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FIH Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy

Phase 1
Recruiting
Conditions
Mesial Temporal Lobe Epilepsy
Interventions
Biological: NRTX-1001
Procedure: Sham Comparator
Registration Number
NCT05135091
Lead Sponsor
Neurona Therapeutics
Brief 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).

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, tolerability, and effects on reducing seizure frequency and epilepsy disease symptoms will be assessed at quarterly 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.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
58
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) 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.
  7. Considered (by Investigator) to be a candidate for temporal lobectomy (TL) or Laser Interstitial Thermal Therapy (LITT) following evaluation at a qualified epilepsy surgery program (National Association of Epilepsy Centers [NAEC] Level 4).

Key

Exclusion Criteria
  1. Epilepsy due to other 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 year prior to screening. A history of cluster seizures is permitted.
  5. Primary or secondary immunodeficiency
  6. Suicide attempts in the past year
  7. Severe psychiatric disorders
  8. Prior lobectomy or LITT procedure
  9. MRI indicating potential malignant lesion
  10. Pregnancy, or currently breastfeeding.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
NRTX-1001 (Stage 2)NRTX-1001Up to 20 subjects.
Sham Comparator (Stage 2)Sham ComparatorUp to 10 subjects.
NRTX-1001 (Stage 1)NRTX-1001Up to 10 subjects.
NRTX-1001 (Phase 1)NRTX-1001Up to 28 subjects
NRTX-1001 (Phase 2)NRTX-1001Up to 20 subjects
Sham Comparator(Phase 2)Sham ComparatorUp to 10 subjects.
Primary Outcome Measures
NameTimeMethod
Frequency of serious or severe AEs1 year

The primary endpoint of the study is the frequency of serious or severe AEs over the period of 1 year after administration, comparing all subjects treated with NRTX-1001 (Phase 1 + Phase 2) with all subjects who receive sham treatment.

Secondary Outcome Measures
NameTimeMethod
Change in seizure frequency1 year

The difference in the median percentage change from baseline in seizure frequency (seizures per 28 days) between subjects that receive NRTX-1001 and those who receive sham treatment, assessed in Phase 2.

Responder rate1 year

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 seizure frequency, assessed in Phase 2.

Trial Locations

Locations (29)

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

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

University of Nebraska

🇺🇸

Omaha, Nebraska, 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

UTHealth Houston

🇺🇸

Houston, Texas, United States

University of Utah Health

🇺🇸

Salt Lake City, Utah, United States

Medical College of Wisconsin

🇺🇸

Milwaukee, Wisconsin, United States

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