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

January 19, 2024 updated by: Neurona Therapeutics

A First-In-Human (FIH) Study of Inhibitory Interneurons (NRTX- 1001) in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (MTLE)

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).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

40

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90095
        • Recruiting
        • University of California Los Angeles
        • Contact:
        • Contact:
        • Principal Investigator:
          • John M Stern, MD
      • Palo Alto, California, United States, 94304
        • Recruiting
        • Stanford University
        • Contact:
        • Contact:
        • Principal Investigator:
          • Kevin Graber, MD
        • Sub-Investigator:
          • Vivek Buch, MD
        • Sub-Investigator:
          • Jaimie Henderson, MD
        • Sub-Investigator:
          • Richard Lafayette, MD
        • Sub-Investigator:
          • Jordan Seliger
      • Sacramento, California, United States, 95817
        • Recruiting
        • University of California Davis
        • Contact:
        • Contact:
        • Principal Investigator:
          • Jack J Lin, MD
      • San Diego, California, United States, 92037
        • Recruiting
        • University of California San Diego
        • Contact:
        • Principal Investigator:
          • Jerry J Shih, MD
        • Contact:
      • San Francisco, California, United States, 94143
        • Recruiting
        • University of California San Francisco
        • Contact:
        • Contact:
        • Principal Investigator:
          • Robert C Knowlton, MD
        • Sub-Investigator:
          • Edward F Chang, MD
        • Sub-Investigator:
          • Paul A Garcia, MD
        • Sub-Investigator:
          • Jeffrey M Gelfand, MD, MAS
        • Sub-Investigator:
          • Brandon E Kopald, PsyD
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • University of Colorado Anschutz Medical Campus
        • Contact:
        • Contact:
        • 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
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Recruiting
        • Rush University Medical Center
        • Contact:
        • Contact:
        • Principal Investigator:
          • Rebecca O'Dwyer, MD
        • Sub-Investigator:
          • Travis Stoub, PhD
        • Sub-Investigator:
          • Sepehr B Sani, MD
      • Chicago, Illinois, United States, 60637
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Recruiting
        • University of Iowa Health Care
        • Contact:
        • Contact:
        • Principal Investigator:
          • Mark Granner, MD
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
    • New York
      • New York, New York, United States, 10016
        • Recruiting
        • NYU Langone Comprehensive Epilepsy Center
        • 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
        • Contact:
        • Contact:
      • Syracuse, New York, United States, 13210
        • Recruiting
        • SUNY Upstate Medical University
        • Contact:
        • Contact:
        • 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
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Recruiting
        • Duke University Hospital
        • Contact:
        • Contact:
        • Principal Investigator:
          • Derek Southwell, MD, PhD
        • 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
        • Sub-Investigator:
          • Joanne Kurtzberg, MD
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Oregon Health and Science University
        • 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
        • Contact:
        • Contact:
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19107
    • Texas
      • Houston, Texas, United States, 77030
    • Utah
      • Salt Lake City, Utah, United States, 84108
        • Recruiting
        • University of Utah Health
        • Sub-Investigator:
          • Brian Johnson, MD
        • Contact:
        • Principal Investigator:
          • Amir Arain, MD
        • Sub-Investigator:
          • Sindhu Richard, 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
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Recruiting
        • Medical College of Wisconsin
        • Contact:
        • Contact:
        • Principal Investigator:
          • Sean Lew, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 55 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  1. Male or Female, age ≥18 to 65
  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, 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.
  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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / 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 Names:
  • 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 Names:
  • GABA-secreting interneurons
Sham Comparator: Sham Comparator (Stage 2)
Up to 10 subjects.
Procedure: Sham Comparator
Sham Comparator.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
1 year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
1 year
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.
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Neurona Therapeutics

Collaborators

California Institute for Regenerative Medicine (CIRM)

Investigators

  • Study Director: David Blum, MD, Neurona Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 16, 2022

Primary Completion (Estimated)

May 1, 2025

Study Completion (Estimated)

May 1, 2026

Study Registration Dates

First Submitted

November 16, 2021

First Submitted That Met QC Criteria

November 16, 2021

First Posted (Actual)

November 26, 2021

Study Record Updates

Last Update Posted (Actual)

January 22, 2024

Last Update Submitted That Met QC Criteria

January 19, 2024

Last Verified

January 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NTE001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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