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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05135091
Recruitment Status : Recruiting
First Posted : November 26, 2021
Last Update Posted : June 21, 2022
Sponsor:
Information provided by (Responsible Party):
Neurona Therapeutics

Brief Summary:
This clinical trial is designed to test whether a single stereotactic intracerebral administration of inhibitory nerve cells into subjects with drug-resistant mesial temporal lobe epilepsy is safe (frequency of adverse events) and effective (seizure frequency).

Condition or disease Intervention/treatment Phase
Mesial Temporal Lobe Epilepsy With Hippocampal Sclerosis Biological: NRTX-1001 Procedure: Sham Comparator Phase 1 Phase 2

Detailed Description:

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.

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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)
Estimated Study Start Date : June 2022
Estimated Primary Completion Date : May 2025
Estimated Study Completion Date : May 2026

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Epilepsy

Arm Intervention/treatment
Experimental: NRTX-1001 (Stage 1)
Up to 10 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

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.




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  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.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 55 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  1. Male or Female, age ≥18 to 55
  2. Focal seizures, clinically defined as TLE
  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 as within one temporal lobe
  6. For subjects entering Stage 1, seizure focus is in the non-dominant hemisphere.
  7. Seizure frequency averages ≥2 per 28-day period over the 6 months prior to screening.

Key Exclusion Criteria:

  1. Epilepsy due to other and/or progressive neurologic disease
  2. Significant other medical condition which would impair safe participation
  3. Primary or secondary immunodeficiency
  4. Suicide attempt in the past year
  5. Severe psychiatric disorders
  6. Chronic indwelling intracranial device
  7. MRI indicating potential malignant lesion
  8. Pregnancy, or currently breastfeeding

Information from the National Library of Medicine

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


Contacts
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Contact: Sheri Madrid, BS, BA 650-255-6885 sheri.madrid@neuronatx.com
Contact: C Priest, PhD 650-392-0372 cpriest@neuronatx.com

Locations
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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: Saurabh Sinha, MD, 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         
Sponsors and Collaborators
Neurona Therapeutics
Investigators
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Study Director: David Blum, MD Neurona Therapeutics
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Responsible Party: Neurona Therapeutics
ClinicalTrials.gov Identifier: NCT05135091    
Other Study ID Numbers: NTE001
First Posted: November 26, 2021    Key Record Dates
Last Update Posted: June 21, 2022
Last Verified: June 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Epilepsy
Epilepsy, Temporal Lobe
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Epilepsies, Partial
Epileptic Syndromes