gene.

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I have been diagnosed with achromatopsia.

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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

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

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.

0 / 5Eligibility criteria met

Find a Location

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

AGTC-402 (Virus Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT02935517 — Phase 1 & 2

Color Blindness Research Study Groups: 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

Color Blindness Clinical Trial 2023: AGTC-402 Highlights & Side Effects. Trial Name: NCT02935517 — Phase 1 & 2

AGTC-402 (Virus Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02935517 — 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 the recruitment phase of this study still open?

"Although it was initially published on August 3rd 2017 and received its last update on July 20th 2022, clinicaltrials.gov states that this medical study has concluded patient recruitment. However, there are 7 other trials still in the process of gathering participants."

Answered by AI

How widespread is the implementation of this research endeavor in U.S.-based medical facilities?

"Presently, this medical trial is taking place in 9 different sites. These locations are split between Houston, Boston and San Francisco with other 6 cities being involved too. It would be wise for prospective participants to pick the closest one to them in order to avoid any unnecessary travel."

Answered by AI