Phase 1/2 Clinical Trial of AAV-RPE65
An open label, dose escalation Phase 1/2 clinical trial of AAV-RPE65 in adult and pediatric patients with RPE65-deficiency is currently ongoing. The primary endpoint of this clinical trial is the safety of delivering AAV-RPE65 through subretinal injection. Secondary endpoints include the outcomes of a range of functional tests and detailed structural analysis of the retina.
MeiraGTx is also conducting an ongoing natural history study of patients with RPE65 deficiency in order to collect structural and function data.