Amyotrophic Lateral SclerosisNCT03020797
A Clinical Trial to Evaluate the Safety and Efficacy of Fycompa in Subjects With Amyotrophic Lateral Sclerosis (ALS)
A study testing Perampanel in people with Amyotrophic Lateral Sclerosis, run by Stony Brook University at 1 site in United States.
Trial summary
This is a pilot trial to test perampanel (Fycompa; Eisai, Inc.) in ALS patients. The investigators will focus on safety and preliminary signs of efficacy. Perampanel is approved by the FDA for treatment of seizures in patients with epilepsy. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Perampanel
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 12 Jan 2017to 8 Feb 2023
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- use of tracheostomy or mechanical ventilation within last 3 months
- hepatic insufficiency or abnormal liver function
- renal insufficiency
- clinically significant psychiatric disorder
- active malignancy
- history of HIV, clinically significant chronic hepatitis, or other active infection
Show all 13Show fewer
- history of stomach or intestinal surgery or condition that could interfere with absorption, distribution, metabolism or secretion of study drug
- history of alcohol or substance abuse within 3 months prior to entry (subjects will be instructed to refrain from alcohol during the study)
- use of strong cytochrome P4503A inhibitors or inducers, anticonvulsants or other drugs known to interact strongly with perampanel.
- pregnancy or lactation
- clinically significant medical condition (other than ALS) that would pose a risk to the subject if they were to participate
- know hypersensitivity to perampanel
- currently participating, or has participated in a study with an investigation or marketed compound within 3 months of entry
Trial locations and contacts
1 site in 1 country.
United States1 site
Stony Brook University Medical Center
Stony Brook, New York, 11794-8121
About this study
This is a pilot trial to test perampanel (Fycompa; Eisai, Inc.) in ALS patients. The investigators will focus on safety and preliminary signs of efficacy. Perampanel is approved by the FDA for treatment of seizures in patients with epilepsy. In this study, perampanel will be used off-label for adults with ALS at an oral medication dose on the low end of the recommended dose range for epilepsy. This study will consist of two treatments arms: perampanel and matching placebo randomized at a 1:1 ratio. Subjects will receive medication for 9 months.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Perampanel, Placebo Oral Tablet
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PerampanelDrug
Also called Fycompa
Placebo Oral TabletDrug
| Study group | Role | What they receive |
|---|---|---|
| perampanel | Experimental | perampanel 2mg QD for 2 weeks perampanel 4mg QD for 2 weeks perampanel 6mg QD for 2 weeks perampanel 8mg QD for 30 weeks Receives: Drug: Perampanel |
| placebo | Placebo comparator | 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 Receives: Drug: Placebo Oral Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events
Measured: 9 months
Secondary outcomes (1)
Efficacy as measured by change in ALSFRS-R score (ALS functional rating scale-revised);
Measured: 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2017 (estimated)
- Study start12 Jan 2017
- Primary completion31 Jan 2023
- Study completion8 Feb 2023
Why it stopped: 1\) Enrolment below study target and 2) too many subjects had early termination due to disease progression.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stony Brook University
- Sponsor type
- University, hospital or other
- Collaborators
- Eisai Inc.
- Responsible party
- Principal investigator, Nurcan Gursoy, Stony Brook University
- Investigators
- Nurcan Gursoy, MD, Principal investigator, Stony Brook Medicine Dept. of Neurology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03020797
- Sponsor's protocol ID
- Eisai-01
- Status last verified
- May 2023
- First submitted
- 15 Dec 2016
- Last updated
- 6 May 2023
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Clinical Trial to Evaluate the Safety and Efficacy of Fycompa in Subjects With Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT03020797
- Acronym / sponsor ID
- Eisai-01
- Conditions
- Amyotrophic Lateral Sclerosis
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: 1\) Enrolment below study target and 2) too many subjects had early termination due to disease progression.
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Stony Brook UniversityUniversity, hospital or other
- Collaborators
- Eisai Inc.
- Study start
- 12 Jan 2017 (actual)
- Primary completion
- 31 Jan 2023 (actual)
- Study completion
- 8 Feb 2023 (actual)
- First posted
- 13 Jan 2017 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a pilot trial to test perampanel (Fycompa; Eisai, Inc.) in ALS patients. The investigators will focus on safety and preliminary signs of efficacy. Perampanel is approved by the FDA for treatment of seizures in patients with epilepsy. In this study, perampanel will be used off-label for adults with ALS at an oral medication dose on the low end of the recommended dose range for epilepsy. This study will consist of two treatments arms: perampanel and matching placebo randomized at a 1:1 ratio. Subjects will receive medication for 9 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| perampanel | Experimental | Drug: Perampanelperampanel 2mg QD for 2 weeks perampanel 4mg QD for 2 weeks perampanel 6mg QD for 2 weeks perampanel 8mg QD for 30 weeks |
| placebo | Placebo comparator | Drug: Placebo Oral Tabletplacebo 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 |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (13 listed)
- use of tracheostomy or mechanical ventilation within last 3 months
- hepatic insufficiency or abnormal liver function
- renal insufficiency
- clinically significant psychiatric disorder
- active malignancy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PerampanelAlso: Fycompa | Drug | perampanel |
| Placebo Oral Tablet | Drug | placebo |
Primary outcome
Incidence of treatment-emergent adverse events
Time frame: 9 months
1 secondary outcome
Efficacy as measured by change in ALSFRS-R score (ALS functional rating scale-revised);
Time frame: 9 months
Results and safety
The study was stopped early (reason given: 1\) Enrolment below study target and 2) too many subjects had early termination due to disease progression.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 May 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Nurcan Gursoy, MD principal investigator
Stony Brook Medicine Dept. of Neurology
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Perampanel in people with Amyotrophic Lateral Sclerosis, run by Stony Brook University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 6 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: 1\) Enrolment below study target and 2) too many subjects had early termination due to disease progression.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events (time frame: 9 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 May 2023.