Phase 1/2 Clinical Trial of AAV-CNGA3
MeiraGTx is currently conducting an open-label, dose-escalation Phase 1/2 clinical trial of AAV-CNGA3 in children aged 3-15 years old with achromatopsia (ACHM) due to mutations in the CNGA3 gene. The primary endpoint of this clinical trial is the safety of delivering AAV-CNGA3 via subretinal administration. Secondary endpoints include the outcomes of a range of functional tests and detailed structural analysis of the retina, including structural analysis of individual photoreceptors.
MeiraGTx is also conducting an ongoing natural history study following over 90 patients with ACHM for up to five years to collect data on the structure and function of their eye.