Perampanel for Amyotrophic Lateral Sclerosis

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Stony Brook University Medical Center, Stony Brook, NY
Amyotrophic Lateral Sclerosis+2 More
Perampanel - Drug
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether perampanel may have positive impacts for individuals with amyotrophic lateral sclerosis.

See full description

Eligible Conditions

  • Amyotrophic Lateral Sclerosis

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Compared to trials

Study Objectives

This trial is evaluating whether Perampanel will improve 1 primary outcome and 1 secondary outcome in patients with Amyotrophic Lateral Sclerosis. Measurement will happen over the course of 9 months.

9 months
Efficacy as measured by change in ALSFRS-R score (ALS functional rating scale-revised);
Incidence of treatment-emergent adverse events

Trial Safety

Safety Estimate

1 of 3

Compared to trials

Side Effects for

Perampanel 12 mg
Dizziness
28%
Fatigue
17%
Somnolence
15%
Vomiting
11%
Irritability
9%
Nasopharyngitis
9%
Headache
9%
Nausea
7%
Ear infection
7%
Depression
6%
Balance disorder
6%
Upper respiratory tract infection
6%
Memory impairment
6%
Oropharyngeal pain
6%
Aggression
6%
Sudden unexplained death in epilepsy
2%
Transient ischaemic attack
2%
Mental status changes
2%
Suicidal ideation
2%
This histogram enumerates side effects from a completed 2021 Phase 4 trial (NCT03288129) in the Perampanel 12 mg ARM group. Side effects include: Dizziness with 28%, Fatigue with 17%, Somnolence with 15%, Vomiting with 11%, Irritability with 9%.

Trial Design

2 Treatment Groups

perampanel
1 of 2
placebo
1 of 2
Experimental Treatment
Non-Treatment Group

This trial requires 60 total participants across 2 different treatment groups

This trial involves 2 different treatments. Perampanel is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are not being studied for commercial purposes.

perampanel
Drug
perampanel 2mg QD for 2 weeks perampanel 4mg QD for 2 weeks perampanel 6mg QD for 2 weeks perampanel 8mg QD for 30 weeks
placebo
Drug
placebo 1 tablet QD for 2 weeks placebo 2 tablets QD for 2 weeks placebo 3 tablets QD for 2 weeks placebo 4 tablets QD for 2 weeks
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Perampanel
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 9 months
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 9 months for reporting.

Who is running the study

Principal Investigator
N. G.
Prof. Nurcan Gursoy, Clinical Assistant Professor
Stony Brook University

Closest Location

Stony Brook University Medical Center - Stony Brook, NY

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 7 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
diagnosis of ALS
first clinical weakness within past 3 years
slow vital capacity >= 60% of predicted within 1 month of treatment
may be on stable dose of riluzole for at least 30 days, or otherwise agree to not initiate riluzole for duration of the trial
may be on stable dose of edaravone for at least 30 days, otherwise agree to not initiate edaravone for duration of the trial
can travel to Stony Brook to receive medical care
must have a monitor who can be contacted at regular intervals to report on subject's clinical/psychiatric status

Patient Q&A Section

How many people get amyotrophic lateral sclerosis a year in the United States?

"The American Cancer Society estimates approximately 20,000 new cases of non-Hodgkin's lymphoma will be diagnosed in 2019 and 5,000 deaths will be attributed to the disease. In 2018, about 36,000 Americans will get the disease. By 2050, about 10 million people will have the disease. The disease is the most common cause of death among men and women under 50 years of age." - Anonymous Online Contributor

Unverified Answer

What are common treatments for amyotrophic lateral sclerosis?

"Several options to treat ALS are available, depending on the progress and severity of the disease and patient preferences. One such option is riluzole, a drugs under development that is used to halt and reverse nerve cell death. It is not approved by US health care providers as a treatment for ALS. A more common treatment for ALS is a multidisciplinary approach that aims to rehabilitate, maintain quality of life, and minimize symptoms. The current US Food and Drug Administration-approved ALS clinical trials provide this type of approach. Because ALS is a genetic disorder, many patients with familial ALS develop certain symptoms more rapidly." - Anonymous Online Contributor

Unverified Answer

What is amyotrophic lateral sclerosis?

