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

January 5, 2026 updated by: Neurona Therapeutics

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

This is a multicenter, single arm, open label clinical trial that is designed to test the safety and preliminary efficacy of single administration inhibitory nerve cells called interneurons (NRTX-1001), into both temporal lobes of subjects with drug-resistant bilateral mesial temporal lobe epilepsy.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

This is a multicenter, single arm, open-label study of NRTX-1001 in subjects with drug-resistant bilateral MTLE, with the objective of evaluating safety and preliminary efficacy in reducing seizure frequency. The subjects will undergo a single stereotactic CT or MRI-guided intracerebral administration of human interneurons into both temporal lobe regions of the brain. NRTX-1001 secretes the inhibitory neurotransmitter gamma-aminobutyric acid (GABA), which is intended to suppress the onset and spread of the seizures. Safety, tolerability, and effects on epilepsy disease symptoms will be assessed at approximately quarterly intervals for 2 years after the administration of NRTX-1001. After the two-year period, subjects will be followed with quarterly phone calls and annual visits in years 3 through 15.

Study Type

Interventional

Enrollment (Estimated)

10

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72205
        • Recruiting
        • University of Arkansas for Medical Sciences
        • Contact:
        • Principal Investigator:
          • Sisira Yadala, MD
    • California
      • Los Angeles, California, United States, 90033
        • Recruiting
        • University of Southern California Keck Hospital
        • Contact:
        • Principal Investigator:
          • Charles Y Liu, MD, PhD
      • Orange, California, United States, 92868
        • Recruiting
        • UC Irvine Medical Center
        • Principal Investigator:
          • Mona Sazgar, MD
        • Contact:
          • UCI Alpha Clinic
          • Phone Number: 949-824-3990
      • Palo Alto, California, United States, 94304
        • Recruiting
        • Stanford University
        • Contact:
        • Principal Investigator:
          • Kevin Graber, MD
      • San Diego, California, United States, 92037
        • Recruiting
        • University of California San Diego
        • Principal Investigator:
          • Jerry J Shih, MD
        • Contact:
      • San Francisco, California, United States, 94143
        • Withdrawn
        • University of California San Francisco
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • University of Colorado Anschutz Medical Campus
        • Principal Investigator:
          • Steven Ojemann, MD
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60637
        • Recruiting
        • University of Chicago
        • Contact:
        • Principal Investigator:
          • Peter Warnke, MD
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Recruiting
        • University of Iowa Health Care
        • Contact:
        • Principal Investigator:
          • Mark Granner, MD
    • New York
      • Syracuse, New York, United States, 13210
        • Recruiting
        • SUNY Upstate Medical University
        • Contact:
        • Principal Investigator:
          • Shahram Izadyar, MD
    • North Carolina
      • Charlotte, North Carolina, United States, 28204
        • Recruiting
        • Atrium Health
        • Contact:
        • Principal Investigator:
          • Rajdeep Singh, MD
      • Durham, North Carolina, United States, 27710
        • Recruiting
        • Duke University Hospital
        • Principal Investigator:
          • Derek Southwell, MD, PhD
        • Contact:
      • Winston-Salem, North Carolina, United States, 27157
        • Recruiting
        • Wake Forest Baptist Medical Center
        • Principal Investigator:
          • Gautam S Popli, MD, MBA
        • Contact:
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Oregon Health and Science University
        • Principal Investigator:
          • David C Spencer, MD
        • Contact:
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • UTHealth Houston
        • Contact:
        • Principal Investigator:
          • Nitin Tandon, MD
    • Virginia
      • Charlottesville, Virginia, United States, 22903
        • Recruiting
        • UVA Health University Medical Center
        • Principal Investigator:
          • Nathan Fountain, MD
        • Contact:
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Recruiting
        • Medical College of Wisconsin
        • Principal Investigator:
          • Sean Lew, MD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Treatment Arm
This is an open-label study of the bilateral intrahippocampal administration of NRTX-1001 in a single dose cohort of up to 10 subjects with drug-resistant bilateral MTLE.
Biological: NRTX-1001
Biological: NRTX-1001 is an inhibitory neural cell therapy investigational product. It is derived from a human stem cell line that has been converted into high-purity inhibitory interneurons that produce GABA. NRTX-1001 is intended to persist long-term and not require repeated administration.
Other Names:
  • GABA-secreting interneurons

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of Serious Adverse Events at end of month 12
Time Frame: 12 months after treatment
12 months after treatment

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in frequency of clinical seizures
Time Frame: 12 months after treatment
Seizure frequency change during months 7-12, compared to frequency of clinical seizures in the 6-month baseline period.
12 months after treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Neurona Therapeutics

Investigators

  • Study Director: Eduardo Dunayevich, MD, Neurona Therapeutics

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 14, 2024

Primary Completion (Estimated)

June 15, 2027

Study Completion (Estimated)

June 15, 2042

Study Registration Dates

First Submitted

May 15, 2024

First Submitted That Met QC Criteria

May 15, 2024

First Posted (Actual)

May 21, 2024

Study Record Updates

Last Update Posted (Actual)

January 7, 2026

Last Update Submitted That Met QC Criteria

January 5, 2026

Last Verified

January 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NTE002

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Epilepsy, Temporal Lobe

Clinical Trials on NRTX-1001

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Czech Republic

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe