Perampanel for Pediatric Epilepsy

Recruiting in Dayton (<99 mi)

+17 other 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

Trial Summary

What is the purpose of this trial?

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 core study for seizure frequency in participants with pediatric epileptic syndrome (Cohort 1) and partial-onset seizures (POS) (Cohort 2).

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.

What data supports the effectiveness of the drug Perampanel for pediatric epilepsy?

Research shows that Perampanel is effective in improving seizure control in children and adolescents with difficult-to-treat epilepsy, and it is generally well-tolerated. It has been used successfully as an additional treatment for epilepsy in patients aged 12 and older, helping to reduce seizures. ^{1 2 3 4 5}

Is Perampanel safe for children with epilepsy?

Perampanel (also known as Fycompa) has been studied for safety in children and adolescents with epilepsy. It is generally considered safe, but like any medication, it can have side effects. Some studies have shown it to be safe in children as young as 7 years old, but more research is needed for younger children. ^{4 5 6 7 8}

How is the drug Perampanel different from other treatments for pediatric epilepsy?

Perampanel is unique because it works by blocking AMPA receptors, which are involved in transmitting signals in the brain that can lead to seizures. Unlike some other epilepsy drugs, it is specifically used as an add-on treatment for partial-onset seizures in patients aged 12 and older, and it does not significantly interact with the liver enzyme system that processes many other medications. ^{1 4 5 7 9}

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

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 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 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]

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 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 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]

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]

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]

References

1. Netherlands pubmed.ncbi.nlm.nih.gov

Effectiveness and tolerability of perampanel in children and adolescents with refractory epilepsies-An Italian observational multicenter study. [2022]

2. Germany pubmed.ncbi.nlm.nih.gov

Effectiveness and tolerability of perampanel in children and adolescents with refractory epilepsies: first experiences. [2022]

3. Korea (South) 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. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

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

5. England pubmed.ncbi.nlm.nih.gov

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

6. England pubmed.ncbi.nlm.nih.gov

The efficacy of perampanel in young children with drug-resistant epilepsy. [2021]

7. Netherlands pubmed.ncbi.nlm.nih.gov

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

8. United States 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. [2022]

9. France pubmed.ncbi.nlm.nih.gov

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