"ALS was an extremely heterogeneous condition presenting, on average, with mild weakness of proximal muscles in the early stages of the disease. The disease was slowly progressive in the vast majority of cases with the progression being rapid in a minority of cases. The pattern of clinical disease progression was, on average, that of a slowly progressive disease similar to, and possibly even more slowly progressive than, amyotrophic lateral sclerosis. No clinically relevant difference from the average natural course of ALS was observed in any of the markers or scores studied; however, biomarkers may serve as potential biomarkers of disease progression. The pattern and distribution of age of onset indicated the same picture of prognosis as the pattern of progression in ALS. [Power](http://www." - Anonymous Online Contributor

Unverified Answer

What are the signs of amyotrophic lateral sclerosis?

"Muscle wasting, weakness, increased resistance, and dysphagia in amyotrophic lateral sclerosis are key signs of this disease. The respiratory system is often involved (bronchiectasis), as well as the gastrointestinal system (gastric outlet- obstruction or fistula). The central nervous system can also experience the neurological involvement and psychiatric disorders. The liver and kidneys are not always involved." - Anonymous Online Contributor

Unverified Answer

What causes amyotrophic lateral sclerosis?

"Many different genetic and environmental factors can cause ALS. Symptoms of the disorder include muscle weakness, numbness and changes in sense of touch. It is unclear why ALS affects only certain nerve cells in the motor system. Because ALS can occur sporadically and seems to be associated with an abnormal buildup of protein clumps called neurofibrillary tangles in the brain, experts suspect that the risk of ALS may be increased by environmental factors such as pollution or viruses.\nquestion: Neurological and cardiac signs of diabetes mellitus: is there an association with the duration of diabetes? answer: There are no specific clinical signs suggestive of carotid artery disease that can be identified in patients with a history of diabetes." - Anonymous Online Contributor

Unverified Answer

Can amyotrophic lateral sclerosis be cured?

"ALS was associated with a significantly higher rate of early death after diagnosis in comparison with the general population, independent of gender. The cause of the excess of early death needs to be elucidated." - Anonymous Online Contributor

Unverified Answer

What is the latest research for amyotrophic lateral sclerosis?

"This article contains the latest research for amyotrophic lateral sclerosis on the subject of drug use as the disease advances. It is important to know that the disease will advance and the research presented here will not always help." - Anonymous Online Contributor

Unverified Answer

Does amyotrophic lateral sclerosis run in families?

"There was no evidence that first-degree family members of patients with ALS demonstrate increased incidence of disease or slower rates of disability, compared with the general population." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets amyotrophic lateral sclerosis?

"In 2013, there were 14,345 new diagnoses of ALS in the United States and about 1 in 4 would die from ALS by age 85. Although there are more women diagnosed with ALS, the number of men who die from the disease seems to be higher. There are also many variations among the different age groups. In fact, there is a higher chance of men dying from ALS, especially in the 55-74 age group, rather than women. If a person does have ALS, it must be very early in life, since the rate of ALS decreases with age. As of 2015, there are no treatments that have halted the degradation of the motor muscles." - Anonymous Online Contributor

Unverified Answer

Is perampanel typically used in combination with any other treatments?

"Perampanel was used frequently in combination with either an ABA or a second medication. There is no published evidence suggesting a clinically important interaction, and the FDA approves perampanel for treating partial-onset and tonic-clonic seizures in adults with either a single or a combination medications as a single therapeutic agent. The combination of perampanel with an ABA is contraindicated, as there is no evidence to support such an additive effect." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of amyotrophic lateral sclerosis?

"Muscle weakness, muscle wasting and the degeneration of motor units are the main causes of this disease. However, the mechanism of its progression is debated." - Anonymous Online Contributor

Unverified Answer

Does perampanel improve quality of life for those with amyotrophic lateral sclerosis?

"In both patients with and without depressive symptoms, perampanel had positive and sustained antipsychotic properties in the short term that normalized psychotic symptoms and significantly improved quality of life. Future studies should prospectively characterize the long-term effects of perampanel." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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