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

NCT ID: NCT05135091

Last Updated: 2026-01-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

88 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-06-16

Study Completion Date

2043-09-30

Brief Summary

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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).

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Detailed Description

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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.

Conditions

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Mesial Temporal Lobe Epilepsy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

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.
Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors
This is a two-phase study. Phase 1/2 is open-label and unmasked. Phase 3 is blinded with participant, part of investigator team, and outcomes assessor masked to treatment assignment.

Study Groups

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NRTX-1001 (Phase 1/2)

Up to 28 subjects

Group Type EXPERIMENTAL

NRTX-1001

Intervention Type BIOLOGICAL

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-1001 (Phase 3)

Approximately 40 subjects

Group Type EXPERIMENTAL

NRTX-1001

Intervention Type BIOLOGICAL

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 Comparator (Phase 3)

Approximately 20 subjects

Group Type SHAM_COMPARATOR

Sham Comparator

Intervention Type PROCEDURE

Sham Comparator

Interventions

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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.

Intervention Type 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.

Intervention Type BIOLOGICAL

Sham Comparator

Sham Comparator

Intervention Type PROCEDURE

Other Intervention Names

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GABA-secreting interneurons GABA-secreting interneurons

Eligibility Criteria

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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)

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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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California Institute for Regenerative Medicine (CIRM)

OTHER

Sponsor Role collaborator

Neurona Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Eduardo Dunayevich, MD

Role: STUDY_DIRECTOR

Neurona Therapeutics

Locations

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Mayo Clinic Arizona Epilepsy Center

Phoenix, Arizona, United States

Site Status RECRUITING

Banner-University of Arizona Medical Center Tucson Comprehensive Epilepsy Program

Tucson, Arizona, United States

Site Status RECRUITING

University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States

Site Status RECRUITING

University of Southern California Keck Hospital

Los Angeles, California, United States

Site Status RECRUITING

University of California Los Angeles

Los Angeles, California, United States

Site Status RECRUITING

UC Irvine Medical Center

Orange, California, United States

Site Status RECRUITING

Stanford University

Palo Alto, California, United States

Site Status RECRUITING

University of California Davis

Sacramento, California, United States

Site Status RECRUITING

University of California San Diego

San Diego, California, United States

Site Status RECRUITING

University of California San Francisco

San Francisco, California, United States

Site Status RECRUITING

University of Colorado Anschutz Medical Campus

Aurora, Colorado, United States

Site Status RECRUITING

George Washington University

Washington D.C., District of Columbia, United States

Site Status RECRUITING

University of Miami

Miami, Florida, United States

Site Status RECRUITING

Rush University Medical Center

Chicago, Illinois, United States

Site Status RECRUITING

University of Chicago

Chicago, Illinois, United States

Site Status RECRUITING

University of Iowa Health Care

Iowa City, Iowa, United States

Site Status RECRUITING

Ochsner Clinic Foundation

New Orleans, Louisiana, United States

Site Status RECRUITING

Beth Israel Deaconess Medical Center

Boston, Massachusetts, United States

Site Status RECRUITING

Wayne State University/Detroit Medical Center Comprehensive Epilepsy Program

Detroit, Michigan, United States

Site Status RECRUITING

NYU Langone Comprehensive Epilepsy Center

New York, New York, United States

Site Status RECRUITING

SUNY Upstate Medical University

Syracuse, New York, United States

Site Status RECRUITING

Atrium Health

Charlotte, North Carolina, United States

Site Status RECRUITING

Duke University Hospital

Durham, North Carolina, United States

Site Status RECRUITING

Atrium Wake Forest Baptist

Winston-Salem, North Carolina, United States

Site Status RECRUITING

Oregon Health and Science University

Portland, Oregon, United States

Site Status RECRUITING

Thomas Jefferson University Hospital

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

UT Southwestern Medical Center

Dallas, Texas, United States

Site Status RECRUITING

UTHealth Houston

Houston, Texas, United States

Site Status RECRUITING

University of Utah Health

Salt Lake City, Utah, United States

Site Status RECRUITING

University of Washington Regional Epilepsy Center

Seattle, Washington, United States

Site Status RECRUITING

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Neurona MedInfo

Role: CONTACT

[email protected]
650-580-3825

Eduardo Dunayevich, MD

Role: CONTACT

[email protected]
650-436-3045

Facility Contacts

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Kayla N Haeger, BSN, RN

Role: primary

[email protected]
480-342-5075 ext. 2-5075

Faten Sebaali, BA/MA

Role: primary

[email protected]
(520) 626-3576

Shellah Rogers, Nurse

Role: primary

[email protected]
501-614-2086

Zoe B Durkin, SC

Role: primary

[email protected]
213-631-8487

Elizabeth Cruz

Role: primary

[email protected]
310-825-6930

UCI Alpha Clinic

Role: primary

[email protected]
949-824-3990

Jordan Seliger, CRC, MA

Role: primary

[email protected]
650-460-9260

Evan Y Shen, BA

Role: primary

[email protected]
916-734-3009

Mateo Santizo

Role: primary

[email protected]
858-583-0929

Sydnie Beausoleil

Role: primary

[email protected]
631-428-9688

Trey Jouard, MS

Role: primary

[email protected]
970-817-4272

Preethy Feit, SC

Role: primary

[email protected]
202-677-6765

Letitia Fisher

Role: primary

[email protected]
305-775-8582

Amanda Sremac, BS

Role: primary

[email protected]
312-942-0539

Agnieszka Stadnik, MS

Role: primary

[email protected]
773-702-8996

Loraine Brenner, MSN

Role: primary

[email protected]
319-356-4361

Neydin Osorio, BS, CRC

Role: primary

[email protected]
504-703-5577

Anna George, MSc

Role: primary

[email protected]
617-632-7031

Kelly X Jia, MD

Role: primary

[email protected]
313-966-9407

Jack C Carter, BS

Role: primary

[email protected]
646-558-0841

Lena F Deb, BA

Role: primary

[email protected]
315-464-9756

Dianelis Diaz, RN, BSN, CRC

Role: primary

[email protected]
704-446-1349

Hazani Benitez-Rosas

Role: primary

[email protected]
919-681-4974

Carolyn W Hedrick, RMA, CCRP

Role: primary

[email protected]
336-716-8694

Claire Dorfman, BA

Role: primary

[email protected]
971-413-9201

Latasha Adams, MBS

Role: primary

[email protected]
215-955-2606

Vamsi Vemireddy, SC

Role: primary

[email protected]
214-645-8159

Eliana M Klier, PhD

Role: primary

[email protected]
713-500-5442

Laura Beeler, BS

Role: primary

[email protected]
928-530-7990

Jennifer Steele, CRC

Role: primary

[email protected]
206-744-2336

Sarah Young, MS

Role: primary

[email protected]
414-955-0989

References

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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.

Reference Type DERIVED
PMID: 37802038 (View on PubMed)

Related Links

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http://www.neuronatherapeutics.com

Related Info

Other Identifiers

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NTE001

Identifier Type: -

Identifier Source: org_study_id

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