Perampanel for Pediatric Epilepsy

Recruiting at 22 trial locations

Overseen By Eisai Medical Information

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Eisai Inc.

Must be taking: Antiepileptic drugs

Must not be taking: Benzodiazepines, Perampanel

Disqualifiers: Pseudo-seizures, Status epilepticus, Unstable psychiatric, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 23 weeks

100 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests perampanel (Fycompa) to evaluate its effectiveness in reducing seizures in children with epilepsy. The focus is on two groups: those with pediatric epileptic syndrome and those with partial-onset seizures. The trial seeks children who have experienced at least four seizures in the past month and are on a stable regimen of up to four other seizure medications. Children meeting these criteria might be suitable for this trial. As a Phase 2 trial, the research measures the treatment's effectiveness in an initial, smaller group, offering a chance to contribute to important findings in epilepsy care.

Do I have to stop taking my current medications for the trial?

The trial does not require you to stop your current medications. You must be on stable doses of 1 to 4 approved antiepileptic drugs (AEDs) for at least 4 weeks before starting the trial.

Is there any evidence suggesting that perampanel is likely to be safe for humans?

Previous studies have shown that perampanel is generally safe for treating epilepsy in children. For example, one study found that 70.4% of children experienced fewer seizures after taking perampanel for six months. The treatment was well-tolerated, with most children not experiencing serious side effects. Another review supports these findings, noting that perampanel is a safe choice for those with partial-onset seizures.

While some side effects have been reported, they are mostly mild and similar to those seen in other epilepsy treatments, such as dizziness or drowsiness. Since perampanel is already approved for use in older children and adults with epilepsy, extensive safety information is available. This evidence suggests that the treatment is reasonably safe for younger patients as well. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for epilepsy?

Perampanel is unique because it offers a new approach to treating pediatric epilepsy, particularly for children as young as one month old. Unlike many existing epilepsy treatments that primarily target sodium or calcium channels, perampanel works differently by selectively blocking AMPA receptors, which are involved in the spread of seizure activity. This distinct mechanism of action provides a novel option that could potentially be more effective for certain young patients. Additionally, perampanel is available in both oral suspension and tablet forms, making it adaptable for different age groups and easier to administer to young children. Researchers are excited about its potential to address unmet needs in pediatric epilepsy management.

What evidence suggests that perampanel might be an effective treatment for pediatric epilepsy?

Research has shown that perampanel, the treatment under study in this trial, can help reduce seizures in children with epilepsy. In one study, 70.4% of patients experienced at least a 50% reduction in seizure frequency after six months of treatment. Another study found that perampanel was effective and well-tolerated by children with hard-to-treat epilepsy. These findings suggest that perampanel may help children with various types of epilepsy experience fewer seizures. ^{3 4 6 7 8}

Are You a Good Fit for This Trial?

This trial is for children with epilepsy: Cohort 1 includes those under 18 years with pediatric epileptic syndrome, and Cohort 2 includes infants up to 2 years old with partial-onset seizures. Participants must have had at least four seizures in the last month and be on stable doses of up to four antiepileptic drugs.

Inclusion Criteria

My epilepsy is not getting worse according to tests or doctor's evaluation.

I am under 18 years old and was born after at least 36 weeks of pregnancy.

I am on 1-4 epilepsy drugs, including possibly medical marijuana, with stable doses for the last 4 weeks.

Exclusion Criteria

I haven't taken perampanel in the last 30 days or stopped it due to side effects or it not working.

If you have had a VNS, RNS, or DBS implanted less than 5 months ago, or if there have been changes to the settings of these devices within the last 4 weeks.

I haven't taken benzodiazepines for reasons other than epilepsy in the last month.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Treatment

Participants receive perampanel during the Core Study, including a 10-week titration period and a 13-week maintenance period

23 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Extension Phase A

Participants continue treatment with perampanel for an additional 33 weeks, followed by a 4-week follow-up period

37 weeks

Extension Phase B

Participants in countries without commercial availability of perampanel or an EAP may continue treatment if deemed beneficial by the investigator

What Are the Treatments Tested in This Trial?

Interventions

• Perampanel

Trial Overview

The study tests Perampanel's effectiveness as an additional treatment for childhood epilepsy. It measures how many participants experience a significant reduction in seizure frequency during the maintenance phase using either oral suspension or tablet forms.

