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Epilepsy, Temporal LobePhase 1/2NCT06422923

A Phase 1/2 Study of NRTX-1001 Neuronal Cell Therapy in Drug-Resistant Bilateral Mesial Temporal Lobe Epilepsy (MTLE)

A Phase 1/2 study testing NRTX-1001 in people with Epilepsy, Temporal Lobe, run by Neurona Therapeutics at 17 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

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. Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Epilepsy, Temporal Lobe
What is being tested?
NRTX-1001
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
17 sitesUnited States
Study dates
14 Nov 2024to 15 Jun 2042
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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).
  • Either
  • 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.
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  • 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.

Who cannot join

  • 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.
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  • 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.

Trial locations and contacts

17 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Neurona MedInfo contact

650-580-3825

neuronamedinfo@neuronatx.com

Eduardo Dunayevich, MD contact

650-436-3045

edunayevich@neuronatx.com

17 sites in 1 country

United States17 sites, 16 recruiting

  • University of Colorado Anschutz Medical Campus

    Aurora, Colorado, 80045

    Pamela D Gerecht, Ph.D. SC, 303-724-4134, pamela.davidgerecht@cuanschutz.edu

    Steven Ojemann, MD

    Recruiting

  • Atrium Health

    Charlotte, North Carolina, 28204

    Dianelis Diaz, RN, BSN, CRC, 704-446-1900, dianelis.diaz@atriumhealth.org

    Rajdeep Singh, MD

    Recruiting

  • UVA Health University Medical Center

    Charlottesville, Virginia, 22903

    Kimberlee E Meegan, BS, CRC III, 434-243-2040, dxh5dv@uvahealth.org

    Nathan Fountain, MD

    Recruiting

  • University of Chicago

    Chicago, Illinois, 60637

    Agnieszka Stadnik, MS, 773-702-8996, astadnik@bsd.uchicago.edu

    Peter Warnke, MD

    Recruiting

  • Duke University Hospital

    Durham, North Carolina, 27710

    Hazani Benitez-Rosas, 919-681-4974, Hazani.benitez-rosas@duke.edu

    Derek Southwell, MD, PhD

    Recruiting

  • UTHealth Houston

    Houston, Texas, 77030

    Eliana M Klier, PhD, 713-500-5442, Eliana.Klier@uth.tmc.edu

    Nitin Tandon, MD

    Recruiting

  • University of Iowa Health Care

    Iowa City, Iowa, 52242

    Loraine Brenner, MSN, 319-356-4361, loraine-brenner@uiowa.edu

    Mark Granner, MD

    Recruiting

  • University of Arkansas for Medical Sciences

    Little Rock, Arkansas, 72205

    Shellah Rogers, Nurse, 501-398-8622, Scrogers2@uams.edu

    Sisira Yadala, MD

    Recruiting

  • University of Southern California Keck Hospital

    Los Angeles, California, 90033

    Zoe B Durkin, SC, 323-422-6625, zoe.durkin@med.usc.edu

    Charles Y Liu, MD, PhD

    Recruiting

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

    Sarah Young, MS, 414-955-0989, scyoung@mcw.edu

    Sean Lew, MD

    Recruiting

  • UC Irvine Medical Center

    Orange, California, 92868

    UCI Alpha Clinic, 949-824-3990

    Mona Sazgar, MD

    Recruiting

  • Stanford University

    Palo Alto, California, 94304

    Jordan Seliger, CRC, MA, 650-460-9260, jseliger@stanford.edu

    Kevin Graber, MD

    Recruiting

  • Oregon Health and Science University

    Portland, Oregon, 97239

    Claire Dorfman, BA, 971-413-9201, dorfman@ohsu.edu

    David C Spencer, MD

    Recruiting

  • University of California San Diego

    San Diego, California, 92037

    Mateo Santizo, CRC, (858)583-0929, msantizo@health.ucsd.edu

    Jerry J Shih, MD

    Recruiting

  • University of California San Francisco

    San Francisco, California, 94143

    Withdrawn

  • SUNY Upstate Medical University

    Syracuse, New York, 13210

    Lena F Deb, BA, 315-464-9756, debl@upstate.edu

    Shahram Izadyar, MD

    Recruiting

  • Wake Forest Baptist Medical Center

    Winston-Salem, North Carolina, 27157

    Carolyn W Hedrick, RMA, CCRP, 336-716- 8694, Carolyn.Hedrick@AdvocateHealth.org

    Gautam S Popli, MD, MBA

    Recruiting

Other trials in United States

About this study

Official title: A Phase 1/2 Study of NRTX-1001 Neuronal Cell Therapy in Drug-Resistant Bilateral Temporal Lobe Epilepsy (MTLE)

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.

Read the detailed description

This is a multicenter, single arm, open-label study of NRTX-1001 in subjects with drug-resistant bilateral MTLE, with the objective of evaluating safety and preliminary efficacy in reducing seizure frequency. The subjects will undergo a single stereotactic CT or MRI-guided intracerebral administration of human interneurons into both temporal lobe regions of the brain. NRTX-1001 secretes the inhibitory neurotransmitter gamma-aminobutyric acid (GABA), which is intended to suppress the onset and spread of the seizures. Safety, tolerability, and effects on epilepsy disease symptoms will be assessed at approximately quarterly 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 15.

Being tested
NRTX-1001
Keywords
Bilateral Temporal Lobe Epilepsy, Bilateral MTLE, Refractory Bilateral MTLE
Medical subject headings
Epilepsy, Temporal Lobe

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • NRTX-1001Biological

    Also called GABA-secreting interneurons

    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.

Study groups in NCT06422923
Study groupRoleWhat they receive
Treatment ArmExperimentalThis is an open-label study of the bilateral intrahippocampal administration of NRTX-1001 in a single dose cohort of up to 10 subjects with drug-resistant bilateral MTLE.
Receives: Biological: NRTX-1001

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of Serious Adverse Events at end of month 12

    Measured: 12 months after treatment

Secondary outcomes (1)

  1. Change in frequency of clinical seizures

    Seizure frequency change during months 7-12, compared to frequency of clinical seizures in the 6-month baseline period.

    Measured: 12 months after treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted21 May 2024
  2. Study start14 Nov 2024
  3. Primary completion15 Jun 2027 (estimated)
  4. Study completion15 Jun 2042 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06422923
Sponsor's protocol ID
NTE002
Status last verified
Jan 2026
First submitted
15 May 2024
Last updated
7 Jan 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing NRTX-1001 in people with Epilepsy, Temporal Lobe, run by Neurona Therapeutics at 17 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 7 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 17 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Neurona MedInfo (650-580-3825, neuronamedinfo@neuronatx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of Serious Adverse Events at end of month 12 (time frame: 12 months after treatment). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06422923.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Jan 2026.

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