Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Cli… (NCT02599922) | Clinical Trial Compass
UnknownPhase 1/2
Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial)
United States32 participantsStarted 2016-04-11
Plain-language summary
This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-401 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGB3 gene. The primary study endpoint will be safety and the secondary study endpoint will be efficacy.
Who can participate
Age range
4 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Male or female subjects with documented mutations in both alleles of the CNGB3 gene;
. Retinal disease consistent with a clinical diagnosis of achromatopsia;
. At least 18 years of age for Groups 1, 2, 3, 4, 5 and 6. At least 6 years of age for Group 4a, and 4-8 years of age for Groups 5a and 7;
. Able to perform tests of visual and retinal function;
. Visual acuity in the study eye not better than 55 ETDRS letters (Snellen equivalent 20/80) based on the average of two examinations at the baseline visit;
. Acceptable laboratory parameters;
. For females of childbearing potential: A negative pregnancy test within 2 days before administration of study agent.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this is a Phase 1/2 trial and the primary outcome being measured is adverse events, what does that mean about how much is still unknown regarding whether this gene therapy is safe or effective for my specific situation?
2This trial targets CNGB3 achromatopsia specifically — how would my doctor confirm whether my achromatopsia is caused by a CNGB3 mutation, and does that affect whether this study would even be relevant to discuss with my care team?
3The trial is listed as 'active not recruiting,' which means they're no longer enrolling new patients — are there other ongoing or upcoming gene therapy trials for achromatopsia that might be worth looking into instead?
4Since AAV gene therapy involves a one-time injection that permanently alters cells, what should I understand about the irreversible nature of this approach before considering any similar treatment?
5Are there any current standard-of-care options or low-vision support tools I should be trying now, while gene therapy research for achromatopsia is still in early clinical stages?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.