FIH Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy
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ClinicalTrials.gov Identifier: NCT05135091 |
Recruitment Status :
Recruiting
First Posted : November 26, 2021
Last Update Posted : April 19, 2023
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Mesial Temporal Lobe Epilepsy With Hippocampal Sclerosis | Biological: NRTX-1001 Procedure: Sham Comparator | Phase 1 Phase 2 |
Subjects will undergo a single stereotactic intracerebral administration of neural cells, called interneurons, that secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA).
Subjects will then take medicines to partially suppress their immune system (aimed to prevent the body from rejecting the cells) for 1 year. Safety, tolerability, evidence of neural cell viability and local inflammation (using MRI scans of the brain), and effects on epilepsy disease symptoms will be assessed for 2 years post-transplant. Subjects will be followed for an additional 13 years with quarterly phone contact and annual visits.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 40 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Intervention Model Description: | This is a two-stage study. Stage 1 is an open-label, single arm, sequential dose escalation. Stage 2 is a parallel, randomized, 2-arm, sham controlled study. |
Masking: | Triple (Participant, Investigator, Outcomes Assessor) |
Masking Description: | This is a two-stage study. Stage 1 is open-label and unmasked. Stage 2 is masked with participant, part of investigator team, and outcomes assessor masked to treatment assignment. |
Primary Purpose: | Treatment |
Official Title: | A First-In-Human (FIH) Study of Inhibitory Interneurons (NRTX- 1001) in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (MTLE) |
Actual Study Start Date : | June 16, 2022 |
Estimated Primary Completion Date : | May 2025 |
Estimated Study Completion Date : | May 2026 |

Arm | Intervention/treatment |
---|---|
Experimental: NRTX-1001 (Stage 1)
Up to 10 subjects.
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Biological: 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.
Other Name: GABA-secreting interneurons |
Experimental: NRTX-1001 (Stage 2)
Up to 20 subjects.
|
Biological: 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.
Other Name: GABA-secreting interneurons |
Sham Comparator: Sham Comparator (Stage 2)
Up to 10 subjects.
|
Procedure: Sham Comparator
Sham Comparator. |
- Frequency of serious or severe AEs [ Time Frame: 1 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 (Stage 1 + Stage 2) with all subjects who receive sham treatment.
- Change in seizure frequency [ Time Frame: 1 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 Stage 2.
- Responder rate [ Time Frame: 1 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 Stage 2.

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Ages Eligible for Study: | 18 Years to 55 Years (Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Key Inclusion Criteria:
- Male or Female, age ≥18 to 55
- Focal seizures, clinically defined as TLE
- 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) of approved ASDs
- Single seizure focus confirmed as within one temporal lobe
- For subjects entering Stage 1, the seizure focus is either a) in the non-dominant hemisphere, or b) in the dominant hemisphere and the subject has a Rey Auditory Verbal Learning Test (RAVLT) assessed within one year of screening, or at the screening visit, that is at least 1.5 standard deviations below the population mean.
- Seizure frequency averages ≥2 per 28-day period over the 6 months prior to screening.
Key Exclusion Criteria:
- Epilepsy due to other and/or progressive neurologic disease
- Significant other medical condition which would impair safe participation
- Primary or secondary immunodeficiency
- Suicide attempt in the past year
- Severe psychiatric disorders
- Chronic indwelling intracranial device
- MRI indicating potential malignant lesion
- Pregnancy, or currently breastfeeding

