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A study to evaluate efficacy and safety of perampanel when administered together with other current antiepileptic medications in children with epilepsy
EU CTIS ID: 2023-507794-17-00
What this study is testing
To evaluate the efficacy of perampanel as measured by the 50% responder rate during the Maintenance Period of the Core Study for seizure frequency (percent of subjects with at least 50% reduction in their seizure frequency per 28 days compared to baseline) in each cohort and each epileptic syndrome.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male or female subject, from age 1 month to less than 18 years for Cohort 1, or age 1 month to less than 2 years for Cohort 2 at the time of informed consent/assent. Subjects below the age of 1 year must have been at least 36 weeks of gestational age at birth. (revised per Amendments 01 and 02)
- Have a diagnosis of epilepsy with a pediatric epileptic syndrome (Cohort 1) or epilepsy with POS with or without secondary generalization (Cohort 2) according to the International League against Epilepsy (ILAE) Classification of Epileptic Seizures (1981). The eligibility criteria for the 2 cohorts are not mutually exclusive as a subject may experience POS as part of an epilepsy syndrome; these subjects are eligible for Cohort 1 if: (1) they are 2 to less than 18 years of age; or (2) they are 1 month to less than 2 years of age AND Cohort 2 is filled. Diagnosis must have been established by clinical history and an EEG that is consistent with the diagnosis; normal interictal EEGs will be allowed provided that the subject meets the other diagnosis criterion (ie, clinical history).
- Subjects must have had equal or greater than 4 seizures over the 4-week interval prior to Visit 2. For Cohort 1, all seizures except simple POS without motor signs are counted toward this inclusion. For Cohort 2, only simple POS with motor signs, complex POS, and complex POS with secondary generalization are counted toward this inclusion for POS.
- Absence of any progressive cause of epilepsy that has been confirmed clinically or based on brain imaging (eg, magnetic resonance imaging [MRI] scan, computed tomography [CT], or ultrasound [only for subjects <1 year old]). (revised per Amendment 02).
- Are currently being treated with stable doses of 1 to a maximum of 4 approved AEDs. A prescription medical marijuana (including products containing cannabidiol) is counted as 1 of the maximum of 4 allowed AEDs; however, it cannot be the only concomitant AED if this product is not an approved AED in the country where the study site is located. Doses must be stable for at least 4 weeks (at least 2 weeks for subjects <6 months old) before Visit 1; only 1 enzyme-inducing AED (EIAED) (defined as carbamazepine, phenytoin, oxcarbazepine, or eslicarbazepine) out of the maximum of 4 AEDs is allowed. (revised per Amendment 01).
You likely can't join if
- Females who are breastfeeding or pregnant at Screening/Baseline (as documented by a positive beta-human chorionic gonadotropin [β-hCG] or human chorionic gonadotropin [hCG] test with a minimum sensitivity of 25 IU/L or equivalent units of β-hCG [or hCG]). A separate baseline assessment is required if a negative screening pregnancy test was obtained more than 72 hours before the 1st dose of study drug.
- Evidence of significant active hepatic disease. Stable elevation of liver enzymes alanine aminotransferase (ALT) and aspartate aminotransferase (AST) due to concomitant medications, will be allowed if they are less than 3 times the upper limit of normal (ULN).
- Evidence of significant active hematological disease; white blood cell (WBC) count equal or less than 2500/μL (2.50 1E+09/L), or an absolute neutrophil count equal or less than 1000/μL (1.00 1E+09/L).
- Clinically significant ECG abnormality, including Fridericia prolonged corrected QT interval (QTcF) defined as greater than 450 msec.
- Have a progressive central nervous system (CNS) disease, including degenerative CNS diseases and progressive tumors.
- Multiple drug allergies or a severe drug reaction to AEDs, including dermatological (eg, Stevens-Johnson syndrome), hematological, or organ toxicity reactions.
