Perampanel Single Ascending Dose Transcranial Magnetic Stimulation Biomarker Study in Amyotrophic Lateral Sclerosis

NCT03793868 | Completed Early Phase 1 FDA Drug

• 1 other identifier: 17-002396
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• Motor threshold (MT)

  Transcranial Magnetic stimulation motor threshold change from baseline

  at 4 hours post dose

Study Arms (3)

Low dose

EXPERIMENTAL

Perampanel 4mg PO x1

Drug: Perampanel

Placebo

PLACEBO COMPARATOR

Receiving placebo

Other: Placebo

High dose

EXPERIMENTAL

Perampanel 8 mg PO x1

Drug: Perampanel

Interventions

Perampanel DRUG

Oral tablet

Also known as: Fycompa

High dose; Low dose

Placebo OTHER

Placebo (not drug)

Placebo

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• History of epilepsy.
• Significant laboratory abnormality (AST or alanine aminotransferase \>3x upper limit of normal, or glomerular filtration rate \<60)
• History of aggressive behavior.
• Subject unwilling to abstain from alcohol for 2 weeks after each dosing.
• History of drug abuse in the last 5 years
• 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.
• Skull defect or other physical contraindication for TMS
• Pacemaker or implanted defibrillator
• 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.

Sponsors & Collaborators

• Mayo Clinic: lead

Study Sites (1)

Mayo Clinic

Jacksonville, Florida, 32224, United States

Location

Related Publications (1)

• 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.

  PMID: 34008857 BACKGROUND

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

perampanel

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Bjorn E Oskarsson, MD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Matching placebo
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Model Details: 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.

Study Record Dates

• First Submitted: December 28, 2018
• First Posted: January 4, 2019
• Study Start: December 4, 2018
• Primary Completion: August 1, 2020
• Study Completion: September 1, 2020
• Last Updated: February 22, 2024

Record last verified: 2024-02

Locations

US (1)