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

A first in human stem cell implantation surgery for drug resistant Temporal Lobe Epilepsy

a study on Epilepsy Seizures

Summary

Eligibility
for people ages 18-75 (full criteria)
Location
at UC Davis UC Irvine UCLA UCSD UCSF
Dates
study started
study ends around
Principal Investigator
by John M Stern, MD (ucla)Mona Sazgar, MD (uci)Jack J Lin, MD (ucdavis)Jerry J Shih, MD (ucsd)Robert C Knowlton, MD (ucsf)
Headshot of John M Stern
John M Stern
Headshot of Jerry J Shih
Jerry J Shih
Headshot of Robert C Knowlton
Robert C Knowlton

Description

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).

Official Title

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

Details

Subjects will undergo a single CT or MRI-guided intracerebral administration of human interneurons that secrete the inhibitory neurotransmitter, gamma-aminobutyric acid (GABA), into the temporal lobe region of the brain where the seizures are thought to arise. NRTX-1001 is intended to suppress the onset and spread of seizures. Safety, efficacy, tolerability, and effects on reducing seizure frequency and epilepsy disease symptoms will be assessed at regular intervals for 2 years after the administration of NRTX-1001. After the two-year period, subjects will be followed with quarterly phone calls and annual visits in years 3 through 5, and then annual visits in years 6 through 15. Subjects will be placed on an immunosuppressant medication regimen for a duration of one year to partially suppress the subjects' immune system to promote the intended long-term persistence of NRTX-1001. This immunosuppressant medication is intended to be discontinued after the first year; however, the NRTX-1001 cells are intended to persist long-term.

Keywords

Mesial Temporal Lobe Epilepsy, Epilepsy, MTLE, seizure, EPIC, focal seizures, hippocampus, Seizures, NRTX-1001

Eligibility

You can join if…

Open to people ages 18-75

  1. Male or Female, age ≥18 to ≤75
  2. Focal seizures, clinically defined as unilateral MTLE
  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 prior to Screening) of approved ASDs
  5. Single seizure focus confirmed within one hippocampus
  6. 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)
  7. 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 CAN'T join if...

  1. Epilepsy due to other medical conditions and/or progressive neurologic disease
  2. Evidence of seizure focus outside of the hippocampus or evidence of seizures of non- focal origin.
  3. Significant other medical conditions which would impair safe participation
  4. History of status epilepticus in the 3 years prior to screening.
  5. Primary or secondary immunodeficiency
  6. Suicide attempts in the past year 3 years
  7. Severe psychiatric disorders
  8. Prior lobectomy or LITT procedure
  9. MRI indicating potential malignant lesion
  10. Pregnancy, or currently breastfeeding.

Locations

  • University of California Los Angeles accepting new patients
    Los Angeles California 90095 United States
  • UC Irvine Medical Center accepting new patients
    Orange California 92868 United States
  • University of California Davis accepting new patients
    Sacramento California 95817 United States
  • University of California San Diego accepting new patients
    San Diego California 92037 United States
  • University of California San Francisco accepting new patients
    San Francisco California 94143 United States
  • University of Southern California Keck Hospital accepting new patients
    Los Angeles California 90033 United States
  • Stanford University accepting new patients
    Palo Alto California 94304 United States

Lead Scientists at University of California Health

  • John M Stern, MD (ucla)
    Dr. John Stern is Professor and Director of the Epilepsy Clinical Program in the Department of Neurology at the Geffen School of Medicine at the University of California, Los Angeles (UCLA). He is also Director of UCLA’s Epilepsy Fellowship Training Program and Medical Director of UCLA’s Seizure Disorder Center.
  • Mona Sazgar, MD (uci)
    Health Sciences Professor, Neurology, School of Medicine. Authored (or co-authored) 23 research publications
  • Jack J Lin, MD (ucdavis)
    Professor, Med: Neurology, School of Medicine. Authored (or co-authored) 55 research publications
  • Jerry J Shih, MD (ucsd)
    Professor Of Clinical, Neurosciences, Vc-health Sciences-schools
  • Robert C Knowlton, MD (ucsf)
    Dr. Robert Knowlton is a neurologist who specializes in diagnosing complex cases of epilepsy, such as those with no clear origin found using standard tests. In his clinical practice, Knowlton combines a variety of advanced diagnostic tests to pinpoint the source of seizures in the brain.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Neurona Therapeutics
Links
Related Info
ID
NCT05135091
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 88 study participants
Last Updated
Contact us about this trial