NCT05135091

Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (EPIC (EPIlepsy Cell Therapy))

A Study of Inhibitory Interneurons (NRTX-1001) in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (MTLE)

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 88 (estimated)
• Sponsor: Neurona Therapeutics · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This clinical trial is designed to test whether a single image-guided intracerebral administration of inhibitory nerve cells, called interneurons (NRTX-1001), into subjects with drug-resistant unilateral mesial temporal lobe epilepsy (MTLE), with or without mesial temporal sclerosis (MTS), is safe (frequency of adverse events) and effective (seizure frequency). NRTX-1001 comprises human interneurons that secrete a neurotransmitter, gamma-aminobutyric acid (GABA).

Detailed description

Subjects will undergo a single CT or MRI-guided intracerebral administration of human interneurons that secrete the inhibitory neurotransmitter, gamma-aminobutyric acid (GABA), into the temporal lobe region of the brain where the seizures are thought to arise. NRTX-1001 is intended to suppress the onset and spread of seizures. Safety, efficacy, tolerability, and effects on reducing seizure frequency and epilepsy disease symptoms will be assessed at regular 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 5, and then annual visits in years 6 through 15. Subjects will be placed on an immunosuppressant medication regimen for a duration of one year to partially suppress the subjects' immune system to promote the intended long-term persistence of NRTX-1001. This immunosuppressant medication is intended to be discontinued after the first year; however, the NRTX-1001 cells are intended to persist long-term.

Conditions

• Mesial Temporal Lobe Epilepsy

Interventions

Type	Name	Description
BIOLOGICAL	NRTX-1001	Biological: NRTX-1001 is an experimental neural cell therapy product candidate derived from an allogeneic human embryonic stem cell line. The stem cells were converted into inhibitory nerve cells that produce GABA.
BIOLOGICAL	NRTX-1001	Biological: NRTX-1001 is an experimental neural cell therapy product candidate derived from an allogeneic human embryonic stem cell line. The stem cells were converted into inhibitory nerve cells that produce GABA.
PROCEDURE	Sham Comparator	Sham Comparator

Timeline

• Start date: 2022-06-16
• Primary completion: 2029-02-01
• Completion: 2043-09-01
• First posted: 2021-11-26
• Last updated: 2026-03-30

Locations

33 sites across 1 country: United States

Regulatory

• FDA-regulated drug study