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).
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
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.
Sequential
AAV9-hSyn1-miGRIK2
GeneticAMT-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.
To evaluate the safety and tolerability of AMT-260 in adults with unilateral refractory MTLE.
Occurrence of Adverse Events during the period of 1 year after AMT-260 administration, including seriousness, severity, and causal relationship to AMT-260.
To evaluate first signs of efficacy of AMT-260.
Change in seizure frequency, comparing baseline to the 1 year period after AMT-260 administration.
To evaluate the biodistribution properties of AMT-260.
Blood, urine, saliva, and CSF samples will be collected and evaluated for vector DNA shedding at each timepoint.
To evaluate the long-term safety and efficacy of AMT-260 in adults with unilateral refractory MTLE.
Occurrence of Adverse Events during the period of 1 to 5 years after AMT-260 administration, including seriousness.
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