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

Purpose

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

  • Mesial Temporal Lobe Epilepsy With Hippocampal Sclerosis

Eligibility

Eligible Ages
Between 18 Years and 65 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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.

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 Design

Phase
Phase 1/Phase 2
Study Type
Interventional
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.
Primary Purpose
Treatment
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.

Arm Groups

ArmDescriptionAssigned Intervention
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.

Recruiting Locations

UTHealth Houston
Houston, Texas 77030
Contact:
Eliana M Klier, PhD
713-500-5442
Eliana.Klier@uth.tmc.edu

More Details

Status
Recruiting
Sponsor
Neurona Therapeutics

Study Contact

Sheri Madrid, BS, BA
949-500-0027
sheri@neuronatx.com

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.