Neuronal cell therapy for drug-resistant epilepsy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• University of Arkansas for Medical Sciences
  Little Rock, Arkansas, United States Recruiting
• University of Southern California Keck Hospital
  Los Angeles, California, United States Recruiting
• UC Irvine Medical Center
  Orange, California, United States Recruiting
• Stanford University
  Palo Alto, California, United States Recruiting
• University of California San Diego
  San Diego, California, United States Recruiting
• University of Colorado Anschutz Medical Campus
  Aurora, Colorado, United States Recruiting

Study contacts

• Study coordinator: Neurona MedInfo
• Email: neuronamedinfo@neuronatx.com
• Phone: 650-580-3825

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.