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Clinical Trials in the USA / NCT05135091
Recruiting Phase 3

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

NCT05135091 · tracked via the Priya Life Science USA tracker
Sponsor
Neurona Therapeutics
Phase
Phase 3
Started
2022-06-16
Last updated
2026-10-30

Condition(s) studied

Mesial Temporal Lobe Epilepsy

Investigational drug(s) / intervention(s)

NRTX-1001 →NRTX-1001 →Sham Comparator

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.

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.

Sham Comparator: Sham Comparator

Study 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 adverse events) and effective (seizure frequency). NRTX-1001 comprises human interneurons that secrete a neurotransmitter, gamma-aminobutyric acid (GABA).

Eligibility

Sex
ALL
Min age
18 Years
Max age
75 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

  • Frequency of serious or severe AEs (Phase 1/2) — 1 year
    The primary endpoint of the study is the frequency of serious or severe AEs over the period of 1 year after administration.
  • Change in disabling seizure frequency (Phase 3) — 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.

Trial sites (36)

FacilityCityRegionStatus
University of Alabama Birmingham Alabama Recruiting
Mayo Clinic Arizona Epilepsy Center Phoenix Arizona Recruiting
Banner-University of Arizona Medical Center Tucson Comprehensive Epilepsy Program Tucson Arizona Recruiting
University of Arkansas for Medical Sciences Little Rock Arkansas Recruiting
University of Southern California Keck Hospital Los Angeles California Recruiting
University of California Los Angeles Los Angeles California Recruiting
UC Irvine Medical Center Orange California Recruiting
Stanford University Palo Alto California Recruiting
University of California Davis Sacramento California Recruiting
University of California San Diego San Diego California Recruiting
University of California San Francisco San Francisco California Recruiting
University of Colorado Anschutz Medical Campus Aurora Colorado Recruiting
George Washington University Washington D.C. District of Columbia Recruiting
University of Miami Miami Florida Recruiting
Rush University Medical Center Chicago Illinois Recruiting
University of Chicago Chicago Illinois Recruiting
University of Iowa Health Care Iowa City Iowa Recruiting
Ochsner Clinic Foundation New Orleans Louisiana Recruiting
Beth Israel Deaconess Medical Center Boston Massachusetts Recruiting
Wayne State University/Detroit Medical Center Comprehensive Epilepsy Program Detroit Michigan Recruiting
Washington University at St. Louis St Louis Missouri Recruiting
Dartmouth Hitchcock Medical Center/ Department of Neurology Lebanon New Hampshire Recruiting
Kaleida Health Buffalo New York Recruiting
Hofstra Northwell Comprehensive Epilepsy Center Great Neck New York Recruiting
NYU Langone Comprehensive Epilepsy Center New York New York Recruiting
SUNY Upstate Medical University Syracuse New York Recruiting
Atrium Health Charlotte North Carolina Recruiting
Duke University Hospital Durham North Carolina Recruiting
Atrium Wake Forest Baptist Winston-Salem North Carolina Recruiting
Oregon Health and Science University Portland Oregon Recruiting
Thomas Jefferson University Hospital Philadelphia Pennsylvania Recruiting
UT Southwestern Medical Center Dallas Texas Recruiting
UTHealth Houston Houston Texas Recruiting
University of Utah Health Salt Lake City Utah Recruiting
University of Washington Regional Epilepsy Center Seattle Washington Recruiting
Medical College of Wisconsin Milwaukee Wisconsin Recruiting

More Neurona Therapeutics trials in the USA

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

Other trials for the same condition

AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05135091 on ClinicalTrials.gov ↗ ← All trials in the USA