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).
In this first clinical study 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 this study). AMT-260 is intended for a one-time administration, without the need to remove or destroy any part of the brain. Participation in this study would not prevent later pursuit of other treatment options. Participants will be monitored through study site visits, telephone calls, blood tests, and questionnaires about how their seizures affect daily life. Participants will record seizures using an electronic seizure diary.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
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.
University of Alabama at Birmingham
Birmingham, Alabama, United States
RECRUITINGTo 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.
Time frame: 1 year
To evaluate first signs of efficacy of AMT-260.
Change in seizure frequency, comparing baseline to the 1 year period after AMT-260 administration.
Time frame: 1 year
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.
Time frame: 1 year
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Mayo Clinic Arizona
Phoenix, Arizona, United States
RECRUITINGStanford University
Palo Alto, California, United States
RECRUITINGMayo Clinic Florida
Jacksonville, Florida, United States
RECRUITINGKansas University Medical Center
Kansas City, Kansas, United States
RECRUITINGJohns Hopkins School of Medicine
Baltimore, Maryland, United States
RECRUITINGMidatlantic Epilepsy and Sleep Center
Bethesda, Maryland, United States
RECRUITINGMassachusetts General Hospital
Boston, Massachusetts, United States
RECRUITINGCorewell Health
Grand Rapids, Michigan, United States
RECRUITINGDartmouth Hitchcock Medical Center
Lebanon, New Hampshire, United States
RECRUITING...and 8 more locations