NTE002 Study
A Phase I/II Clinical Trial of NRTX-1001 Inhibitory Cell Therapy in Drug-Resistant Bilateral Mesial Temporal Lobe Epilepsy
NRTX-1001 is an investigational cell therapy (experimental cell therapy). An investigational cell therapy is one that the FDA and other health authorities have not yet approved to be safe and effective.
NRTX-1001 has undergone preclinical laboratory testing and received approval from the FDA to be tested in people.
NTE002 is the name of the clinical study designed to investigate the safety and efficacy of NRTX-1001 as a potential treatment for drug-resistant bilateral (meaning, on both sides) mesial temporal lobe epilepsy. Clinical sites involved in the study are currently screening adult patients in the United States for participation.
Purpose of the Study
This study is being conducted to gather information about the use of NRTX-1001 to treat seizures in people who have drug-resistant epilepsy that is confirmed to start in the temporal lobes of their brain.
Epilepsy occurs when nerve cells send out abnormally ‘excited’ signals. A certain naturally occurring neurotransmitter, or messenger in the brain, called Gamma Aminobutyric Acid (GABA) can reduce or block these excitatory signals. NRTX-1001 is a cell therapy of a specific type of nerve cell called an interneuron that releases GABA. These interneurons originate from human stem cells that have been developed and matured into interneurons.
It is hoped that through implantation of NRTX-1001, the interneurons that make up NRTX-1001 may work by naturally forming connections in the brain and releasing GABA that will quiet the nearby neurons and help limit the excitability signals, resulting in fewer seizures.
How are NRTX-1001 cells delivered?
What is NRTX-1001?
- NRTX-1001 is made from human stem cells that have been developed into interneurons (like the interneurons already in your brain).
- NRTX-1001 cells produce GABA, a brain neurotransmitter thought to inhibit seizure activity.
GABAergic interneuron cell therapy may restore missing inhibition and rebalance neural activity.
Qualifying for the Study
Are 18-75 years old
Have focal seizures, clinically defined as Temporal Lobe Epilepsy
Seizures haven’t been controlled by taking at least 2 seizure medications
Seizure origin confirmed to be coming from both hippocampi
Seizure frequency is at least 4 per 28-day period over the past 6 months
No prior exposure to gene or cell therapy treatments of any kind
Pre-existing electrode/device implants may be allowed
Study Expenses
Neurona will reimburse the study subject and caregiver for reasonable travel expenses to confirm their eligibility and discuss participation with their doctor.
Once enrolled, study subjects should not have to spend any of their own money to participate in the trial. All study costs are paid for by Neurona, including a service to help coordinate and pay for their travel arrangements.
Study Site Locations
Sites Currently Screening and Enrolling Patients
| State | City | Physician | Institution |
|---|---|---|---|
| CA | La Jolla | Jerry Shih, MD | University of California, San Diego Neurological Institute Epilepsy Center |
| CA | Palo Alto | Kevin Graber, MD | Stanford Comprehensive Epilepsy Center |
| CA | San Francisco | Robert Knowlton, MD | University of California, San Francisco |
| IL | Chicago | Peter Warnke, MD | University of Chicago Comprehensive Epilepsy Center |
| NC | Raleigh | Derek Southwell, MD, Ph.D. | Duke University Medical Center |
| NY | Syracuse | Shahram Izadyar, MD | SUNY Upstate Medical University |
Resources
Disease awareness, patient advocacy, diversity in clinical trials
Commitment to Diversity and Inclusion in the NTE002 Study
Neurona is committed to diversity, equity, and inclusion of all races, ethnicities, and genders in the NTE002 clinical trial. The diversity of our clinical trial participants will best inform safety and efficacy of our cells in all populations. If you choose to participate in this trial, you will represent the safety and efficacy of our study for your demographic and community.
