FIH Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy

About

Brief Summary

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).

Primary Purpose

Treatment

Study Type

Interventional

Phase

Phase 1/Phase 2

Eligibility

Gender

All

Healthy Volunteers

No

Minimum Age

18 Years

Maximum Age

75 Years

Key Inclusion Criteria:

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) of approved ASDs
Single seizure focus confirmed within one hippocampus
Seizure frequency averages ≥4 per 28-day period, including at least 2 clinical focal seizures per 28-day period with objective manifestations or more severe types, over the 6 months prior to the Screening Visit.
Considered (by Investigator) to be a candidate for temporal lobectomy (TL) or Laser Interstitial Thermal Therapy (LITT) following evaluation at a qualified epilepsy surgery program (National Association of Epilepsy Centers [NAEC] Level 4).

Key Exclusion Criteria:

Epilepsy due to other 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 year prior to screening. A history of cluster seizures is permitted.
Primary or secondary immunodeficiency
Suicide attempts in the past year
Severe psychiatric disorders
Prior lobectomy or LITT procedure
MRI indicating potential malignant lesion
Pregnancy, or currently breastfeeding.