Phase 12 Study of Single-Dose NRTX-1001 Neuronal Cell Therapy for Adults with Drug-Resistant Bilateral Temporal Lobe Epilepsy

What this Trial Is About

Researchers are evaluating the safety and early effects of a new neuronal cell therapy called NRTX-1001 for adults with drug-resistant bilateral mesial temporal lobe epilepsy MTLE. This open-label, single-arm study involves up to 10 subjects and aims to reduce seizure frequency by targeting both temporal lobes in patients whose seizures have not improved with medication. The investigational treatment uses inhibitory nerve cells called interneurons to potentially suppress seizure activity by releasing a natural brain chemical called GABA. Participants will receive a single administration of NRTX-1001 through a stereotactic CT or MRI-guided injection directly into both temporal lobes of the brain. The therapy is designed to persist long-term, so repeated doses are not planned. This phase 12 trial observes subjects closely for safety and seizure changes over two years after treatment. Following this period, participants will continue with less frequent follow-up phone calls every three months and yearly visits through year 15 to monitor long-term outcomes. During the study, participants will undergo evaluations approximately every three months for two years, including clinical assessments and seizure monitoring. Safety and tolerability will be specifically tracked, with the main outcome being the rate of serious adverse events at 12 months post-treatment. Seizure frequency changes will also be measured as a secondary outcome. After the initial two years, ongoing safety and health status will be followed through scheduled calls and visits for up to 15 years, emphasizing long-term monitoring and support.

A Phase 1/2 Study of NRTX-1001 Neuronal Cell Therapy in Drug-Resistant Bilateral Mesial Temporal Lobe Epilepsy (MTLE)