How Is the Trial Designed?

Group I: Perampanel Experimental Treatment 2 Interventions

Participants aged 1 month to less than 18 years with pediatric epileptic syndrome (Cohort 1) or aged 1 month to less than 2 years with POS (Cohort 2) will receive perampanel oral suspension or perampanel tablets, once daily up to 56 weeks.

Perampanel is already approved in European Union, United States for the following indications:

Approved in European Union as Fycompa for:

• Partial-onset epilepsy (with or without secondary generalized seizures)
• Primary generalized tonic-clonic seizures

Approved in United States as Fycompa for:

• Partial-onset seizures (with or without secondary generalized seizures)
• Primary generalized tonic-clonic seizures
• Monotherapy and adjunctive therapy for focal epilepsy (with or without secondary generalized seizures) in patients aged 4 years and older

Who Is Running the Clinical Trial?

Eisai Inc.

Lead Sponsor

Trials

524

Recruited

161,000+

Founded

Eisai Inc. was established in 1995 as the U.S. subsidiary of Eisai Co., Ltd.

Headquarters

Woodcliff Lake, NJ, USA

Known For

Neurology and Oncology

Top Products

Aricept (donepezil), Lenvima (lenvatinib), Leqembi (lecanemab), Halaven (eribulin)

Lynn Kramer

Eisai Inc.

Chief Medical Officer since 2019

MD

Tatsuyuki Yasuno

Eisai Inc.

Chief Executive Officer since 2023

MBA from Kellogg School of Management, Northwestern University; Bachelor of Political Science from Waseda University

Published Research Related to This Trial

In a systematic review of 21 studies involving 1968 children and adolescents, perampanel (PER) was found to reduce seizure frequency by at least 50% in 51.5% of patients, with complete seizure cessation in 20.6%, indicating its efficacy as an antiepileptic drug.

The safety profile of PER showed that 40.8% of patients experienced adverse events, with the most common being drowsiness (15.3%) and irritability (9.3%), but it was generally well tolerated, with only 9.2% discontinuing due to these adverse effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy, tolerability and safety of perampanel in children and adolescents with epilepsy: Systematic review and meta-analysis. Sun, S., Li, X., Liu, X.[2023]

Perampanel, an AMPA glutamate receptor antagonist approved for partial epilepsy, showed only modest efficacy in clinical trials, with a response rate about 20% higher than placebo after 19 weeks, and no evidence of being superior to other antiepileptic drugs.

The drug is associated with frequent adverse effects such as irritability, aggression, and weight gain, along with potential long-term risks like cardiac disorders and stunted growth, raising concerns about its overall safety and therapeutic value.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Perampanel. Just another anticonvulsant for partial epilepsy: no progress. [2021]

In a study of 85 patients with focal-onset seizures, low-dose perampanel (4-6 mg/day) significantly improved seizure control compared to high doses (8-12 mg/day), with a 50% responder rate of 88.6% versus 40.0%.

Perampanel was also more effective when added to first-line anti-seizure medications, achieving a 50% responder rate of 83.5% compared to 33.3% for second-line medications, and was well tolerated with dizziness being the most common side effect.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Perampanel as First Add-On Therapy in Patients with Focal-Onset Seizures in the FAME Trial: Post hoc Analyses of Efficacy and Safety Related to Maintenance Dose and Background Antiepileptic Drug Therapy. Kim, JH., Kim, DW., Lee, SK., et al.[2022]

Perampanel is a newly approved antiepileptic drug in the EU, specifically indicated for use as an additional treatment for partial-onset seizures in patients aged 12 and older.

It is designed to be used alongside other medications, highlighting its role as an adjunctive therapy in managing epilepsy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Perampanel: a new add-on treatment for epilepsy. [2021]

In a 52-week observational study involving 483 patients with refractory epilepsy, perampanel was found to have a safety profile consistent with previous clinical studies, with 62.3% of patients reporting at least one treatment-emergent adverse event (TEAE), primarily mild to moderate in severity.

