This clinical trial is designed to test whether a single image-guided intracerebral administration of inhibitory nerve cells, called interneurons (NRTX-1001), into subjects with drug-resistant unilateral mesial temporal lobe epilepsy (MTLE), with or without mesial temporal sclerosis (MTS), is safe (frequency of adverse events) and effective (seizure frequency). NRTX-1001 comprises human interneurons that secrete a neurotransmitter, gamma-aminobutyric acid (GABA).
Male or Female, age ≥18 to ≤75
Focal seizures, clinically defined as unilateral MTLE
Has failed to achieve seizure control despite adequate trials of at least 2 ASDs at appropriate doses
Currently on stable doses (at least 1 month prior to Screening) of approved ASDs
Epilepsy due to other medical conditions and/or progressive neurologic disease
Evidence of seizure focus outside of the hippocampus or evidence of seizures of non- focal origin.
Significant other medical conditions which would impair safe participation
History of status epilepticus in the 3 years prior to screening.
Parallel
NRTX-1001
Biological/VaccineBiological: NRTX-1001 is an experimental neural cell therapy product candidate derived from an allogeneic human embryonic stem cell line. The stem cells were converted into inhibitory nerve cells that produce GABA.
NRTX-1001
Biological/VaccineBiological: NRTX-1001 is an experimental neural cell therapy product candidate derived from an allogeneic human embryonic stem cell line. The stem cells were converted into inhibitory nerve cells that produce GABA.
Sham Comparator
Procedure/SurgerySham Comparator
Frequency of serious or severe AEs (Phase 1/2)
The primary endpoint of the study is the frequency of serious or severe AEs over the period of 1 year after administration.
Change in disabling seizure frequency (Phase 3)
The difference in median percent change from baseline in diary-reported disabling seizure frequency (seizures per 28 days) between subjects that receive NRTX-1001 and those who receive sham treatment.
Responder rate (Phase 3)
The difference in the 50% responder rate, e.g., the difference between the fraction of subjects who receive NRTX-1001 and those that receive sham treatment who experience at least a 50% reduction from baseline in disabling seizure frequency.
Responder rate (Phase 3)
The difference in the 75% responder rate, e.g., the difference between the fraction of subjects who receive NRTX-1001 and those that receive sham treatment who experience at least a 75% reduction from baseline in disabling seizure frequency.
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Neurona Therapeutics
Lead sponsor
California Institute for Regenerative Medicine (CIRM)
Collaborator