See the full eligibility criteria
Who can join
- Male or female subject, from age 1 month to less than 18 years for Cohort 1, or age 1 month to less than 2 years for Cohort 2 at the time of informed consent/assent. Subjects below the age of 1 year must have been at least 36 weeks of gestational age at birth. (revised per Amendments 01 and 02)
- Have a diagnosis of epilepsy with a pediatric epileptic syndrome (Cohort 1) or epilepsy with POS with or without secondary generalization (Cohort 2) according to the International League against Epilepsy (ILAE) Classification of Epileptic Seizures (1981). The eligibility criteria for the 2 cohorts are not mutually exclusive as a subject may experience POS as part of an epilepsy syndrome; these subjects are eligible for Cohort 1 if: (1) they are 2 to less than 18 years of age; or (2) they are 1 month to less than 2 years of age AND Cohort 2 is filled. Diagnosis must have been established by clinical history and an EEG that is consistent with the diagnosis; normal interictal EEGs will be allowed provided that the subject meets the other diagnosis criterion (ie, clinical history).
- Subjects must have had equal or greater than 4 seizures over the 4-week interval prior to Visit 2. For Cohort 1, all seizures except simple POS without motor signs are counted toward this inclusion. For Cohort 2, only simple POS with motor signs, complex POS, and complex POS with secondary generalization are counted toward this inclusion for POS.
- Absence of any progressive cause of epilepsy that has been confirmed clinically or based on brain imaging (eg, magnetic resonance imaging [MRI] scan, computed tomography [CT], or ultrasound [only for subjects <1 year old]). (revised per Amendment 02).
- Are currently being treated with stable doses of 1 to a maximum of 4 approved AEDs. A prescription medical marijuana (including products containing cannabidiol) is counted as 1 of the maximum of 4 allowed AEDs; however, it cannot be the only concomitant AED if this product is not an approved AED in the country where the study site is located. Doses must be stable for at least 4 weeks (at least 2 weeks for subjects <6 months old) before Visit 1; only 1 enzyme-inducing AED (EIAED) (defined as carbamazepine, phenytoin, oxcarbazepine, or eslicarbazepine) out of the maximum of 4 AEDs is allowed. (revised per Amendment 01).
What rules you out
- Females who are breastfeeding or pregnant at Screening/Baseline (as documented by a positive beta-human chorionic gonadotropin [β-hCG] or human chorionic gonadotropin [hCG] test with a minimum sensitivity of 25 IU/L or equivalent units of β-hCG [or hCG]). A separate baseline assessment is required if a negative screening pregnancy test was obtained more than 72 hours before the 1st dose of study drug.
- Evidence of significant active hepatic disease. Stable elevation of liver enzymes alanine aminotransferase (ALT) and aspartate aminotransferase (AST) due to concomitant medications, will be allowed if they are less than 3 times the upper limit of normal (ULN).
- Evidence of significant active hematological disease; white blood cell (WBC) count equal or less than 2500/μL (2.50 1E+09/L), or an absolute neutrophil count equal or less than 1000/μL (1.00 1E+09/L).
- Clinically significant ECG abnormality, including Fridericia prolonged corrected QT interval (QTcF) defined as greater than 450 msec.
- Have a progressive central nervous system (CNS) disease, including degenerative CNS diseases and progressive tumors.
- Multiple drug allergies or a severe drug reaction to AEDs, including dermatological (eg, Stevens-Johnson syndrome), hematological, or organ toxicity reactions.
- Concomitant use of felbamate as an AED for less than 2 years or where the dose has not been stable for at least 8 weeks before Visit 1. They must not have a history of WBC count below equal or less than 2500/μL (2.50 1E+09/L), platelets below 100,000, liver function tests above 3 times the ULN, or other indication of hepatic or bone marrow dysfunction while receiving felbamate. If subjects received felbamate in the past, it must have been discontinued 8 weeks before Visit 1 to be eligible for study participation.
- Concomitant or recent (within last 5 months before Visit 1) use of vigabatrin or any evidence of vigabatrin-associated clinically significant vision abnormality.
