Safety and Efficacy of a Bilateral Single Subretinal Injection of rAAV.hCNGA3 in Adult and Minor Patients With CNGA3-linked Achromatopsia Investigated in a Randomized, Wait List Controlled, Observer-masked Trial
The purpose of this study is to proof the safety and efficacy of a single bilateral subretinal injection of rAAV.hCNGA3 in adult and minor patients with CNGA3-linked achromatopsia.
• clinical diagnosis of achromatopsia
• 6-12 years of age
• confirmed mutation in CNGA3
• BCVA ≥ 20/400
• a minimal outer nuclear layer thickness of 10µm at 3° eccentricity (normal = 38±6µm)
• ability to understand and willingness to consent to study protocol
• no infection with Human Immundeficiency Virus (HIV)
• Male patients must agree to use condoms during the first 6 months post treatment.
• Female patients of childbearing potential must agree to use an effective method of birth control during the first 6 months post treatment.
• negative pregnancy test in women with childbearing potential (a woman who is two years post-menopausal or surgically sterile is not considered to be of childbearing potential)