RecruitingPhase 3NCT05135091

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

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


Sponsor

Neurona Therapeutics

Enrollment

88 participants

Start Date

Jun 16, 2022

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 18 YearsMax Age: 75 Years

Inclusion Criteria7

  • 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)

Exclusion Criteria10

  • 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
  • Severe psychiatric disorders
  • Prior lobectomy or LITT procedure
  • MRI indicating potential malignant lesion
  • Pregnancy, or currently breastfeeding.

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Interventions

BIOLOGICALNRTX-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.

BIOLOGICALNRTX-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.

PROCEDURESham Comparator

Sham Comparator


Locations(33)

Mayo Clinic Arizona Epilepsy Center

Phoenix, Arizona, United States

Banner-University of Arizona Medical Center Tucson Comprehensive Epilepsy Program

Tucson, Arizona, United States

University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States

University of Southern California Keck Hospital

Los Angeles, California, United States

University of California Los Angeles

Los Angeles, California, United States

UC Irvine Medical Center

Orange, California, United States

Stanford University

Palo Alto, California, United States

University of California Davis

Sacramento, California, United States

University of California San Diego

San Diego, California, United States

University of California San Francisco

San Francisco, California, United States

University of Colorado Anschutz Medical Campus

Aurora, Colorado, United States

George Washington University

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

University of Miami

Miami, Florida, United States

Rush University Medical Center

Chicago, Illinois, United States

University of Chicago

Chicago, Illinois, United States

University of Iowa Health Care

Iowa City, Iowa, United States

Ochsner Clinic Foundation

New Orleans, Louisiana, United States

Beth Israel Deaconess Medical Center

Boston, Massachusetts, United States

Wayne State University/Detroit Medical Center Comprehensive Epilepsy Program

Detroit, Michigan, United States

Dartmouth Hitchcock Medical Center/ Department of Neurology

Lebanon, New Hampshire, United States

Hofstra Northwell Comprehensive Epilepsy Center

Great Neck, New York, United States

NYU Langone Comprehensive Epilepsy Center

New York, New York, United States

SUNY Upstate Medical University

Syracuse, New York, United States

Atrium Health

Charlotte, North Carolina, United States

Duke University Hospital

Durham, North Carolina, United States

Atrium Wake Forest Baptist

Winston-Salem, North Carolina, United States

Oregon Health and Science University

Portland, Oregon, United States

Thomas Jefferson University Hospital

Philadelphia, Pennsylvania, United States

UT Southwestern Medical Center

Dallas, Texas, United States

UTHealth Houston

Houston, Texas, United States

University of Utah Health

Salt Lake City, Utah, United States

University of Washington Regional Epilepsy Center

Seattle, Washington, United States

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

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