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

General Information
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.
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.
Clinical trials phase	Phase 2
Start date (estimated)	2024-11-14
End date (estimated)	2040-07-15
Clinical feature	Label epilepsy Link http://purl.obolibrary.org/obo/DOID_1826 Description A brain disease that is characterized by the occurrance of at least two unprovoked seizures resulting from a persistent epileptogenic abnormality of the brain that is able to spontaneously generate paroxysmal activity and typically manifested by sudden brief episodes of altered or diminished consciousness, involuntary movements, or convulsions.
Administrative Information
NCT number	NCT06422923
ICTRP weblink	https://trialsearch.who.int/Trial2.aspx?TrialID=NCT06422923
Other study identifiers	Name NTE002
Source weblink	https://clinicaltrials.gov/study/NCT06422923
Regulatory body approval	Name Food and Drug Administration (FDA) Country United States
Public contact	Email holly@neuronatx.com Public email holly@neuronatx.com First name Holly Last name Finefrock Phone +1 650-436-3045 City San Francisco Country United States
Sponsors	Neurona Therapeutics
Cells
Which differentiated cell type is used	Label interneuron Link http://purl.obolibrary.org/obo/CL_0000099 Description Most generally any neuron which is not motor or sensory. Interneurons may also refer to neurons whose axons remain within a particular brain region as contrasted with projection neurons which have axons projecting to other brain regions.
Recruitment
Recruitment Status	Recruiting
Estimated number of participants	10