Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (EPIC (EPIlepsy Cell Therapy)) (EPIC)

March 26, 2026 updated by: Neurona Therapeutics

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

This clinical trial is designed to test whether a single image-guided intracerebral administration of inhibitory nerve cells, called interneurons (NRTX-1001), into subjects with drug-resistant unilateral mesial temporal lobe epilepsy (MTLE), with or without mesial temporal sclerosis (MTS), is safe (frequency of adverse events) and effective (seizure frequency). NRTX-1001 comprises human interneurons that secrete a neurotransmitter, gamma-aminobutyric acid (GABA).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Subjects will undergo a single CT or MRI-guided intracerebral administration of human interneurons that secrete the inhibitory neurotransmitter, gamma-aminobutyric acid (GABA), into the temporal lobe region of the brain where the seizures are thought to arise. NRTX-1001 is intended to suppress the onset and spread of seizures. Safety, efficacy, tolerability, and effects on reducing seizure frequency and epilepsy disease symptoms will be assessed at regular 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 5, and then annual visits in years 6 through 15. Subjects will be placed on an immunosuppressant medication regimen for a duration of one year to partially suppress the subjects' immune system to promote the intended long-term persistence of NRTX-1001. This immunosuppressant medication is intended to be discontinued after the first year; however, the NRTX-1001 cells are intended to persist long-term.

Study Type

Interventional

Enrollment (Estimated)

88

Phase

  • Phase 3

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
    • Arizona
      • Phoenix, Arizona, United States, 85054
        • Recruiting
        • Mayo Clinic Arizona Epilepsy Center
        • Contact:
        • Principal Investigator:
          • Jonathon J Parker, MD, PhD
      • Tucson, Arizona, United States, 85724-5023
        • Recruiting
        • Banner-University of Arizona Medical Center Tucson Comprehensive Epilepsy Program
        • Principal Investigator:
          • David Labiner, MD
        • Contact:
    • Arkansas
      • Little Rock, Arkansas, United States, 72205
        • Recruiting
        • University of Arkansas for Medical Sciences
        • Principal Investigator:
          • Sisira Yadala, MD
        • Contact:
    • California
      • Los Angeles, California, United States, 90095
        • Recruiting
        • University of California Los Angeles
        • Principal Investigator:
          • John M Stern, MD
        • Contact:
      • Los Angeles, California, United States, 90033
        • Recruiting
        • University of Southern California Keck Hospital
        • Principal Investigator:
          • Charles Y Liu, MD, PhD
        • Contact:
      • Orange, California, United States, 92868
        • Recruiting
        • UC Irvine Medical Center
        • Contact:
        • Principal Investigator:
          • Mona Sazgar, MD
      • Palo Alto, California, United States, 94304
        • Recruiting
        • Stanford University
        • Contact:
        • Principal Investigator:
          • Kevin Graber, MD
      • Sacramento, California, United States, 95817
        • Recruiting
        • University of California Davis
        • Contact:
        • Principal Investigator:
          • Jack J Lin, 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
        • Recruiting
        • University of California San Francisco
        • Principal Investigator:
          • Robert C Knowlton, MD
        • Contact:
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • University of Colorado Anschutz Medical Campus
        • Contact:
        • Principal Investigator:
          • Lesley Kaye, MD
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20037
        • Recruiting
        • George Washington University
        • Contact:
        • Principal Investigator:
          • Mohamad Koubeissi, MD
    • Florida
      • Miami, Florida, United States, 33136
        • Recruiting
        • University of Miami
        • Principal Investigator:
          • Jonathan R. Jagid, MD
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Recruiting
        • Rush University Medical Center
        • Contact:
        • Principal Investigator:
          • Rebecca O'Dwyer, MD
      • 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
    • Louisiana
      • New Orleans, Louisiana, United States, 70121
        • Recruiting
        • Ochsner Clinic Foundation
        • Contact:
        • Principal Investigator:
          • Fawad Khan, MD
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Recruiting
        • Beth Israel Deaconess Medical Center
        • Contact:
        • Principal Investigator:
          • Joshua P Aronson, MD
    • Michigan
      • Detroit, Michigan, United States, 48201
        • Recruiting
        • Wayne State University/Detroit Medical Center Comprehensive Epilepsy Program
        • Principal Investigator:
          • Maysaa Basha, MD
        • Contact:
    • New Hampshire
      • Lebanon, New Hampshire, United States, 03756
        • Recruiting
        • Dartmouth Hitchcock Medical Center/ Department of Neurology
        • Contact:
        • Principal Investigator:
          • Krzysztof A, Bujarski, MD
    • New York
      • Great Neck, New York, United States, 11021
        • Recruiting
        • Hofstra Northwell Comprehensive Epilepsy Center
        • Contact:
        • Principal Investigator:
          • Sean Hwang, MD
      • New York, New York, United States, 10016
        • Recruiting
        • NYU Langone Comprehensive Epilepsy Center
        • Principal Investigator:
          • Orrin Devinsky, MD
        • Contact:
      • Syracuse, New York, United States, 13210
        • Recruiting
        • SUNY Upstate Medical University
        • Contact:
        • Principal Investigator:
          • Robert L Beach, MD, PhD
    • North Carolina
      • Charlotte, North Carolina, United States, 28204
        • Recruiting
        • Atrium Health
        • Principal Investigator:
          • Rajdeep Singh, MD
        • Contact:
      • 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
        • Atrium Wake Forest Baptist
        • Contact:
        • Principal Investigator:
          • Gautam S Popli, MBBS, MBA
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Oregon Health and Science University
        • Principal Investigator:
          • David C Spencer, MD
        • Contact:
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19107
        • Recruiting
        • Thomas Jefferson University Hospital
        • Principal Investigator:
          • Michael Sperling, MD
        • Contact:
    • Texas
      • Dallas, Texas, United States, 75390
        • Recruiting
        • UT Southwestern Medical Center
        • Contact:
        • Principal Investigator:
          • Sasha Alick-Lindstrom, MD
      • Houston, Texas, United States, 77030
        • Recruiting
        • UTHealth Houston
        • Contact:
        • Principal Investigator:
          • Nitin Tandon, MD
    • Utah
      • Salt Lake City, Utah, United States, 84108
        • Recruiting
        • University of Utah Health
        • Principal Investigator:
          • Amir Arain, MD
        • Contact:
    • Washington
      • Seattle, Washington, United States, 98104
        • Recruiting
        • University of Washington Regional Epilepsy Center
        • Contact:
        • Principal Investigator:
          • Shahin Hakimian, MD, PhD
    • 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

