NRTX-1001 Neural Cell Therapy for Epilepsy
Recruiting in Palo Alto (17 mi)
+22 other locations
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 1 & 2
Recruiting
Sponsor: Neurona Therapeutics
Must be taking: ASDs
Disqualifiers: Neurologic disease, Immunodeficiency, Psychiatric disorders, others
No Placebo Group
Trial Summary
What is the purpose of this trial?
This trial tests if injecting special brain cells into the brain can help patients with hard-to-treat epilepsy. The new cells release a calming chemical to reduce seizures. Patients will also take medicines to prevent their bodies from rejecting the new cells.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but it mentions that participants must be on stable doses of approved anti-seizure drugs for at least one month before joining.
What data supports the effectiveness of the NRTX-1001 treatment for epilepsy?
Research shows that stem cell therapies, like those using neural stem cells, have potential in treating epilepsy by integrating into brain circuits and possibly reducing seizures. Additionally, studies on similar stem cell treatments have shown promise in reducing seizure frequency in drug-resistant epilepsy.12345
How is the NRTX-1001 treatment different from other epilepsy treatments?
Eligibility Criteria
This trial is for adults aged 18 to 65 with drug-resistant mesial temporal lobe epilepsy. Participants must have a history of focal seizures, be on stable epilepsy medication doses, and have confirmed seizure focus in one temporal lobe. They should not have attempted suicide in the past year, severe psychiatric disorders, pregnancy or breastfeeding status, progressive neurological diseases other than epilepsy, significant medical conditions that impair participation, immunodeficiency or chronic intracranial devices.Inclusion Criteria
I have been on a stable dose of my seizure medication for at least a month.
I have had an average of 2 or more seizures every month for the last 6 months.
I still have seizures despite trying at least 2 different seizure medications.
See 4 more
Exclusion Criteria
Your MRI shows a possible cancerous growth.
My epilepsy is caused by a progressive neurological condition.
You tried to harm yourself in the last year.
See 5 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive a single stereotactic intracerebral administration of inhibitory nerve cells (NRTX-1001) and take immunosuppressive medicines for 1 year
1 year
Follow-up
Participants are monitored for safety, tolerability, neural cell viability, local inflammation, and effects on epilepsy symptoms
2 years
Regular MRI scans and assessments
Long-term follow-up
Participants are followed for an additional 13 years with quarterly phone contact and annual visits
13 years
Quarterly phone contact, annual visits
Treatment Details
Interventions
- NRTX-1001 (Neural Cell Therapy)
- Sham Comparator (Procedure)
Trial OverviewThe study tests NRTX-1001 neural cell therapy's safety and effectiveness against placebo (sham comparator) in reducing seizure frequency. It involves a single stereotactic injection of inhibitory nerve cells directly into the brain of subjects with unilateral mesial temporal lobe epilepsy who haven't responded to at least two anti-seizure drugs.
Participant Groups
3Treatment groups
Experimental Treatment
Placebo Group
Group I: NRTX-1001 (Stage 2)Experimental Treatment1 Intervention
Up to 20 subjects.
Group II: NRTX-1001 (Stage 1)Experimental Treatment1 Intervention
Up to 10 subjects.
Group III: Sham Comparator (Stage 2)Placebo Group1 Intervention
Up to 10 subjects.
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Mayo ClinicPhoenix, AZ
Oregon Health and Science UniversityPortland, OR
Mayo Clinic Arizona Epilepsy CenterPhoenix, AZ
Banner-University of Arizona Medical Center Tucson Comprehensive Epilepsy ProgramTucson, AZ
More Trial Locations
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Who Is Running the Clinical Trial?
Neurona TherapeuticsLead Sponsor
California Institute for Regenerative Medicine (CIRM)Collaborator
References
Antiepileptogenesis and disease modification: Clinical and regulatory issues. [2022]This is a summary report of clinical and regulatory issues discussed at the 2018 NINDS workshop, entitled "Accelerating Therapies for Antiepileptogenesis and Disease Modification." The intent of the workshop was to optimize and accelerate development of therapies for antiepileptogenesis (AEG) and disease modification in the epilepsies. The working group discussed nomenclature for antiepileptogenic therapies, subdividing them into "antiepileptogenic therapies" and "disease modifying therapies," both of which are urgently needed. We use the example of traumatic brain injury to explain issues and complexities in designing a trial for disease-preventing antiepileptogenic therapies, including identifying timing of intervention, selecting the appropriate dose, and the need for biomarkers. We discuss the recent trials of vigabatrin to prevent onset and modify epilepsy outcome in children with tuberous sclerosis (Epistop and PreVeNT). We describe a potential approach to a disease modification trial in adults, using patients with temporal lobe epilepsy. Finally, we discuss regulatory hurdles for antiepileptogenesis and disease-modifying trials.
