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

June 6, 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

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
88

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • Recruiting
        • University of Alabama
        • Contact:
        • Principal Investigator:
          • Jerzy Szaflarski, MD, PhD
    • 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:
    • Missouri
      • St Louis, Missouri, United States, 63100
        • Recruiting
        • Washington University at St. Louis
        • Contact:
        • Principal Investigator:
          • R. Edward Hogan, MD
    • 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Neurona Therapeutics

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
California Institute for Regenerative Medicine (CIRM)

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 16, 2022

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 1, 2029

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 1, 2043

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 16, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 16, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 26, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 9, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 6, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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