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Phase 2 Completed N=21 Treatment

Pharmacokinetic Study With an Oral Suspension of Perampanel as Adjunctive Therapy in Pediatric Subjects With Epilepsy

Source: ClinicalTrials.gov NCT02914314 ↗
Enrolled (actual)
21
Serious AEs
33.3%
Results posted
Sep 2023
Primary outcomePrimary: Dose Normalized Area Under the Concentration-Time Curve for Dosing Interval at Steady State (AUCtau,ss) of Perampanel During the Maintenance Period of the Core Phase for Non-EIAED Participants — 3840; 6110; 6030; 5810 nanogram*hour per milliliter (ng*h/mL)

Summary

The purpose of this study is to evaluate the pharmacokinetics (PK) of perampanel during the Maintenance Period of the Core Study following oral suspension administration given as an adjunctive therapy in pediatric participants from 1 month to less than 4 years of age with epilepsy.

Outcome Measures

OutcomeResultp-value
PRIMARY
Dose Normalized Area Under the Concentration-Time Curve for Dosing Interval at Steady State (AUCtau,ss) of Perampanel During the Maintenance Period of the Core Phase for Non-EIAED Participants
3840; 6110; 6030; 5810
PRIMARY
Dose Normalized Maximum (Peak) Steady-state Concentration (Cmax,ss) of Perampanel During the Maintenance Period of the Core Phase for Non-EIAED Participants
305; 388; 386; 341
PRIMARY
Dose Normalized Average Steady-state Drug Concentration (Css,Av) of Perampanel During the Maintenance Period of the Core Phase for Non-EIAED Participants
160; 255; 251; 242
PRIMARY
Dose Normalized Minimum Observed Steady State Plasma Concentration (Cmin,ss) of Perampanel During the Maintenance Period of the Core Phase for Non-EIAED Participants
114; 203; 199; 203
PRIMARY
Dose Normalized AUCtau,ss of Perampanel During the Maintenance Period of the Core Phase for EIAED Participants
5220; 3300; 2510
PRIMARY
Dose Normalized Cmax,ss of Perampanel During the Maintenance Period of the Core Phase for EIAED Participants
341; 276; 201
PRIMARY
Dose Normalized Css,Av of Perampanel During the Maintenance Period of the Core Phase for EIAED Participants
217; 137; 105
PRIMARY
Dose Normalized Cmin,ss of Perampanel During the Maintenance Period of the Core Phase for EIAED Participants
172; 90.5; 69.2
SECONDARY
Core Phase and Extension Phase: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
3; 5; 9; 3; 4; 5
SECONDARY
Core Phase and Extension Phase: Mean Change From Baseline in Hematology Laboratory Parameters: Basophils, Eosinophils, Leukocytes, Lymphocytes, Monocytes, Neutrophils, and Platelets
0.03; -0.02; -0.06; 0.00; 0.03; 0.04
SECONDARY
Core Phase and Extension Phase: Mean Change From Baseline in Hematology Laboratory Parameter: Erythrocytes
0.44; 0.16; 0.10; -0.10; 0.27; 0.39
SECONDARY
Core Phase and Extension Phase: Mean Change From Baseline in Hematology Laboratory Parameter: Hemoglobin
14.00; 5.20; 3.14; -0.25; 10.00; 12.80
SECONDARY
Core Phase and Extension Phase: Mean Change From Baseline in Clinical Chemistry Laboratory Parameters: Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Gamma Glutamyl Transferase, Lactate Dehydrogenase
-2.25; -16.00; -13.71; 6.00; 36.50; -16.00
SECONDARY
Core Phase and Extension Phase: Mean Change From Baseline in Clinical Chemistry Laboratory Parameter: Bilirubin
-0.30; 0.64; -0.46; 0.70; -0.10; 1.24
SECONDARY
Core Phase and Extension Phase: Mean Change From Baseline in Clinical Chemistry Laboratory Parameters: Calcium, Chloride, Cholesterol, Glucose, Phosphate, Potassium, Sodium, Triglycerides, Urea Nitrogen
-0.08; 0.02; -0.00; -0.01; -0.05; 0.05
SECONDARY
Core Phase and Extension Phase: Mean Change From Baseline in Clinical Chemistry Laboratory Parameters: Creatinine, Direct Bilirubin, Urate
2.25; 1.80; -1.28; 4.51; 6.51; 3.61
SECONDARY
Core Phase and Extension Phase: Mean Change From Baseline in Clinical Chemistry Laboratory Parameters: Albumin, Globulin, Protein
0.50; 2.00; -0.29; -1.00; 2.00; 5.60
SECONDARY
Core Phase and Extension Phase: Mean Change From Baseline in Vital Sign Parameters: Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)
-1.00; 0.60; 0.33; 10.33; 27.00; 4.00
SECONDARY
Core Phase and Extension Phase: Mean Change From Baseline in Vital Sign Parameter: Pulse Rate
5.25; -4.40; -14.89; -21.67; -9.25; -15.80
SECONDARY
Core Phase and Extension Phase: Mean Change From Baseline in Vital Sign Parameter: Respiratory Rate
-5.33; -3.00; -4.78; -5.33; -4.33; -4.80
SECONDARY
Core Phase and Extension Phase: Mean Change From Baseline in Vital Sign Parameter: Body Temperature
0.10; -0.02; 0.02; 0.13; 0.47; 0.02
SECONDARY
Core Phase and Extension Phase: Mean Change From Baseline in Height
4.76; 5.20; 5.06; 3.63; 17.65; 12.78
SECONDARY
Core Phase and Extension Phase: Mean Change From Baseline in Weight
1.88; 1.20; 1.01; 0.77; 3.88; 3.24
SECONDARY
Core Phase and Extension Phase: Mean Change From Baseline in Head Circumference
1.87; 1.50; 1.14; -0.15; 4.02; 2.50
SECONDARY
Core and Extension Phase: Mean Change From Baseline in ECG Parameters: Single Beat Heart Rate-corrected QT Interval (QTcB), QT Interval Corrected According to the Formula of Fridericia (QTcF), PR and QT Intervals, QRS Duration, and Aggregate RR Interval
-9.0; -7.0; -3.8; 17.0; -29.75; -5.60
SECONDARY
Core Phase and Extension Phase: Mean Change From Baseline in ECG Parameter: ECG Ventricular Rate
-7.5; 8.6; -12.2; 18.7; -36.25; -4.40
SECONDARY
Core Phase and Extension Phase: Mean Change From Baseline in Thyroid Stimulating Hormone (TSH): Thyrotropin
1.12; -1.02; 1.33; -0.90; 0.05; -0.89
SECONDARY
Core Phase and Extension Phase: Mean Change From Baseline in Insulin-like Growth Factor 1 (IGF-1)
-2.00; 7.00; 3.60; 1.00; 6.00; 4.75
SECONDARY
Core Phase and Extension Phase: Mean Change From Baseline in Free Thyroxine, and Free Triiodothyronine
0.90; -1.30; -1.80; -3.90; 2.55; 0.83

