A Phase 1/2 Study of NRTX-1001 Neuronal Cell Therapy in Drug-Resistant Bilateral Mesial Temporal Lobe Epilepsy (MTLE)
1 other identifier
interventional
10
1 country
17
Brief Summary
This is a multicenter, single arm, open label clinical trial that is designed to test the safety and preliminary efficacy of single administration inhibitory nerve cells called interneurons (NRTX-1001), into both temporal lobes of subjects with drug-resistant bilateral mesial temporal lobe epilepsy.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_1
Started Nov 2024
Longer than P75 for phase_1
17 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 15, 2024
CompletedFirst Posted
Study publicly available on registry
May 21, 2024
CompletedStudy Start
First participant enrolled
November 14, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 15, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
June 15, 2042
January 7, 2026
January 1, 2026
2.6 years
May 15, 2024
January 5, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Incidence of Serious Adverse Events at end of month 12
12 months after treatment
Secondary Outcomes (1)
Change in frequency of clinical seizures
12 months after treatment
Study Arms (1)
Treatment Arm
EXPERIMENTALThis is an open-label study of the bilateral intrahippocampal administration of NRTX-1001 in a single dose cohort of up to 10 subjects with drug-resistant bilateral MTLE.
Interventions
Biological: NRTX-1001 is an inhibitory neural cell therapy investigational product. It is derived from a human stem cell line that has been converted into high-purity inhibitory interneurons that produce GABA. NRTX-1001 is intended to persist long-term and not require repeated administration.
Eligibility Criteria
You may qualify if:
- Male or female, age 18-75 years.
- Subjects of childbearing potential will use highly effective contraception.
- Proven history of focal seizures of hippocampal origin with bilateral seizure foci confirmed by scalp or intracranial ictal EEG (including confirmation by recordings from responsive neurostimulation \[RNS\] electrodes when applicable).
- Either
- bilateral hippocampal sclerosis on MRI (evidenced by increased FLAIR signal intensity in both hippocampi or by visual assessment showing reduced volume compared to normal) or
- bilateral temporal hypometabolism on 18-Flourodeoxyglucose Positron Emission Tomography (FDG PET) (assessed by visual assessment, comparing temporal regions to frontal/parietal lobe neocortex). In this case, ictal EEG recordings must also include intracranial confirmation.
- a combination of unilateral instances of the evidence described in a. and b. (e.g., one side can be evidenced by criterion a. and the other side by criterion b.) MRI or PET scans used for assessment must have been acquired within 3 years of screening.
- Subject has had at least four clinical focal seizures, including at least two clinical focal seizures with objective manifestations, on average, per month for the 6 months prior to screening.
- Subject has previously had adequate (in opinion of investigator) therapeutic trials of at least two Anti-Seizure Medicines (ASMs).
- Current ASM regimen, and doses of other drugs known to affect seizure frequency (e.g., antidepressants), have been stable for at least three months prior to enrollment.
- Subject can converse and read in English or Spanish. Able to participate in required study procedures and provide signed informed consent.
You may not qualify if:
- Epilepsy due to other and/or progressive neurologic disease.
- Evidence of seizure focus outside hippocampus (either by seizure semiology or EEG findings).
- Seizures of non-focal origin.
- History of status epilepticus in the year prior to screening, as guided by ILAE criteria (Trinka 2015) in the judgement of the PI. A history of cluster seizures is permitted.
- Psychogenic Non-Epileptic Seizures (PNES) within the past 3 years.
- Severe psychiatric disorders.
- Primary or secondary immunodeficiency.
- Pregnancy, or currently breastfeeding.
- Suicide attempts in past year.
- Significant other medical conditions which would impair safe participation.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (17)
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72205, United States
University of Southern California Keck Hospital
Los Angeles, California, 90033, United States
UC Irvine Medical Center
Orange, California, 92868, United States
Stanford University
Palo Alto, California, 94304, 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
University of Chicago
Chicago, Illinois, 60637, United States
University of Iowa Health Care
Iowa City, Iowa, 52242, 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
Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, 27157, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
UTHealth Houston
Houston, Texas, 77030, United States
UVA Health University Medical Center
Charlottesville, Virginia, 22903, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
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 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 15, 2024
First Posted
May 21, 2024
Study Start
November 14, 2024
Primary Completion (Estimated)
June 15, 2027
Study Completion (Estimated)
June 15, 2042
Last Updated
January 7, 2026
Record last verified: 2026-01
Data Sharing
- IPD Sharing
- Will not share