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

Overview

This clinical trial is designed to test whether a single stereotactic intracerebral administration of inhibitory nerve cells into subjects with drug-resistant mesial temporal lobe epilepsy is safe (frequency of adverse events) and effective (seizure frequency).

Description

Subjects will undergo a single stereotactic intracerebral administration of neural cells, called interneurons, that secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA).

Subjects will then take medicines to partially suppress their immune system (aimed to prevent the body from rejecting the cells) for 1 year. Safety, tolerability, evidence of neural cell viability and local inflammation (using MRI scans of the brain), and effects on epilepsy disease symptoms will be assessed for 2 years post-transplant. Subjects will be followed for an additional 13 years with quarterly phone contact and annual visits.

Eligibility

Key Inclusion Criteria:

1. Male or Female, age ≥18 to 65
2. Focal seizures, clinically defined as TLE
3. Has failed to achieve seizure control despite adequate trials of at least 2 ASDs at appropriate doses
4. Currently on stable doses (at least 1 month) of approved ASDs
5. Single seizure focus confirmed as within one temporal lobe
6. For subjects entering Stage 1, the seizure focus is either a) in the non-dominant hemisphere, or b) in the dominant hemisphere and the subject has a Rey Auditory Verbal Learning Test (RAVLT) assessed within one year of screening, or at the screening visit, that is at least 1.5 standard deviations below the population mean.
7. Seizure frequency averages ≥2 per 28-day period over the 6 months prior to screening.

Key Exclusion Criteria:

1. Epilepsy due to other and/or progressive neurologic disease
2. Significant other medical condition which would impair safe participation
3. Primary or secondary immunodeficiency
4. Suicide attempt in the past year
5. Severe psychiatric disorders
6. Chronic indwelling intracranial device
7. MRI indicating potential malignant lesion
8. Pregnancy, or currently breastfeeding