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

Launched by NEURONA THERAPEUTICS · May 15, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called NRTX-1001, which involves using special nerve cells to help people with a specific type of epilepsy known as drug-resistant bilateral mesial temporal lobe epilepsy (MTLE). This condition causes frequent seizures that are hard to control with traditional medications. The trial aims to find out if this treatment is safe and if it can help reduce the number of seizures in participants.

To be eligible, participants must be between 18 and 75 years old and have a history of focal seizures that start in the hippocampus, which is part of the brain. They should have tried at least two anti-seizure medications without success and have experienced several seizures in the months leading up to the trial. Participants will receive the treatment in both sides of their brain and will be closely monitored for safety and effectiveness. It's important to know that this trial is currently recruiting, and those interested should be able to read and understand English or Spanish to participate fully in the study.

Gender

ALL

Eligibility criteria

• Key Inclusion Criteria:
• • 1. Male or female, age 18-75 years.
• • 2. Subjects of childbearing potential will use highly effective contraception.
• • 3. 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).
• • 4. Either
• • 1. 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
• • 2. 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
• • 3. 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.
• • 5. 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.
• • 6. Subject has previously had adequate (in opinion of investigator) therapeutic trials of at least two Anti-Seizure Medicines (ASMs).
• • 7. 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.
• • 8. Subject can converse and read in English or Spanish. Able to participate in required study procedures and provide signed informed consent.
• Key Exclusion Criteria:
• • 1. Epilepsy due to other and/or progressive neurologic disease.
• • 2. Evidence of seizure focus outside hippocampus (either by seizure semiology or EEG findings).
• • 3. 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.
• • 4. Seizures of non-focal origin.
• • 5. 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.
• • 6. Psychogenic Non-Epileptic Seizures (PNES) within the past 3 years.
• • 7. Severe psychiatric disorders.
• • 8. Primary or secondary immunodeficiency.
• • 9. Pregnancy, or currently breastfeeding.
• • 10. Suicide attempts in past year.
• • 11. Significant other medical conditions which would impair safe participation.

Trial Officials