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Mesial Temporal Lobe EpilepsyPhase 3EPICNCT05135091

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

A Phase 3 study testing NRTX-1001 in people with Mesial Temporal Lobe Epilepsy, run by Neurona Therapeutics at 36 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

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… Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Mesial Temporal Lobe Epilepsy
What is being tested?
NRTX-1001
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
88 participants
Where
36 sitesUnited States
Study dates
16 Jun 2022to Sep 2043
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Male or Female, age ≥18 to ≤75
  • Focal seizures, clinically defined as unilateral MTLE
  • Has failed to achieve seizure control despite adequate trials of at least 2 ASDs at appropriate doses
  • Currently on stable doses (at least 1 month prior to Screening) of approved ASDs
  • Single seizure focus confirmed within one hippocampus
  • 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)
  • 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)

Who cannot join

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

Trial locations and contacts

36 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Neurona MedInfo contact

650-580-3825

neuronamedinfo@neuronatx.com

Eduardo Dunayevich, MD contact

650-436-3045

edunayevich@neuronatx.com

36 sites in 1 country

United States36 sites, 36 recruiting

  • University of Colorado Anschutz Medical Campus

    Aurora, Colorado, 80045

    Trey Jouard, MS, 970-817-4272, Trey.Jouard@cuanschutz.edu

    Lesley Kaye, MD

    Recruiting

  • University of Alabama

    Birmingham, Alabama, 35294

    Jennifer Pilkington, SC, jlpilkington@uabmc.edu

    Jerzy Szaflarski, MD, PhD

    Recruiting

  • Beth Israel Deaconess Medical Center

    Boston, Massachusetts, 02215

    Anna George, MSc, 617-632-7031, ageorge5@bidmc.harvard.edu

    Joshua P Aronson, MD

    Recruiting

  • Kaleida Health

    Buffalo, New York, 14202

    Eric Miller, emiller@ubns.com

    Arie Weinstock, MD

    Recruiting

  • Atrium Health

    Charlotte, North Carolina, 28204

    Dianelis Diaz, RN, BSN, CRC, 704-446-1349, dianelis.diaz@atriumhealth.org

    Rajdeep Singh, MD

    Recruiting

  • Rush University Medical Center

    Chicago, Illinois, 60612

    Amanda Sremac, BS, 312-942-0539, Amanda_C_Sremac@rush.edu

    Rebecca O'Dwyer, MD

    Recruiting

  • University of Chicago

    Chicago, Illinois, 60637

    Agnieszka Stadnik, MS, 773-702-8996, astadnik@bsd.uchicago.edu

    Peter Warnke, MD

    Recruiting

  • UT Southwestern Medical Center

    Dallas, Texas, 75390

    Vamsi Vemireddy, SC, 214-645-8159, vamsidhara.vemireddy@utsouthwestern.edu

    Sasha Alick-Lindstrom, MD

    Recruiting

  • Wayne State University/Detroit Medical Center Comprehensive Epilepsy Program

    Detroit, Michigan, 48201

    Kelly X Jia, MD, 313-966-9407, xjia@med.wayne.edu

    Maysaa Basha, MD

    Recruiting

  • Duke University Hospital

    Durham, North Carolina, 27710

    Hazani Benitez-Rosas, 919-681-4974, Hazani.benitez-rosas@duke.edu

    Derek Southwell, MD, PhD

    Recruiting

  • Hofstra Northwell Comprehensive Epilepsy Center

    Great Neck, New York, 11021

    Isabella Henderson, 212-434-4837, IHenderson@northwell.edu

    Sean Hwang, MD

    Recruiting

  • UTHealth Houston

    Houston, Texas, 77030

    Eliana M Klier, PhD, 713-500-5442, Eliana.Klier@uth.tmc.edu

    Nitin Tandon, MD

    Recruiting

  • University of Iowa Health Care

    Iowa City, Iowa, 52242

    Loraine Brenner, MSN, 319-356-4361, loraine-brenner@uiowa.edu

