Home › Trials › NCT05135091

Neural cell therapy for drug-resistant epilepsy

A Study of Inhibitory Interneurons (NRTX-1001) in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (MTLE)

PHASE3 · Neurona Therapeutics · NCT05135091

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

Quick facts

PhasePHASE3
Study typeInterventional
Enrollment88 (estimated)
Ages18 Years to 75 Years
SexAll
SponsorNeurona Therapeutics (industry)
Locations33 sites (Phoenix, Arizona and 32 other locations)
Trial IDNCT05135091 on ClinicalTrials.gov

What this trial studies

This clinical trial tests the safety and effectiveness of NRTX-1001, a therapy involving the administration of inhibitory nerve cells into the brain of patients with drug-resistant unilateral mesial temporal lobe epilepsy (MTLE). Participants will receive a single image-guided intracerebral injection of human interneurons that secrete GABA, aimed at reducing seizure frequency. The study will monitor participants for safety and seizure control over a two-year period, followed by long-term follow-up. An immunosuppressant regimen will be used to enhance the persistence of the injected cells.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 to 75 with unilateral MTLE who have not achieved seizure control despite trials of at least two anti-seizure medications.

Not a fit: Patients with epilepsy due to other neurological diseases or those with seizure foci outside the hippocampus may not benefit from this therapy.

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 novel, other studies involving neural cell therapies for epilepsy have shown promising results.

Eligibility criteria

Show full inclusion / exclusion criteria 
Key Inclusion Criteria:

1. Male or Female, age ≥18 to ≤75
2. Focal seizures, clinically defined as unilateral MTLE
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 prior to Screening) of approved ASDs
5. Single seizure focus confirmed within one hippocampus
6. 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. (Phase 1/2 only)
7. Disabling seizure frequency of ≥2 per 28-day period averaged over 3 months prior to the Screening visit and over a prospective 10-week baseline period prior to Randomization (Phase 3 Only)

Key Exclusion Criteria:

1. Epilepsy due to other medical conditions and/or progressive neurologic disease
2. Evidence of seizure focus outside of the hippocampus or evidence of seizures of non- focal origin.
3. Significant other medical conditions which would impair safe participation
4. History of status epilepticus in the 3 years prior to screening.
5. Primary or secondary immunodeficiency
6. Suicide attempts in the past year 3 years
7. Severe psychiatric disorders
8. Prior lobectomy or LITT procedure
9. MRI indicating potential malignant lesion
10. Pregnancy, or currently breastfeeding.

Where this trial is running

Phoenix, Arizona and 32 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 →

Conditions: Mesial Temporal Lobe Epilepsy, Epilepsy, MTLE, seizure, EPIC, focal seizures, hippocampus

Last reviewed 2026-05-15 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.