- Benzodiazepines for any indications other than epilepsy (eg, anxiety/sleep disorders) prohibited from 1 month before Visit 1 and during the study. Benzodiazepines for seizure control and as rescue medication are allowed. (revised per Amendments 01 and 02)
- A vagal nerve stimulator (VNS), responsive neurostimulator (RNS), or deep brain stimulator (DBS) implanted less than 5 months before Visit 1 or changes in parameter less than 4 weeks before Visit 1 (or thereafter during the study).
- On a ketogenic diet for which the diet is not stable regimen for at least 4 weeks before Visit 1.
- Females of childbearing potential who: • Within 28 days before study entry, did not use a combination of a barrier method with a highly effective method of contraception, which includes any of the following: (revised per Amendment 01) o Total abstinence (if it is their preferred and usual lifestyle). o An intrauterine device or intrauterine hormone-releasing system (IUS). o A contraceptive implant. o An oral contraceptive (with additional barrier method). Subject must be on a stable dose of the same oral contraceptive product for at least 28 days before dosing and throughout the study and for 28 days after study drug discontinuation. o Have a vasectomized partner with confirmed azoospermia. Do not agree to use a combination of a barrier method with a highly effective method of contraception (as described above) throughout the entire study period and for 28 days after study drug discontinuation. (revised per Amendment 01). For sites outside of the EU, it is permissible that if a highly effective method of contraception is not appropriate or acceptable to the subject, then the subject must agree to use a medically acceptable method of contraception, ie, double-barrier methods of contraception such as latex or synthetic condom plus diaphragm or cervical/vault cap with spermicide. NOTE: All postpuberty females should be considered to be of childbearing potential unless they have been sterilized surgically (ie, bilateral tubal ligation, total hysterectomy, or bilateral oophorectomy, all with surgery at least 1 month before dosing).
- History of or a concomitant medical condition that in the opinion of the investigator would preclude the subject’s participation in a clinical study or compromise the subject’s ability to safely complete the study.
- Use of perampanel within 30 days before Visit 1, or perampanel was discontinued due to adverse reactions (perampanel-related) or lack of efficacy in case of previous exposure.
- Subjects with rare hereditary problems of galactose intolerance, the Lapp lactase deficiency or glucose-galactose malabsorption.
- Have participated in a study involving administration of an investigational drug or device within 4 weeks before Visit 1, or within approximately 5 half-lives of the previous investigational compound, whichever is longer.
- Hypersensitivity to the active substance or to any of the excipients of the study drug. (revised per Amendment 01)
- Weight less than 4.0 kg at Visit 1. (revised per Amendment 02).
- Current or history of pseudo-seizures (psychogenic nonepileptic seizures) within approximately 5 years before Visit 1.
- Have a history of status epilepticus that required hospitalization during the 6 months before to Visit 1.
- Have an unstable psychiatric diagnosis that may confound subjects’ ability to participate in the study or that may prevent completion of the protocol specified tests (eg, significant suicide risk, including suicidal behavior and ideation within 6 months before Visit 1, current psychotic disorder, acute mania).
- Any suicidal ideation with intent with or without a plan within 6 months before Visit 2 (ie, answering “Yes” to questions 4 or 5 on the Suicidal Ideation section of the Columbia-Suicide Severity Rating Scale (C-SSRS) in subjects aged 6 and above or based on the opinion of the Investigator for subject less than 6 years.
- Are scheduled or confirmed or both to have epilepsy surgery within 6 months after Visit 1; however, those who have previously documented “failed” epilepsy surgery will be allowed.
- Evidence of clinically significant disease (eg, cardiac, respiratory, gastrointestinal, renal disease) that in the opinion of the investigator could affect the subject’s safety or interfere with the study assessments.
- Evidence of moderate or severe renal insufficiency as defined by estimated glomerular filtration rates (eGFRs) based on Bedside Schwartz equation [eGFR=0.413×Height (cm)/Serum Creatinine (mg/dL)] of 31 to less than 60 mL/min/1.73m2 and less than 30 mL/min/1.73m2, respectively. (revised per Amendment 01)
The study team makes the final eligibility decision.
Where it's taking place
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.