    Mark Granner, MD

    Recruiting

  • Dartmouth Hitchcock Medical Center/ Department of Neurology

    Lebanon, New Hampshire, 03756

    Anastasia Kanishcheva, PM, 603) 650-0260, anastasia.kanishcheva@hitchcock.org

    Krzysztof A, Bujarski, MD

    Recruiting

  • University of Arkansas for Medical Sciences

    Little Rock, Arkansas, 72205

    Shellah Rogers, Nurse, 501-614-2086, Scrogers2@uams.edu

    Sisira Yadala, MD

    Recruiting

  • University of Southern California Keck Hospital

    Los Angeles, California, 90033

    Zoe B Durkin, SC, 213-631-8487, zoe.durkin@med.usc.edu

    Charles Y Liu, MD, PhD

    Recruiting

  • University of California Los Angeles

    Los Angeles, California, 90095

    Elizabeth Cruz, 310-825-6930, ElizabethCruz@mednet.ucla.edu

    John M Stern, MD

    Recruiting

  • University of Miami

    Miami, Florida, 33136

    Letitia Fisher, 305-775-8582, lfisher@miami.edu

    Jonathan R. Jagid, MD

    Recruiting

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

    Sarah Young, MS, 414-955-0989, scyoung@mcw.edu

    Sean Lew, MD

    Recruiting

  • Ochsner Clinic Foundation

    New Orleans, Louisiana, 70121

    Neydin Osorio, BS, CRC, 504-703-5577, neydin.tejeda@ochsner.org

    Fawad Khan, MD

    Recruiting

  • NYU Langone Comprehensive Epilepsy Center

    New York, New York, 10016

    Jack C Carter, BS, 646-558-0841, jack.carter@nyulangone.org

    Orrin Devinsky, MD

    Recruiting

  • UC Irvine Medical Center

    Orange, California, 92868

    UCI Alpha Clinic, 949-824-3990, alphaclinic@hs.uci.edu

    Mona Sazgar, MD

    Recruiting

  • Stanford University

    Palo Alto, California, 94304

    Jordan Seliger, CRC, MA, 650-460-9260, jseliger@stanford.edu

    Kevin Graber, MD

    Recruiting

  • Thomas Jefferson University Hospital

    Philadelphia, Pennsylvania, 19107

    Latasha Adams, MBS, 215-955-2606, Latasha.adams2@jefferson.edu

    Michael Sperling, MD

    Recruiting

  • Mayo Clinic Arizona Epilepsy Center

    Phoenix, Arizona, 85054

    Kayla N Haeger, BSN, RN, 480-342-5075 ext. 2-5075, Haeger.Kayla@mayo.edu

    Jonathon J Parker, MD, PhD

    Recruiting

  • Oregon Health and Science University

    Portland, Oregon, 97239

    Claire Dorfman, BA, 971-413-9201, dorfman@ohsu.edu

    David C Spencer, MD

    Recruiting

  • University of California Davis

    Sacramento, California, 95817

    Evan Y Shen, BA, 916-734-3009, evshen@ucdavis.edu

    Jack J Lin, MD

    Recruiting

  • University of Utah Health

    Salt Lake City, Utah, 84108

    Laura Beeler, BS, 928-530-7990, laura.beeler@hsc.utah.edu

    Amir Arain, MD

    Recruiting

  • University of California San Diego

    San Diego, California, 92037

    Mateo Santizo, 858-583-0929, msantizo@health.ucsd.edu

    Jerry J Shih, MD

    Recruiting

  • University of California San Francisco

    San Francisco, California, 94143

    Sydnie Beausoleil, 631-428-9688, Sydnie.Beausoleil@ucsf.edu

    Robert C Knowlton, MD

    Recruiting

  • University of Washington Regional Epilepsy Center

    Seattle, Washington, 98104

    Jennifer Steele, CRC, 206-744-2336, recresearch@uw.edu

    Shahin Hakimian, MD, PhD

    Recruiting

  • Washington University at St. Louis

    St Louis, Missouri, 63100

    Victoria Taylor, SC, 314-362-7871, v.taylor@wustl.edu

    R. Edward Hogan, MD

    Recruiting

  • SUNY Upstate Medical University

    Syracuse, New York, 13210

    Lena F Deb, BA, 315-464-9756, debl@upstate.edu

    Robert L Beach, MD, PhD

    Recruiting

  • Banner-University of Arizona Medical Center Tucson Comprehensive Epilepsy Program

