FIH Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy

General Information

Summary This clinical trial is designed to test whether a single stereotactic intracerebral administration of inhibitory nerve cells into subjects with drug-resistant mesial temporal lobe epilepsy is safe (frequency of adverse events) and effective (seizure frequency).
Description Subjects will undergo a single stereotactic intracerebral administration of neural cells, called interneurons, that secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). Subjects will then take medicines to partially suppress their immune system (aimed to prevent the body from rejecting the cells) for 1 year. Safety, tolerability, evidence of neural cell viability and local inflammation (using MRI scans of the brain), and effects on epilepsy disease symptoms will be assessed for 2 years post-transplant. Subjects will be followed for an additional 13 years with quarterly phone contact and annual visits.
Clinical trials phase Phases 1/2
Start date (estimated) 2022-06-16
End date (estimated) 2026-05-31
Clinical feature
Label epilepsy
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 NCT05135091
ICTRP weblink
Other study identifiers
Name NTE001
Source weblink
Regulatory body approval
Name Food and Drug Administration (FDA)
United States
Public contact
Public email
First name Sheri
Last name Madrid
Phone +1 949-500-0027
United States
Sponsors Neurona Therapeutics


Which differentiated cell type is used
Label interneuron
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 Status Recruiting
Estimated number of participants 40