Phase 1/2 Clinical Trial of AAV-RPGR
MeiraGTx is currently conducting an open-label, dose-escalation Phase 1/2 clinical trial of AAV-RPGR in adult and pediatric patients with XLRP caused by mutations in RPGR open reading frame 15 (RPGR ORF15). The primary endpoint of this clinical trial is the safety of delivering AAV-RPGR through subretinal injection. Secondary endpoints include the outcomes of a range of functional tests and detailed structural analysis of the retina, including structural analysis of individual photoreceptors.
In 2020, MeiraGTx announced six, nine, and 12-month results from the dose escalation phase of the Phase 1/2 trial, with data demonstrating statistically significant improvement in retinal sensitivity and vision-guided mobility.
MeiraGTx is also conducting an ongoing natural history study in XLRP caused by mutations in RPGR ORF15. The study includes approximately 70 patients who will be followed for up to five years in order to collect structural and functional data.