ALSPhase 2NCT03019419
Perampanel for Sporadic Amyotrophic Lateral Sclerosis (ALS)
A Phase 2 study testing Perampanel in people with ALS, run by Tokyo Medical University at 12 sites in Japan.
Trial summary
To investigate the safety and the efficacy of perampanel in patients with sporadic amyotrophic lateral sclerosis Read more about this study
- Who is it for?
- Ages 40 to 78 years
- Conditions the study is for
- ALS
- What is being tested?
- Perampanel
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 66 participants
- Where
- 12 sitesJapan
- Study dates
- 24 Apr 2017to 23 Dec 2020
- Phase
- Phase 2
- 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 40 to 78 years
- All sexes
- No healthy volunteers
- Adult, Older adult
[Eligibility Criteria for Interim Registration]
- Patients who are able to submit written informed consent. If patients are duly capable of study consent but are unable to sign by themselves due to aggravation of disease condition, written informed consent can be obtained from a legally authorized representative who can sign on behalf of the patients after confirming the patients' agreement to study participation.
- Patients who are male or female aged 40 years to 78 years at the time of obtaining informed consent
- Patients who have clinically definite ALS, clinically probable ALS, or clinically probable-laboratory supported ALS as specified in the revised El Escorial Airlie House diagnostic criteria.
- The sum of the 3 respiratory items of the ALSFRS-R must total 12 points or more
- Patients within 2-year elapsed time period from disease onset at the time of obtaining informed consent
- Patients who can visit study site for out-patient treatment
[Eligibility Criteria for Registration]
Subjects who meet the following criteria in addition to the inclusion criteria for the interim registration
- The progression on score of ALSFRS-R during 12 weeks of observation period must be between -2 and -5
- Patients who has not initiated newly introduced riluzole therapy after starting the observation period. Or those who has not received dose escalation or resumed administration of riluzole therapy after previous down titration or discontinuation
- Patients who has not initiated newly introduced edaravone therapy after starting the observation period
- Patients who are judged to be eligible for continuation of the study by the investigators
[Exclusion Criteria]
- Patients who underwent tracheostomy.
- Patients who experienced non-invasive positive pressure ventilation.
- Patients whose percent-predicted forced vital capacity (%FVC) is ≤80%.
- Patients with progressive bulbar palsy type.
- Patients with cognitive impairment, severe disease in the renal, cardiovascular, or hematological system.
- Patients with hepatic disease.
- Patients with malignant tumor.
- Pregnant women or women with a possibility of becoming pregnant.
- Patients who participated in another clinical study within 12 weeks before starting the observation period.
- Patients who has initiated perampanel therapy in the past or at present.
- Patients who are judged to be ineligible for study entry by the investigators.
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
Japan12 sites
Kitasato University East Hospital
Kanagawa
Kumamoto Saishunso National Hospital
Kumamoto
Nagoya University Hospital
Nagoya
Okayama University Hospital
Okayama
Hokkaido University Hospital
Sapporo
Tohoku University Hospital
Sendai
Shiga University of Medical Science Hospital
Shiga
Tokyo Medical University
Tokyo, 160-0023
The University of Tokyo Hospital
Tokyo
Tokyo Metropolitan Neurological Hospital
Tokyo
University of Tsukuba Hospital
Tsukuba
Yamaguchi University Hospital
Yamaguchi
About this study
Official title: Perampanel for Sporadic Amyotrophic Lateral Sclerosis (ALS): A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Phase 2 Trials
To investigate the safety and the efficacy of perampanel in patients with sporadic amyotrophic lateral sclerosis
Read the detailed description
To evaluate the effect of peramanel for 48 weeks on progression of disease in subjects with ALS, as measured by ALS Functional Rating Scale-Revised (ALSFRS-R)
- Conditions
- ALS
- Being tested
- Perampanel, placebo
- Keywords
- sporadic ALS, perampanel
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PerampanelDrug
Also called non-competitive AMAP antagonist
4mg/d or 8mg/d
placeboDrug
placebo
| Study group | Role | What they receive |
|---|---|---|
| Perampanel 4mg | Experimental | Once daily 4mg of perampanel with dose-escalation tolerable from 2mg to 4mg for 48 weeks Receives: Drug: Perampanel |
| Perampanel 8mg | Experimental | Once daily 8mg of perampanel with dose-escalation tolerable from 2mg to 8mg for 48 weeks Receives: Drug: Perampanel |
| Placebo | Placebo comparator | Once daily placebo for control for 48 weeks Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in ALS Functional rating scale
Measured: 48 weeks
Secondary outcomes (3)
Change in ALS Functional rating scale
Measured: 12, 24, 36, and 48 weeks
Manual Muscle Test
Measured: 12, 24, 36, and 48 weeks
Percent-predicted forced vital capacity
Measured: 12, 24, 36, and 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jan 2017 (estimated)
- Study start24 Apr 2017
- Primary completion23 Dec 2020
- Study completion23 Dec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ALSStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 66 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tokyo Medical University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Tomohiro Haga, Study director, The University of Tokyo Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03019419
- Sponsor's protocol ID
- A2016-J000-001
- Status last verified
- Jan 2021
- First submitted
- 10 Jan 2017
- Last updated
- 15 Jan 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Perampanel for Sporadic Amyotrophic Lateral Sclerosis (ALS): A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Phase 2 Trials
- ClinicalTrials.gov ID
- NCT03019419
- Acronym / sponsor ID
- A2016-J000-001
- Conditions
- ALS
- Keywords
- sporadic ALS, perampanel
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 66 participants (actual)
- Lead sponsor
- Tokyo Medical UniversityUniversity, hospital or other
- Study start
- 24 Apr 2017 (actual)
- Primary completion
- 23 Dec 2020 (actual)
- Study completion
- 23 Dec 2020 (actual)
- First posted
- 12 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.
To investigate the safety and the efficacy of perampanel in patients with sporadic amyotrophic lateral sclerosis
Detailed description
To evaluate the effect of peramanel for 48 weeks on progression of disease in subjects with ALS, as measured by ALS Functional Rating Scale-Revised (ALSFRS-R)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Perampanel 4mg | Experimental | Drug: PerampanelOnce daily 4mg of perampanel with dose-escalation tolerable from 2mg to 4mg for 48 weeks |
| Perampanel 8mg | Experimental | Drug: PerampanelOnce daily 8mg of perampanel with dose-escalation tolerable from 2mg to 8mg for 48 weeks |
| Placebo | Placebo comparator | Drug: placeboOnce daily placebo for control for 48 weeks |
Who may qualify
- Ages
- 40 to 78 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
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: non-competitive AMAP antagonist4mg/d or 8mg/d | Drug | Perampanel 4mg, Perampanel 8mg |
| placeboplacebo | Drug | Placebo |
Primary outcome
Change in ALS Functional rating scale
Time frame: 48 weeks
3 secondary outcomes
Change in ALS Functional rating scale
Time frame: 12, 24, 36, and 48 weeks
Manual Muscle Test
Time frame: 12, 24, 36, and 48 weeks
Percent-predicted forced vital capacity
Time frame: 12, 24, 36, and 48 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jan 2021.
- 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.
Tomohiro Haga study director
The University of Tokyo Hospital
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Perampanel in people with ALS, run by Tokyo Medical University at 12 sites in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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: finished, as of the sponsor's last update on 15 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in Japan. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Change in ALS Functional rating scale (time frame: 48 weeks). 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 15 Jan 2021.