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05135091
Contact: Sheri Madrid, BS, BA | 650-255-6885 | sheri.madrid@neuronatx.com | |
Contact: C Priest, PhD | 650-392-0372 | cpriest@neuronatx.com |
United States, California | |
Stanford University | Recruiting |
Palo Alto, California, United States, 94304 | |
Contact: Jordan Seliger, CRC, MA 650-460-9260 jseliger@stanford.edu | |
Contact: Ruba Shaik, CRC, BS 650-546-1436 rubas@stanford.edu | |
Principal Investigator: Kevin Graber, MD | |
Sub-Investigator: Vivek Buch, MD | |
Sub-Investigator: Jaimie Henderson, MD | |
Sub-Investigator: Richard Lafayette, MD | |
Sub-Investigator: Jordan Seliger | |
University of California San Diego | Recruiting |
San Diego, California, United States, 92037 | |
Contact: Christian P Fulinara, BS 858-249-3038 chfulinara@health.ucsd.edu | |
Contact: Alpha Stem Cell Clinic 844-317-7836 alphastemcellclinic@ucsd.edu | |
Principal Investigator: Jerry J Shih, MD | |
United States, Colorado | |
University of Colorado Anschutz Medical Campus | Recruiting |
Aurora, Colorado, United States, 80045 | |
Contact: Trey Jouard, MS 970-817-4272 Trey.Jouard@cuanschutz.edu | |
Contact: Christopher Mizenko, MS 303-724-4117 Christopher.Mizenko@cuanschutz.edu | |
Principal Investigator: Lesley Kaye, MD | |
Sub-Investigator: Steven Ojemann, MD | |
Sub-Investigator: Daniel Kramer, MD | |
Sub-Investigator: Amanda Piquet, MD | |
Sub-Investigator: Naveed Chaudhry, MD | |
Sub-Investigator: Thomas Wodushek, PhD | |
Sub-Investigator: Christopher Mizenko | |
Sub-Investigator: Trey Jouard | |
Sub-Investigator: Michelle Sandoval | |
United States, Illinois | |
Rush University Medical Center | Recruiting |
Chicago, Illinois, United States, 60612 | |
Contact: Amanda Sremac, BS 312-942-0539 Amanda_C_Sremac@rush.edu | |
Contact: Rebecca McWilliams, BS 312-563-1025 Rebecca_McWilliams@rush.edu | |
Principal Investigator: Rebecca O'Dwyer, MD | |
Sub-Investigator: Travis Stoub, PhD | |
Sub-Investigator: Sepehr B Sani, MD | |
United States, New York | |
NYU Langone Comprehensive Epilepsy Center | Recruiting |
New York, New York, United States, 10016 | |
Contact: Latoya T King, MBS, MS, MPH 646-558-0838 Latoya.King@nyulangone.org | |
Contact: Irene Jun, BS 646-558-0818 Irene.Jun@nyulangone.org | |
Principal Investigator: Orrin Devinsky, MD | |
Sub-Investigator: Daniel Friedman, MD | |
Sub-Investigator: Werner Doyle, MD | |
Sub-Investigator: Thomas Wisniewski, MD | |
Sub-Investigator: Bruce Solitar, MD | |
SUNY Upstate Medical University | Recruiting |
Syracuse, New York, United States, 13210 | |
Contact: Lena F Deb, BA 315-464-9756 debl@upstate.edu | |
Contact: Marielle N De Masi, BS 315-464-9756 demasim@upstate.edu | |
Principal Investigator: Robert L Beach, MD, PhD | |
Sub-Investigator: Corey A McGraw, MD | |
Sub-Investigator: Harish Babu, MD, PhD | |
Sub-Investigator: Shahram Izadyar, MD | |
Sub-Investigator: Xiangping Zhou, MD, PhD | |
United States, North Carolina | |
Duke University Hospital | Recruiting |
Durham, North Carolina, United States, 27710 | |
Contact: Jodi Lennon, BSN, RN 919-613-9129 jodi.lennon@duke.edu | |
Contact: Beth Perry, RN, CCRP 919-681-2695 beth.perry@duke.edu | |
Principal Investigator: Derek Southwell, MD, PhD | |
Sub-Investigator: Joanne Kurtzberg, MD | |
Sub-Investigator: Austin Dixon, MD, MBA | |
Sub-Investigator: Sidney Gospe III, MD, PhD | |
Sub-Investigator: Jill Stuart, PhD | |
Sub-Investigator: Mitchell E Horwitz, MD | |
Sub-Investigator: Beth Shaz, MD, MBA | |
Sub-Investigator: Matthew Luedke, MD | |
United States, Oregon | |
Oregon Health and Science University | Recruiting |
Portland, Oregon, United States, 97239 | |
Contact: Monica Dolan, R.N. 503-494-7224 dolan@ohsu.edu | |
Contact: Megan Pattle, R.N. 503-494-7191 rossme@ohsu.com | |
Principal Investigator: David C Spencer, MD | |
Sub-Investigator: Kim Burchiel, MD | |
Sub-Investigator: Paul Motika, MD | |
Sub-Investigator: Lia Ernst, MD | |
Sub-Investigator: David Pettersson, MD | |
Sub-Investigator: Michael Lane, MD | |
Sub-Investigator: Tyler Duffield, PhD | |
United States, Pennsylvania | |
Thomas Jefferson University Hospital | Recruiting |
Philadelphia, Pennsylvania, United States, 19107 | |
Contact: Mary E Sweeney, BA 215-955-2524 Mary.Sweeney2@jefferson.edu | |
Contact: Emily Patunas, CRC 215-955-8648 Emily.Patunas@jefferson.edu | |
Principal Investigator: Michael Sperling, MD | |
United States, Utah | |
University of Utah Health | Recruiting |
Salt Lake City, Utah, United States, 84108 | |
Contact: Laura Beeler, BS 801-585-9266 laura.beeler@hsc.utah.edu | |
Contact: Marissa Castillo, BS 801-585-9266 marissa.castillo@hsc.utah.edu | |
Principal Investigator: Amir Arain, MD | |
Sub-Investigator: Sindhu Richard, MD | |
Sub-Investigator: Brian Johnson, MD | |
Sub-Investigator: Blake Newman, MD | |
Sub-Investigator: Angela Peters, MD | |
Sub-Investigator: Katherine Krulisky, MD | |
Sub-Investigator: Shervin Rahimpour, MD | |
Sub-Investigator: Michelle Miranda, PhD, MPH | |
Sub-Investigator: Jonathan Galli, MD | |
United States, Wisconsin | |
Medical College of Wisconsin | Recruiting |
Milwaukee, Wisconsin, United States, 53226 | |
Contact: Sarah Cornell, MS 414-955-0989 scornell@mcw.edu | |
Contact: Kaitlin Goetschel, BS 414-955-7398 kgoetschel@mcw.edu | |
Principal Investigator: Sean Lew, MD |
Study Director: | David Blum, MD | Neurona Therapeutics |
Responsible Party: | Neurona Therapeutics |
ClinicalTrials.gov Identifier: | NCT05135091 |
Other Study ID Numbers: |
NTE001 |
First Posted: | November 26, 2021 Key Record Dates |
Last Update Posted: | April 19, 2023 |
Last Verified: | April 2023 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Epilepsy Epilepsy, Temporal Lobe Hippocampal Sclerosis Brain Diseases Central Nervous System Diseases Nervous System Diseases |
Epilepsies, Partial Epileptic Syndromes Malformations of Cortical Development, Group I Malformations of Cortical Development Nervous System Malformations Congenital Abnormalities |