NCT06422923 · Neurona Therapeutics
A Phase 1/2 Study of NRTX-1001 Neuronal Cell Therapy in Drug-Resistant Bilateral Mesial Temporal Lobe Epilepsy (MTLE)
What this study is about
This is a conducted at multiple hospitals, single treatment group$1, open label clinical trial that is designed to test the safety and preliminary effectiveness of single administration inhibitory nerve cells called interneurons (NRTX-1001), into both temporal lobes of subjects with drug-resistant bilateral mesial temporal lobe epilepsy.
View original scientific description
This is a multicenter, single arm, open label clinical trial that is designed to test the safety and preliminary efficacy of single administration inhibitory nerve cells called interneurons (NRTX-1001), into both temporal lobes of subjects with drug-resistant bilateral mesial temporal lobe epilepsy.
Interventions
BIOLOGICAL
NRTX-1001
Biological: NRTX-1001 is an inhibitory neural cell therapy investigational product. It is derived from a human stem cell line that has been converted into high-purity inhibitory interneurons that produce GABA. NRTX-1001 is intended to persist long-term and not require repeated administration.
Primary outcome measures
Incidence of Serious Adverse Events at end of month 12
Time frame: 12 months after treatment
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Male or female, age 18-75 years.
- Subjects of childbearing potential will use highly effective contraception.
- Proven history of focal seizures of hippocampal origin with bilateral seizure foci confirmed by scalp or intracranial ictal EEG (including confirmation by recordings from responsive neurostimulation \[RNS\] electrodes when applicable).
- bilateral hippocampal sclerosis on MRI (evidenced by increased FLAIR signal intensity in both hippocampi or by visual assessment showing reduced volume compared to normal) or
- bilateral temporal hypometabolism on 18-Flourodeoxyglucose Positron Emission Tomography (FDG PET) (assessed by visual assessment, comparing temporal regions to frontal/parietal lobe neocortex). In this case, ictal EEG recordings must also include intracranial confirmation. or
- a combination of unilateral instances of the evidence described in a. and b. (e.g., one side can be evidenced by criterion a. and the other side by criterion b.) MRI or PET scans used for assessment must have been acquired within 3 years of screening.
- Subject has had at least four clinical focal seizures, including at least two clinical focal seizures with objective manifestations, on average, per month for the 6 months prior to screening.
- Subject has previously had adequate (in opinion of investigator) therapeutic trials of at least two Anti-Seizure Medicines (ASMs).
- Current ASM regimen, and doses of other drugs known to affect seizure frequency (e.g., antidepressants), have been stable for at least three months prior to enrollment.
- Subject can converse and read in English or Spanish. Able to participate in required study procedures and provide signed informed consent. Key
Exclusion criteria
- Epilepsy due to other and/or progressive neurologic disease.
- Evidence of seizure focus outside hippocampus (either by seizure semiology or EEG findings).
- MRI indicating potential malignant lesion (low-grade glioma of any type is excluded) in any location or non-malignant potentially epileptogenic lesion outside the hippocampus. Small (\<2 cm) non invasive meningioma, remote from the affected temporal lobe, is not exclusionary.
- Seizures of non-focal origin.
- History of status epilepticus in the year prior to screening, as guided by ILAE criteria (Trinka 2015) in the judgement of the PI. A history of cluster seizures is permitted.
- Psychogenic Non-Epileptic Seizures (PNES) within the past 3 years.
- Severe psychiatric disorders.
- Primary or secondary immunodeficiency.
- Pregnancy, or currently breastfeeding.
- Suicide attempts in past year.
- Significant other medical conditions which would impair safe participation.
Where
- Little Rock, Arkansas
- Los Angeles, California
- Orange, California
- Palo Alto, California
- San Diego, California
- San Francisco, California
- Aurora, Colorado
- Chicago, Illinois
- Iowa City, Iowa
- Syracuse, New York
- Charlotte, North Carolina
- Durham, North Carolina
And 5 more locations — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jan 7, 2026 · Source of record for eligibility and locations