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Neuronal cell therapy for drug-resistant epilepsy

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

Phase1; Phase2 Interventional Neurona Therapeutics · NCT06422923

This study is testing a new treatment that involves giving special nerve cells to people with hard-to-treat epilepsy to see if it can help reduce their seizures.

Quick facts

PhasePhase1; Phase2
Study typeInterventional
Enrollment10 (estimated)
Ages18 Years to 75 Years
SexAll
SponsorNeurona Therapeutics Industry-sponsored
Locations17 sites (Little Rock, Arkansas and 16 other locations)
Trial IDNCT06422923 on ClinicalTrials.gov

What this trial studies

This multicenter, single-arm, open-label clinical trial evaluates the safety and preliminary efficacy of NRTX-1001, a treatment involving the administration of inhibitory nerve cells into the temporal lobes of patients with drug-resistant bilateral mesial temporal lobe epilepsy (MTLE). Participants will receive a single stereotactic CT or MRI-guided intracerebral administration of these human interneurons, which secrete GABA to help suppress seizures. The study will monitor safety, tolerability, and seizure frequency over a two-year period, with follow-up assessments continuing for up to 15 years.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18-75 with a confirmed diagnosis of drug-resistant bilateral mesial temporal lobe epilepsy.

Not a fit: Patients with epilepsy that is not drug-resistant or those with other types of epilepsy may not benefit from this treatment.

Why it matters

Potential benefit: If successful, this therapy could significantly reduce seizure frequency and improve quality of life for patients with drug-resistant epilepsy.

How similar studies have performed: While this approach is innovative, similar studies using neuronal cell therapies for epilepsy have shown promise, indicating potential for success.

Eligibility criteria

Show full inclusion / exclusion criteria 
Key Inclusion Criteria:

1. Male or female, age 18-75 years.
2. Subjects of childbearing potential will use highly effective contraception.
3. Proven history of focal seizures of hippocampal origin with bilateral seizure foci confirmed by scalp or intracranial ictal EEG (including confirmation by recordings from responsive neurostimulation \[RNS\] electrodes when applicable).
4. Either

   1. bilateral hippocampal sclerosis on MRI (evidenced by increased FLAIR signal intensity in both hippocampi or by visual assessment showing reduced volume compared to normal) or
   2. bilateral temporal hypometabolism on 18-Flourodeoxyglucose Positron Emission Tomography (FDG PET) (assessed by visual assessment, comparing temporal regions to frontal/parietal lobe neocortex). In this case, ictal EEG recordings must also include intracranial confirmation.

      or
   3. a combination of unilateral instances of the evidence described in a. and b. (e.g., one side can be evidenced by criterion a. and the other side by criterion b.) MRI or PET scans used for assessment must have been acquired within 3 years of screening.
5. Subject has had at least four clinical focal seizures, including at least two clinical focal seizures with objective manifestations, on average, per month for the 6 months prior to screening.
6. Subject has previously had adequate (in opinion of investigator) therapeutic trials of at least two Anti-Seizure Medicines (ASMs).
7. Current ASM regimen, and doses of other drugs known to affect seizure frequency (e.g., antidepressants), have been stable for at least three months prior to enrollment.
8. Subject can converse and read in English or Spanish. Able to participate in required study procedures and provide signed informed consent.

Key Exclusion Criteria:

1. Epilepsy due to other and/or progressive neurologic disease.
2. Evidence of seizure focus outside hippocampus (either by seizure semiology or EEG findings).
3. MRI indicating potential malignant lesion (low-grade glioma of any type is excluded) in any location or non-malignant potentially epileptogenic lesion outside the hippocampus. Small (\<2 cm) non invasive meningioma, remote from the affected temporal lobe, is not exclusionary.
4. Seizures of non-focal origin.
5. History of status epilepticus in the year prior to screening, as guided by ILAE criteria (Trinka 2015) in the judgement of the PI. A history of cluster seizures is permitted.
6. Psychogenic Non-Epileptic Seizures (PNES) within the past 3 years.
7. Severe psychiatric disorders.
8. Primary or secondary immunodeficiency.
9. Pregnancy, or currently breastfeeding.
10. Suicide attempts in past year.
11. Significant other medical conditions which would impair safe participation.

Where this trial is running

Little Rock, Arkansas and 16 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Epilepsy, Temporal LobeBilateral Temporal Lobe EpilepsyBilateral MTLERefractory Bilateral MTLE
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.