Eligibility Criteria

Inclusion Criteria

  • Male or female, from 1 month to less than 4 years of age (and of at least 36 weeks gestational age) at the time of consent
  • Have a minimum weight of 4 kilograms (kg) (8.8 pounds [lb])
  • Have a diagnosis of epilepsy with any type of seizure according to the International League Against Epilepsy's (ILAE) Classification of Epileptic Seizures (1981). Diagnosis should have been established at least 2 weeks (≤6 months of age) or 4 weeks (>6 months of age) before Visit 1, by clinical history and an electroencephalogram (EEG) that is consistent with epilepsy; normal interictal EEGs will be allowed provided that the participant meets the other diagnosis criterion (i.e., clinical history)
  • Have had brain imaging (computed tomography [CT] or magnetic resonance imaging [MRI]) before Visit 1 that ruled out a progressive cause of epilepsy
  • Have had 1 or more seizure(s) before Visit 1
  • Currently being treated with a stable dose (i.e., unchanged for at least 5 half-lives) of 1 to a maximum of 4 antiepileptic drugs (AEDs) (at least 6, but not more than 8, in the age group of 1 to less than 24 months, and up to 13 subjects in the age group of 2 to less than 4 years, will be taking 1 EIAEDs [that is, carbamazepine (CBZ), oxcarbazepine (OXC), phenytoin (PHT), or eslicarbazepine (ESL)] out of the maximum of 4 AEDs. The remaining participants cannot be taking any EIAEDs).
  • Have been on their current concomitant AED(s) with a stable dose for at least 2 weeks or 5 half-lives, whichever is longer, before Visit 1
  • Must have discontinued all restricted medications (example, medications known to be inducers of cytochrome P450 3A) at least 2 weeks or 5 half-lives (whichever is longer) before Visit 1
  • If entering the Extension Phase, must have completed the last visit of the Maintenance Period of the Core Study

Exclusion Criteria

  • Have a history of status epilepticus that required hospitalization during the 3 months before Visit 1
  • Have seizures due to treatable medical conditions, such as those arising due to metabolic disturbances, toxic exposure, or an active infection
  • Have epilepsy secondary to progressive central nervous system (CNS) disease or any other progressive neurodegenerative disease, including tumors
  • Have had epilepsy surgery within 1 year of Visit 1
  • Are scheduled and/or confirmed to have epilepsy surgery within 6 months after Visit 1
  • Used intermittent rescue benzodiazepines (i.e., 1 to 2 doses over a 24-hour period considered one-time rescue) 2 or more times in the 2 weeks before Visit 1
  • Current use of felbamate, or any evidence of ongoing hepatic or bone marrow dysfunction associated with prior felbamate treatment. (Prior use of felbamate must be discontinued at least 8 weeks before Visit 1.)
  • Current use of vigabatrin or any evidence of clinically significant vision abnormality associated with prior vigabatrin treatment. (Prior use of vigabatrin must be discontinued at least 2 weeks before Visit 1.)
  • Are on ketogenic diet regimen that has not been stable for at least 4 weeks before Visit 1
  • Concomitant use of other drugs known to influence the CNS, (other than AEDs for epilepsy), where the dose has not been stabilized for at least 5 half-lives or 2 weeks, whichever is longer, before Visit 1
  • Have any concomitant illnesses/co-morbidities that could severely affect the participant's safety or study conduct
  • Have evidence of clinically significant disease (e.g., cardiac, respiratory, gastrointestinal, renal disease) that in the opinion of the investigator(s) could affect the participant's safety or study conduct
  • Have clinically significant laboratory abnormalities or any clinically acute or chronic disease
  • Have evidence of significant active hepatic disease. Stable elevation of liver enzymes, alanine aminotransferase (ALT) and aspartate aminotransferase (AST) due to concomitant medication(s), will be allowed if they are less than 3 times the upper limit of normal (ULN)
  • Have clinical evid
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02914314). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.

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