Clinical trial NCT03793868: Perampanel Single Ascending Dose Transcranial Magnetic Stimulation Biomarker Study in Amyotrophic Lateral Sclerosis
Official title: Perampanel Single Ascending Dose Transcranial Magnetic Stimulation Biomarker Study in Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: EARLY_PHASE1
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: Mayo Clinic
Sponsor class: OTHER
First posted: 2019-01-04
Enrollment: 22
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: This is a pilot study for the assessment of transcranial magnetic stimulation as a biomarker for ALS and to obtain information to guide a dose selection for future biomarker driven Phase 2 studies of Perampanel in ALS.
Primary purpose: OTHER

Brief summary: To evaluate if transcranial magnetic stimulation can be used as a biomarker in Amyotrophic Lateral sclerosis (ALS).

Detailed description: To measure the effect on motor threshold (MT) by transcranial magnetic stimulation (TMS) after a single dose of perampanel at two dose levels.

Eligibility criteria: Inclusion Criteria:

1. A probable laboratory supported, probable or definitive ALS diagnosis by revised El Escorial criteria.
2. Sporadic or familial ALS.
3. Ages of 18-70.
4. Agree to use reliable contraception
5. Randomization will occur after a baseline MT has been established; any subject in whom a MT cannot be established will be excluded.
6. Caregiver willing to report adverse behavioral events. -

Exclusion Criteria:

1. History of epilepsy.
2. Significant laboratory abnormality (AST or alanine aminotransferase \>3x upper limit of normal, or glomerular filtration rate \<60)
3. History of aggressive behavior.
4. Subject unwilling to abstain from alcohol for 2 weeks after each dosing.
5. History of drug abuse in the last 5 years
6. Other severe medical conditions, including psychiatric conditions, which would cause an increased risk in the opinion of the investigator, including but not limited to renal failure and liver failure.
7. Skull defect or other physical contraindication for TMS
8. Pacemaker or implanted defibrillator
9. Inability to take study capsule by mouth

Females only: Subject is pregnant \[as confirmed by a positive serum human chorionic gonadotropin (hCG) test for females of reproductive potential (FRP) only\], subject is breastfeeding, or subject is of reproductive potential and does not agree to follow use of reliable contraception.

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Primary outcomes:
measure: Motor threshold (MT)
timeFrame: at 4 hours post dose
Secondary outcomes:
Other outcomes:
Locations:
city: Jacksonville
country: United States
facility: Mayo Clinic
state: Florida
Study officials:
affiliation: Mayo Clinic
name: Bjorn E Oskarsson, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Oskarsson B, Mauricio EA, Shah JS, Li Z, Rogawski MA. Cortical excitability threshold can be increased by the AMPA blocker Perampanel in amyotrophic lateral sclerosis. Muscle Nerve. 2021 Aug;64(2):215-219. doi: 10.1002/mus.27328. Epub 2021 May 27.