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
- 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
- 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
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental NRTX-1001 (Stage 1) |
Up to 10 subjects. |
|
|
Experimental NRTX-1001 (Stage 2) |
Up to 20 subjects. |
|
|
Sham Comparator Sham Comparator (Stage 2) |
Up to 10 subjects. |
|
Recruiting Locations
Mayo Clinic Arizona Epilepsy Center
Phoenix, Arizona 85054
Phoenix, Arizona 85054
Banner-University of Arizona Medical Center Tucson Comprehensive Epilepsy Program
Tucson, Arizona 85724-5023
Tucson, Arizona 85724-5023
University of California Los Angeles
Los Angeles, California 90095
Los Angeles, California 90095
Stanford University
Palo Alto, California 94304
Palo Alto, California 94304
University of California Davis
Sacramento, California 95817
Sacramento, California 95817
University of California San Diego
San Diego, California 92037
San Diego, California 92037
University of California San Francisco
San Francisco, California 94143
San Francisco, California 94143
University of Colorado Anschutz Medical Campus
Aurora, Colorado 80045
Aurora, Colorado 80045
Rush University Medical Center
Chicago, Illinois 60612
Chicago, Illinois 60612
University of Chicago
Chicago, Illinois 60637
Chicago, Illinois 60637
University of Iowa Health Care
Iowa City, Iowa 52242
Iowa City, Iowa 52242
Beth Israel Deaconess Medical Center
Boston, Massachusetts 02215
Boston, Massachusetts 02215
Wayne State University/Detroit Medical Center Comprehensive Epilepsy Program
Detroit, Michigan 48201
Detroit, Michigan 48201
NYU Langone Comprehensive Epilepsy Center
New York, New York 10016
New York, New York 10016
SUNY Upstate Medical University
Syracuse, New York 13210
Syracuse, New York 13210
Duke University Hospital
Durham, North Carolina 27710
Durham, North Carolina 27710
Atrium Wake Forest Baptist
Winston-Salem, North Carolina 27157
Winston-Salem, North Carolina 27157
Oregon Health and Science University
Portland, Oregon 97239
Portland, Oregon 97239
Thomas Jefferson University Hospital
Philadelphia, Pennsylvania 19107
Philadelphia, Pennsylvania 19107
UTHealth Houston
Houston, Texas 77030
Houston, Texas 77030
University of Utah Health
Salt Lake City, Utah 84108
Salt Lake City, Utah 84108
Medical College of Wisconsin
Milwaukee, Wisconsin 53226
Milwaukee, Wisconsin 53226
More Details
- NCT ID
- NCT05135091
- Status
- Recruiting
- Sponsor
- Neurona Therapeutics
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.