    Tucson, Arizona, 85724-5023

    Faten Sebaali, BA/MA, (520) 626-3576, fsebaali@arizona.edu

    David Labiner, MD

    Recruiting

  • George Washington University

    Washington D.C., District of Columbia, 20037

    Preethy Feit, SC, 202-677-6765, pfeit@mfa.gwu.edu

    Mohamad Koubeissi, MD

    Recruiting

  • Atrium Wake Forest Baptist

    Winston-Salem, North Carolina, 27157

    Carolyn W Hedrick, RMA, CCRP, 336-716-8694, cwhedric@wakehealth.edu

    Gautam S Popli, MBBS, MBA

    Recruiting

Other trials in United States

About this study

Official title: 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).

Read the 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.

Being tested
NRTX-1001, Sham Comparator
Keywords
Epilepsy, MTLE, seizure, EPIC, focal seizures, hippocampus
Medical subject headings
Epilepsy, Seizures

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • NRTX-1001Biological

    Also called GABA-secreting interneurons

    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.

  • NRTX-1001Biological

    Also called GABA-secreting interneurons

    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.

  • Sham ComparatorProcedure or surgery

    Sham Comparator

Study groups in NCT05135091
Study groupRoleWhat they receive
NRTX-1001 (Phase 1/2)ExperimentalUp to 28 subjects
Receives: Biological: NRTX-1001
NRTX-1001 (Phase 3)ExperimentalApproximately 40 subjects
Receives: Biological: NRTX-1001
Sham Comparator (Phase 3)Sham comparatorApproximately 20 subjects
Receives: Procedure: Sham Comparator

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Frequency of serious or severe AEs (Phase 1/2)

    The primary endpoint of the study is the frequency of serious or severe AEs over the period of 1 year after administration.

    Measured: 1 year

  2. Change in disabling seizure frequency (Phase 3)

    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.

    Measured: 4-6 months after surgery

Secondary outcomes (2)

  1. Responder rate (Phase 3)

    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.

    Measured: 4-6 months after surgery

  2. Responder rate (Phase 3)

    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.

    Measured: 4-6 months after surgery

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Nov 2021
  2. Study start16 Jun 2022
  3. Primary completionFeb 2029 (estimated)
  4. Study completionSep 2043 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
This is a two-phase study. Phase 1/2 is an open-label, single arm, sequential dose escalation. Phase 3 is a multicenter, double-blind, randomized, parallel group, sham controlled study.
Masking
Triple blind
Who is masked
Participant, Investigator, Outcomes assessor
Enrolment
88 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05135091
Sponsor's protocol ID
NTE001
Acronym
EPIC
Status last verified
Mar 2026
First submitted
16 Nov 2021
Last updated
30 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Bershteyn M, Broer S, Parekh M, Maury Y, Havlicek S, Kriks S, Fuentealba L, Lee S, Zhou R, Subramanyam G, Sezan M, Sevilla ES, Blankenberger W, Spatazza J, Zhou L, Nethercott H, Traver D, Hampel P, Kim H, Watson M, Salter N, Nesterova A, Au W, Kriegstein A, Alvarez-Buylla A, Rubenstein J, Banik G, Bulfone A, Priest C, Nicholas CR. Human pallial MGE-type GABAergic interneuron cell therapy for chronic focal epilepsy. Cell Stem Cell. 2023 Oct 5;30(10):1331-1350.e11. doi: 10.1016/j.stem.2023.08.013.

    PubMed 37802038

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing NRTX-1001 in people with Mesial Temporal Lobe Epilepsy, run by Neurona Therapeutics at 36 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 30 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 36 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Neurona MedInfo (650-580-3825, neuronamedinfo@neuronatx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Frequency of serious or severe AEs (Phase 1/2) (time frame: 1 year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05135091, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Oct 2026.

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