Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (EPIC (EPIlepsy Cell Therapy))
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: NRTX-1001, NRTX-1001, Sham Comparator.
- Who it may be relevant to
- Registry conditions: Mesial Temporal Lobe Epilepsy. Basic parameters: 18 years — 75 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
A Study of Inhibitory Interneurons (NRTX-1001) in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (MTLE)
Overview
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).
Detailed description
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.
Interventions
- 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. - 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. - Procedure Sham Comparator
Sham Comparator
Primary outcome measures
- Frequency of serious or severe AEs (Phase 1/2) [Time frame: 1 year]
- Change in disabling seizure frequency (Phase 3) [Time frame: 4-6 months after surgery]
Secondary outcome measures (2)
- Responder rate (Phase 3) [Time frame: 4-6 months after surgery]
- Responder rate (Phase 3) [Time frame: 4-6 months after surgery]
Eligibility criteria
Inclusion criteria
- Male or Female, age ≥18 to ≤75
- Focal seizures, clinically defined as unilateral MTLE
- Has failed to achieve seizure control despite adequate trials of at least 2 ASDs at appropriate doses
- Currently on stable doses (at least 1 month prior to Screening) of approved ASDs
- Single seizure focus confirmed within one hippocampus
- 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)
- 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)
Exclusion criteria
- Epilepsy due to other medical conditions and/or progressive neurologic disease
- Evidence of seizure focus outside of the hippocampus or evidence of seizures of non- focal origin.
- Significant other medical conditions which would impair safe participation
- History of status epilepticus in the 3 years prior to screening.
- Primary or secondary immunodeficiency
- Suicide attempts in the past year 3 years
- Severe psychiatric disorders
- Prior lobectomy or LITT procedure
- MRI indicating potential malignant lesion
- Pregnancy, or currently breastfeeding.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Triple blind
- Primary purpose
- Treatment
Study locations
United States · 35 centers
- University of Alabama — Birmingham
- Mayo Clinic Arizona Epilepsy Center — Phoenix
- Banner-University of Arizona Medical Center Tucson Comprehensive Epilepsy Program — Tucson
- University of Arkansas for Medical Sciences — Little Rock
- University of Southern California Keck Hospital — Los Angeles
- University of California Los Angeles — Los Angeles
- UC Irvine Medical Center — Orange
- Stanford University — Palo Alto
- … and 27 more centers
Publications
- Bershteyn M, Broer S, Parekh M, Maury Y, Havlicek S, Kriks S, Fuentealba L, Lee S, Zhou R, Subramanyam G, Sezan M, Sevilla ES, Blankenberger W, Spatazza J, Zhou L, Nethercott H, Traver D, Hampel P, Kim H, Watson M, Salter N, Nesterova A, Au W, Kriegstein A, Alvarez-Buylla A, Rubenstein J, Banik G, Bulfone A, Priest C, Nicholas CR. Human pallial MGE-type GABAergic interneuron cell therapy for chron PMID 37802038
Identifiers
NCT: NCT05135091 · NTE001