AGTC’s Clarity Clinical Trials are studying gene therapies, which are treatments that replace a mutated gene with a working copy of the same gene. The purpose of our research is to test whether the gene therapies are safe and to see if they can improve vision and other symptoms of achromatopsia.

Right now, we are enrolling volunteers, currently between the ages of 4-8 years old, for two achromatopsia clinical trials. These trials are Phase 1 and Phase 2 studies, which means they are still in the early stages (there are 4 phases of clinical research). In Phases 1 and 2, our goals are simply to make sure the treatment is safe and to see whether it has any positive effect on the vision of people with achromatopsia.

CNGB3 STUDY: https://clinicaltrials.gov/ct2/show/NCT02599922?cond=achromatopsia

CNGA3 STUDY: https://clinicaltrials.gov/ct2/show/NCT02935517?cond=achromatopsia

What happens in the study?

The gene therapy treatment we are studying is given through a one-time injection into the back of the retina of one eye only. The injection is given in a hospital by a retinal surgeon, and requires a 1 to 2 day stay at the surgery clinical site so we can watch for immediate side effects from the surgery. After that, you will have other follow-up visits at the clinical site where you were initially screened (which may be different from the surgical site).

Both of our achromatopsia trials are currently enrolling participants, currently between the ages of 4-8 years old. Our patient partners are a critical part of moving the research forward to help people with achromatopsia. If your child is interested in joining one of our trials and advancing this work, we look forward to hearing from you!

Is this study right for me?

Our achromatopsia trials are enrolling people, between the ages of 4-8 years old, whose achromatopsia is caused by mutations in either the CNGB3 or CNGA3 gene.

Gene mutations can be identified with a simple blood or saliva test. Before your child enrolls in the trial, their gene mutation will be confirmed by their doctor or the trial team. If you don’t know your child’s mutation and want to know how to get tested to see if they are eligible for AGTC’s achromatopsia trial, click here.

Both of these trials are open to anyone currently between the ages of 4-8 years old who has been diagnosed with achromatopsia, without any other health conditions that may make it hard or risky for them to undergo the treatment. There are other, more specific eligibility criteria for each trial that can be discussed with a doctor who is working on the study.

Source: https://achromatopsiatrials.com/about-our-trials/