A Phase 1/2 Study of NRTX-1001 Neuronal Cell Therapy in Drug-Resistant Bilateral Temporal Lobe Epilepsy (MTLE)
Neurona Therapeutics
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 assesse…
Eligibility
- Age range
- 18–75 years
- Sex
- All
- Healthy volunteers
- No
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 hypo…
Interventions
- BiologicalNRTX-1001
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.
Locations (17)
- University of Arkansas for Medical SciencesLittle Rock, Arkansas
- University of Southern California Keck HospitalLos Angeles, California
- UC Irvine Medical CenterOrange, California
- Stanford UniversityPalo Alto, California
- University of California San DiegoSan Diego, California
- University of California San FranciscoSan Francisco, California