18 years to 55 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

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: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: NRTX-1001 (Phase 1/2)
Up to 28 subjects
Biological: NRTX-1001
Biological: NRTX-1001 is an experimental neural cell therapy product candidate derived from an allogeneic human embryonic stem cell line. The stem cells were converted into inhibitory nerve cells that produce GABA.
Other Names:
  • GABA-secreting interneurons
Experimental: NRTX-1001 (Phase 3)
Approximately 40 subjects
Biological: NRTX-1001
Biological: NRTX-1001 is an experimental neural cell therapy product candidate derived from an allogeneic human embryonic stem cell line. The stem cells were converted into inhibitory nerve cells that produce GABA.
Other Names:
  • GABA-secreting interneurons
Sham Comparator: Sham Comparator (Phase 3)
Approximately 20 subjects
Procedure: Sham Comparator
Sham Comparator

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Frequency of serious or severe AEs (Phase 1/2)
Time Frame: 1 year
The primary endpoint of the study is the frequency of serious or severe AEs over the period of 1 year after administration.
1 year
Change in disabling seizure frequency (Phase 3)
Time Frame: 4-6 months after surgery
The difference in median percent change from baseline in diary-reported disabling seizure frequency (seizures per 28 days) between subjects that receive NRTX-1001 and those who receive sham treatment.
4-6 months after surgery

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Responder rate (Phase 3)
Time Frame: 4-6 months after surgery
The difference in the 50% responder rate, e.g., the difference between the fraction of subjects who receive NRTX-1001 and those that receive sham treatment who experience at least a 50% reduction from baseline in disabling seizure frequency.
4-6 months after surgery
Responder rate (Phase 3)
Time Frame: 4-6 months after surgery
The difference in the 75% responder rate, e.g., the difference between the fraction of subjects who receive NRTX-1001 and those that receive sham treatment who experience at least a 75% reduction from baseline in disabling seizure frequency.
4-6 months after surgery

Collaborators and Investigators

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

Sponsor

Neurona Therapeutics

Collaborators

California Institute for Regenerative Medicine (CIRM)

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.

General Publications

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)

June 16, 2022

Primary Completion (Estimated)

February 1, 2029

Study Completion (Estimated)

September 1, 2043

Study Registration Dates

First Submitted

November 16, 2021

First Submitted That Met QC Criteria

November 16, 2021

First Posted (Actual)

November 26, 2021

Study Record Updates

Last Update Posted (Actual)

March 30, 2026

Last Update Submitted That Met QC Criteria

March 26, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NTE001

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.

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