An Open-Label, Observational, Prospective, Multicenter Study to Evaluate the Long-Term Efficacy and Safety of Perampanel as Monotherapy in Subjects Age 4 Years and Older with Focal Onset Seizures: PORTABLE Study

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Observational study

Study drug and medical condition

• Name of medicine:
  FYCOMPA
• Study drug International non-proprietary name (INN) or common name:
  PERAMPANEL
• Anatomical Therapeutic Chemical (ATC) code:
  (N03AX22) perampanel

  perampanel
• Medical condition to be studied:
  Epilepsy

Population studied

• Short description of the study population:
  Inclusion Criteria; 1. Male or female, age 4 years or older at the time of informed consent (in the case of minors, consent from a legal representative [eg, one parent] is required); 2. Diagnosed with FOS (with or without FBTCS) according to International League Against Epilepy (ILAE) 2017 classification ; 3. Newly diagnosed or recurrent epilepsy. For subjects with recurrent epilepsy, they must have relapsed at least 2 years after the end of the last antiepileptic drug (AED) treatment.; 4. Subjects for whom the decision to initiate perampanel has been made, according to the judgment of the investigator; ; Exclusion Criteria; 1. A history of receiving any AED (including AED used as rescue treatment) for more than 2 weeks in total within 2 years before Day 0; 2. Previously treated with perampanel at any time ; 3. A history of hypersensitivity to any of the excipients of perampanel; 4. Severe hepatic impairment; 5. Subjects who have participated in a study involving administration of an investigational drug/biologics or device within 4 weeks or within approximately 5 half-lives of the investigational drug/biologics,whichever is longer, before screening.; 6. Not appropriate for the study according to the judgment of the investigator
• Age groups:
  Children (2 to < 12 years)

  Adults (18 to < 65 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)
• Estimated number of subjects:
  60

Study design details

• Study design:
  This is a non-interventional, prospective study in subjects with a diagnosis of FOS (with or without FBTCS), for whom the treating physician has made the decision to initiate perampanel as monotherapy.
• Main study objective:
  The primary objective of the study is to evaluate 24-month seizure freedom in response to perampanel monotherapy in subjects age 4 years and older with focal onset seizures (FOS) (with or without focal-to-bilateral tonic clonic seizures [FBTCS])
• Setting:
  Subjects will be identified by sites from the electronic/paper medical and pharmacy records of patients attending their usual epilepsy clinic. ; The clinical decision to initiate perampanel as monotherapy will already have been made before the subject is identified for the study – ie, the decision to add perampanel is independent of the decision to enroll the subject in the study. ; Before any study procedures, signed informed consent/assent form must be first obtained (or, in the case of children, the legal representative will sign consent on behalf of the subject). ; Subjects will then enter into Screening Period for eligibility determination. Demography and medical history (including epilepsy history and baseline seizure frequency based on retrospective collection from 3 months before Day 0 [day of first dose of perampanel]) will be collected. ; Subjects who have provided informed consent to participate in sleep measurements will also be provided with a HARU 1 wearable device and instructed in its use for recording of baseline sleep parameters. ; Subjects who are eligible will then enter the Treatment Period and perampanel monotherapy will be initiated. During the Treatment Period, data will be collected prospectively via 2 types of assessments: on site and off site
• Comparators:
  N/A
• Data analysis plan:
  Data will be analyzed according to the Statistical Analysis Plan (SAP). Data will be analyzed using SAS or other validated statistical software as necessary. Demography and medical history will be tabulated.; ; Study Endpoints; Primary Endpoint; 1. Pragmatic seizure free rate at 24 months; ; Secondary Endpoints; 1. The pragmatic seizure-free rate at 6, 12, 18, and 30 months; 2. The sustained seizure-free rate at 12 and 24 months; 3. Retention rate at 6, 12, 18, 24, and 30 months; 4. Incidence of TEAEs ; 5. Change from baseline in EQ-5D-5L and PedsQL; ; Exploratory Endpoints; 1. Change from baseline in total sleep time, sleep efficacy, sleep latency, WASO, and duration of sleep stage; 2. Study drug compliance rate; 3. Clinical factors to predict seizure freedom at 3, 12 and 24 months

Documents

Study report

e2007-m081-515CSRVD-red (2).pdf

Study publications

UMIN-CTR PORTABLE Study