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

[...] eizures per 30-day period in the 3 months prior to screening, including at least 2 seizures during the Screening Period. On a stable type and dose regimen of up to a maximum of 4 approved Anti Seizure Drugs for at least 46 months prior to screening. Confirmed unilateral hippocampal [...] n devices will be up to discretion of the Investigator). Any other contraindications for generalized anesthesia or surgery or intra-operative magnetic resonance imaging (MRI). Medications that could confound clinical (e.g., antipsycho [...] ure or comply with the procedures and study visit schedule. BilateralAny seizures wi [...] [...]

[...] eizures per 30-day period in the 3 months prior to screening, including at least 2 seizures during the Screening Period. On a stable type and dose regimen of up to a maximum of 4 approved Anti Seizure Drugs for at least 64 months prior to screening. Confirmed unilateral hippocampal [...] n devices will be up to discretion of the Investigator). Any other contraindications for generalized anesthesia or surgery or intra-operative magnetic resonance imaging (MRI). Medications that could confound clinical (e.g., antipsycho [...] ure or comply with the procedures and study visit schedule. AnyBilateral seizuresor [...] [...]

Inclusion Criteria: Diagnosis of unilateral refractory MTLE History of seizures with onan average of ≥ 2 documented focal onset impaired awareness seizures or focal to bilateral tonic-clonic seizures per 30-day period in the 3 months prior to screening. On a stable type and do [...] nction, inconsistent with disease pathology- related MRI and/or (18F)FDG-PET findings. Women of childbearing potential (WOC [...] irth control consistently and correctly throughout the study and for at least 1 year following AMT-260 administration. For WOCBP only: Negative pregnancy test. Exclusion Criteri [...] viral therapy) and labo [...]

TheIn first-in-human Phase I/IIa U.S. trial consists of two parts. Thethis first partclinical is a multi-center, open-label trial with two dosing cohorts of six patients each to assess safety, tolerability, and first signs of efficacystudy of AMT-260, two dose levels will be studied to find the best dose of AMT-260. All eligible participants will receive AMT-260 at one of the two dose levels (i.e., there is no placebo in patientsthis with refractory unilateral MTLEstudy). TheAMT-260 secondis intended for a one-time administration, without the need to remove or destroy any part isof expectedthe [...] [...]

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

Inclusion Criteria: Adult, 18-65 years of age, inclusive, capable of giving informed consent. Diagnosis of unilateral refractory MTLE for ≥360 days, confirmed by an Epilepsy Monitoring Unit. History of seizures with on average ≥ 2 focal onset impaired awareness seizures per 30-day period duringin the Retrospective Period (3 months prior to screening). Currently onOn a stable type and dose regimen of up to a maximum of 4 approved ASDs,Anti Seizure Drugs for ≥3at least 6 months prior to the Retrospective Periodscreening. Confirmed unilateral hippocampal pathology and concordant unilateral seizur [...] [...]

SeizureTo Frequency Qualityevaluate first signs of Lifeefficacy inof Epilepsy InventoryAMT-31 (QOLIE 31) Patient Health Questionnaire (PHQ9) State Trait Anxiety Inventory (STAI) Pittsburgh Sleep Quality Index (PSQI) miRNA in human biofluid (copies/qPCR reaction)260. AAV9To vectorevaluate inthe humanbiodistribution biofluidproperties (copies/qPCRof reaction)AMT-260.

AdverseTo Eventsevaluate the safety and tolerability of AMT-260 in adults with unilateral refractory MTLE.

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