Treatment of refractory epilepsy patients with autologous mesenchymal stem cells reduces seizure frequency: An open label study. [2018]Existing anti-epileptic drugs (AED) have limited efficiency in many patients, necessitating the search for alternative approaches such as stem cell therapy. We report the use of autologous patient-derived mesenchymal stem cells (MSC) as a therapeutic agent in symptomatic drug-resistant epilepsy in a Phase I open label clinical trial (registered as NCT02497443).
The Clinical Research Landscape of Pediatric Drug-Resistant Epilepsy. [2021]To characterize the clinical research landscape of pediatric drug-resistant epilepsy (DRE) with a focus on neurotechnology.
Stem cells as a potential therapy for epilepsy. [2022]Neural stem cells and neural progenitors (NSC/NPs) hold great promise in neuro-restorative therapy due to their remarkable capacity for self-renewal, plasticity, and ability to integrate into host brain circuitry. Some types of epilepsy would appear to be excellent targets for this type of therapy due to known alterations in local circuitry based on loss or malfunction of specific types of neurons in specific brain structures. Potential sources for NSC/NPs include the embryonic blastocyst, the fetal brain, and adult brain and non-neural tissues. Each of these cell types has potential strengths and weaknesses as candidates for clinical therapeutic agents. This article reviews some of the major types of NSC/NPs and how they have been studied with regard to synaptic integration into host brain circuits. It also reviews how these transplanted cells develop and interact with host brain cells in animal models of epilepsy. The field is still wide open with a number of very promising results but there are also some major challenges that will need to be addressed prior to considering clinical applications for epilepsy.
Interneuron Transplantation as a Treatment for Epilepsy. [2018]Stem-cell therapy has extraordinary potential to address critical, unmet needs in the treatment of human disease. One particularly promising approach for the treatment of epilepsy is to increase inhibition in areas of the epileptic brain by grafting new inhibitory cortical interneurons. When grafted from embryos, young γ-aminobutyric acid (GABA)ergic precursors disperse, functionally mature into host brain circuits as local-circuit interneurons, and can stop seizures in both genetic and acquired forms of the disease. These features make interneuron cell transplantation an attractive new approach for the treatment of intractable epilepsies, as well as other brain disorders that involve increased risk for epilepsy as a comorbidity. Here, we review recent efforts to isolate and transplant cortical interneuron precursors derived from embryonic mouse and human cell sources. We also discuss some of the important challenges that must be addressed before stem-cell-based treatment for human epilepsy is realized.
Commentary: the prospect of cell-based therapy for epilepsy. [2021]About 30% of patient with epilepsy do not respond to available antiepileptic drugs. In addition to seizure suppression, novel approaches are needed to prevent or alleviate epileptogenic process after various types of brain injuries. The use of cell transplants as factories to produce endogeneous anticonvulsants or as bricks to repair abnormal ictogenic and epileptogenic neuronal circuits has generated hope that cell-based therapies could become a novel therapeutic category in the treatment arsenal of epilepsy. Herein we summarize the current status and future perspectives of cell-based therapies in the treatment of epilepsy.
A Review on Hematopoietic Stem Cell Treatment for Epilepsy. [2022]Epilepsy responds to pharmacotherapy in its initial stages. The response of some forms of epilepsy, like the refractory epilepsy, is extremely low. Surgical management of epilepsy is associated with complications, which necessitates the search for novel and modern strategies for the treatment of epilepsy. Neuroprotection and neuronal regeneration are the major targets that must be accomplished by the new strategies. Hematopoietic Stem Cell (HSCs) therapy for epilepsy has shown promising results in pre-clinical studies with marginal clinical effects. This review explores the characteristics, mechanism of action, and clinical significance of HSCs therapy for the treatment of epilepsy.
Seizure suppression in amygdala-kindled mice by transplantation of neural stem/progenitor cells derived from mouse embryonic stem cells. [2019]Embryonic stem cells (ES cells) differentiate into multiple cell lineages including neural cells. The present study optimized the method to induce differentiation of gamma-aminobutyric acid-producing neurons (GABAergic neurons) from ES cell-derived neural stem/progenitor cells (NS/PCs), and transplanted these ES cell-derived GABAergic neurons producing neural progenitors into kindled epileptic mice, and analyzed the morphological and functional recovery from epilepsy. The response of kindling was evaluated by the modified Racine scale. Following stage 5 kindling, the mice were divided into two groups. Group 1 received NS/PCs derived from the ES cells ubiquitously expressing green fluorescent protein transplanted into the dorsal hippocampal area. Group 2 received microinjections of only the medium. After transplantation, the recovery of seizures was evaluated by the modified Racine scale again. All mice were perfused and fixed for immunohistochemical analysis after finishing the kindling experiment. In Group 1, one mouse was classified as stage 0, five as stage 3, and one as stage 4 recovering from stage 5 at 6 weeks after transplantation. In Group 2, all mice remained in stage 5. The transplanted cells were examined immunohistochemically using neuronal and GABAergic markers. In the transplanted mice, substantial hippocampal GABAergic re-innervation and seizure-suppressing effects were observed. NS/PCs derived from ES cells have high potential for use in transplantation therapy for clinically intractable epilepsies.