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Recruiting Phase 1/2

AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy

NCT06063850 · tracked via the Priya Life Science USA tracker
Phase
Phase 1/2
Started
2024-06-12
Last updated
2026-08-24

Condition(s) studied

Mesial Temporal Lobe Epilepsy

Investigational drug(s) / intervention(s)

AAV9-hSyn1-miGRIK2

AAV9-hSyn1-miGRIK2: AMT-260 is an AAV9 gene therapy product that locally delivers miRNA silencing technology to target the GRIK2 gene and suppress aberrantly expressed GluK2 containing kainate receptors. Intervention will be a one-time intracerebral administration of AMT-260.

Study summary

The main goals of this clinical study are to learn if AMT-260 is safe and tolerable and works to reduce the frequency of seizures in adults with unilateral mesial temporal lobe epilepsy (MTLE).

Eligibility

Sex
ALL
Min age
18 Years
Max age
75 Years
Healthy volunteers
No
Inclusion Criteria: * Diagnosis of unilateral refractory MTLE * History of seizures with an average of ≥ 2 documented focal impaired awareness seizures or focal to bilateral tonic-clonic seizures per 30-day period over the 3 month Retrospective Period. * On a stable type and dose regimen of up to a maximum of 4 approved Anti Seizure Drugs for at least 1 month prior to the Retrospective Period. * Confirmed unilateral hippocampal pathology and concordant unilateral seizure focus * No evidence of focal neurocognitive dysfunction, inconsistent with disease pathology- related MRI and/or (18F)FDG-PET findings. * Women of childbearing potential (WOCBP) and fertile male subjects must be willing and able to use highly effective methods of birth control consistently and correctly throughout the study. * For WOCBP only: Negative pregnancy test. Exclusion Criteria: * Implanted devices that would contraindicate MRI; MRI-compatible devices must be implanted ≥3 months prior to Screening (vagus nerve stimulation devices will be up to discretion of the Investigator). * Any other contraindications to general anesthesia, surgery, or intra-operative magnetic resonance imaging (MRI). * Medications that could confound clinical (e.g., antipsychotic medication and anti-viral therapy) and laboratory evaluations or could affect a participant's safety or their ability to undergo the neurosurgical procedure or comply with the procedures and study visit schedule. * Bilateral or multifocal epilepsy; or inability to confidently lateralize seizure onset. * Dementia or other progressive neurological disorders and progressive brain lesions. * Previous major disease-unrelated neurosurgical intervention due to intracranial tumor, trauma, or bleeding and/or history of previous intracranial surgery for treatment of epileptic seizures, not including diagnostic stereo-EEG. * MRI evidence of epileptogenic, extra-temporal lesions, or dual temporal lobe pathology. * Status epilepticus within the year prior to Screening.

Primary outcome measure(s)

  • To evaluate the safety and tolerability of AMT-260 in adults with unilateral refractory MTLE. — 1 year
    Occurrence of Adverse Events during the period of 1 year after AMT-260 administration, including seriousness, severity, and causal relationship to AMT-260.

Trial sites (18)

FacilityCityRegionStatus
University of Alabama at Birmingham Birmingham Alabama Recruiting
Mayo Clinic Arizona Phoenix Arizona Recruiting
Stanford University Palo Alto California Completed
Mayo Clinic Florida Jacksonville Florida Recruiting
Kansas University Medical Center Kansas City Kansas Recruiting
Johns Hopkins School of Medicine Baltimore Maryland Recruiting
Midatlantic Epilepsy and Sleep Center Bethesda Maryland Completed
Massachusetts General Hospital Boston Massachusetts Recruiting
Corewell Health Grand Rapids Michigan Recruiting
Dartmouth Hitchcock Medical Center Lebanon New Hampshire Recruiting
Northeast Regional Epilepsy Group Hackensack New Jersey Recruiting
Robert Wood Johnson Hospital New Brunswick New Jersey Recruiting
Cleveland Clinic Cleveland Ohio Recruiting
Ohio State University Columbus Ohio Recruiting
Hospital of the University of Pennsylvania Philadelphia Pennsylvania Recruiting
Allegheny Health Network Pittsburgh Pennsylvania Recruiting
Baylor Scott & White Medical Center Austin Texas Recruiting
Medical College of Wisconsin Milwaukee Wisconsin Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06063850 on ClinicalTrials.gov ↗ ← All trials in the USA