Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (EPIC (EPIlepsy Cell Therapy))
EPIC
A Study of Inhibitory Interneurons (NRTX-1001) in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (MTLE)
1 other identifier
interventional
88
1 country
33
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Jun 2022
Longer than P75 for phase_3
33 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 16, 2021
CompletedFirst Posted
Study publicly available on registry
November 26, 2021
CompletedStudy Start
First participant enrolled
June 16, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 1, 2029
ExpectedStudy Completion
Last participant's last visit for all outcomes
September 1, 2043
March 30, 2026
March 1, 2026
6.6 years
November 16, 2021
March 26, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
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.
1 year
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.
4-6 months after surgery
Secondary Outcomes (2)
Responder rate (Phase 3)
4-6 months after surgery
Responder rate (Phase 3)
4-6 months after surgery
Study Arms (3)
NRTX-1001 (Phase 1/2)
EXPERIMENTALUp to 28 subjects
NRTX-1001 (Phase 3)
EXPERIMENTALApproximately 40 subjects
Sham Comparator (Phase 3)
SHAM COMPARATORApproximately 20 subjects
Interventions
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.
Eligibility Criteria
You may qualify if:
- 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)
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (33)
Mayo Clinic Arizona Epilepsy Center
Phoenix, Arizona, 85054, United States
Banner-University of Arizona Medical Center Tucson Comprehensive Epilepsy Program
Tucson, Arizona, 85724-5023, United States
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72205, United States
University of Southern California Keck Hospital
Los Angeles, California, 90033, United States
University of California Los Angeles
Los Angeles, California, 90095, United States
UC Irvine Medical Center
Orange, California, 92868, United States
Stanford University
Palo Alto, California, 94304, United States
University of California Davis
Sacramento, California, 95817, United States
University of California San Diego
San Diego, California, 92037, United States
University of California San Francisco
San Francisco, California, 94143, United States
University of Colorado Anschutz Medical Campus
Aurora, Colorado, 80045, United States
George Washington University
Washington D.C., District of Columbia, 20037, United States
University of Miami
Miami, Florida, 33136, United States
Rush University Medical Center
Chicago, Illinois, 60612, United States
University of Chicago
Chicago, Illinois, 60637, United States
University of Iowa Health Care
Iowa City, Iowa, 52242, United States
Ochsner Clinic Foundation
New Orleans, Louisiana, 70121, United States
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215, United States
Wayne State University/Detroit Medical Center Comprehensive Epilepsy Program
Detroit, Michigan, 48201, United States
Dartmouth Hitchcock Medical Center/ Department of Neurology
Lebanon, New Hampshire, 03756, United States
Hofstra Northwell Comprehensive Epilepsy Center
Great Neck, New York, 11021, United States
NYU Langone Comprehensive Epilepsy Center
New York, New York, 10016, United States
SUNY Upstate Medical University
Syracuse, New York, 13210, United States
Atrium Health
Charlotte, North Carolina, 28204, United States
Duke University Hospital
Durham, North Carolina, 27710, United States
Atrium Wake Forest Baptist
Winston-Salem, North Carolina, 27157, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
Thomas Jefferson University Hospital
Philadelphia, Pennsylvania, 19107, United States
UT Southwestern Medical Center
Dallas, Texas, 75390, United States
UTHealth Houston
Houston, Texas, 77030, United States
University of Utah Health
Salt Lake City, Utah, 84108, United States
University of Washington Regional Epilepsy Center
Seattle, Washington, 98104, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
Related Publications (1)
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.
PMID: 37802038DERIVED
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Eduardo Dunayevich, MD
Neurona Therapeutics
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
- Masking Details
- 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.
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 16, 2021
First Posted
November 26, 2021
Study Start
June 16, 2022
Primary Completion (Estimated)
February 1, 2029
Study Completion (Estimated)
September 1, 2043
Last Updated
March 30, 2026
Record last verified: 2026-03
Data Sharing
- IPD Sharing
- Will not share