The most common clinically important TEAEs included dizziness (13.9%) and balance disorders (5.6%), with 31.7% of patients experiencing such events, indicating that while perampanel is effective, monitoring for these side effects is essential in real-world settings.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A post-approval observational study to evaluate the safety and tolerability of perampanel as an add-on therapy in adolescent, adult, and elderly patients with epilepsy. Maguire, M., Ben-Menachem, E., Patten, A., et al.[2022]

In a study of 58 pediatric patients with refractory epilepsies, perampanel (PER) demonstrated a 31% response rate, with 9% achieving complete seizure control after 3 months of treatment.

The treatment was generally well-tolerated, although some patients experienced adverse effects such as reduced vigilance or fatigue (16 cases) and behavioral changes (14 cases).

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effectiveness and tolerability of perampanel in children and adolescents with refractory epilepsies: first experiences. Biró, A., Stephani, U., Tarallo, T., et al.[2022]

In a study of 38 children under 7 years old with refractory epilepsy, perampanel showed a 44% response rate for seizure reduction after 6 months, indicating its efficacy in this young population.

The treatment was generally well-tolerated, with only 22% of patients experiencing adverse effects, suggesting that perampanel can be a safe option for young children, particularly those with conditions like tuberous sclerosis and Dravet syndrome.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The efficacy of perampanel in young children with drug-resistant epilepsy. Chang, FM., Fan, PC., Weng, WC., et al.[2021]

In a study of 62 children and adolescents with refractory epilepsy, Perampanel (PER) demonstrated a 50% response rate in reducing seizure frequency, indicating its efficacy as an add-on therapy.

The treatment was generally well-tolerated, with a retention rate of 77.4% over an average follow-up of 6.6 months, although 30.6% of patients experienced adverse events, including behavioral disturbances and dizziness.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effectiveness and tolerability of perampanel in children and adolescents with refractory epilepsies-An Italian observational multicenter study. De Liso, P., Vigevano, F., Specchio, N., et al.[2022]

Perampanel has been shown to be effective in reducing seizure frequency in patients with epilepsy, demonstrating significant clinical efficacy in multiple studies.

The safety profile of perampanel indicates that while it is generally well-tolerated, it can cause side effects such as dizziness and fatigue, which should be monitored in patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Perampanel (Fycompa): A Review of Clinical Efficacy and Safety in Epilepsy. Greenwood, J., Valdes, J.[2020]

Citations

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sciencedirect.com

sciencedirect.com/science/article/abs/pii/S0920121125001913

Effectiveness and Tolerability of Perampanel as First Add ...

Effectiveness was consistently higher throughout visits in patients receiving perampanel as first vs. late add-on treatment, with higher ...

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pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC12175630/

Effectiveness and safety of perampanel for pediatric ...

Effectiveness outcomes. After six months of PER treatment in routine clinical care, the 50% responder rate and seizure‐free rates were 70.4% (169/240) ...

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clinicaltrials.gov

clinicaltrials.gov/study/NCT04015141

NCT04015141 | A Study to Evaluate Efficacy and Safety of ...

The purpose of the study is to evaluate the efficacy of perampanel as measured by the 50 percent (%) responder rate during the maintenance period of the ...

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pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC9666151/

Effectiveness and tolerability of adjunctive perampanel in ...

Overall, perampanel exhibited good effectiveness, safety, and tolerability in the treatment of pediatric patients (aged 4–12 years) with refractory epilepsy.

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nature.com

nature.com/articles/s41598-023-40594-8

Long-term efficacy and safety of adjunctive perampanel in ...

Seizure reduction rates of 100% and 50% responder rates were comparable to those observed in previous short-term studies. Remarkably, fewer ...

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fda.gov

fda.gov/media/130828/download

Pediatric Postmarketing Pharmacovigilance Review

EXECUTIVE SUMMARY. This review evaluates FDA Adverse Event Reporting System (FAERS) reports for Fycompa. (perampanel) in pediatric patients ...

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pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC5083075/

A Review of Clinical Efficacy and Safety in Epilepsy - PMC

Perampanel is a safe and effective adjunctive treatment option for patients with POS and PGTC seizures. Additional evidence suggests perampanel may also be ...

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mednexus.org

mednexus.org/doi/10.1002/ped4.12463

Effectiveness and safety of perampanel for pediatric ...

After a 6-month treatment regimen with PER, 70.4% of the patients experienced a reduced seizure frequency of at least 50%, and 22.1% achieved ...