Evidence map›Paper›PMID 40474686›Full record

Trial reportMuscle & nerve2025

Efficacy and Safety of Once Daily Dosing vs. Approved On/Off Dosing of Edaravone Oral Suspension Up to 48 Weeks in Patients With Amyotrophic Lateral Sclerosis (Study MT-1186-A02).

Jeffrey Rothstein, Angela Genge, Shari De Silva, Lorne Zinman, Marvin Chum, Adriano Chio, Gen Sobue, Masashi Aoki, Hiide Yoshino, Manabu Doyu and 8 more

Registry-linked trialAbstract readMulticenter StudyRandomized Controlled TrialClinical Trial, Phase III
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Muscle & nerve, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04569084 (A Phase 3b, Multicenter, Randomized, Double-Blind Study to Evaluate Efficacy and Safety of Oral Edaravone Administered for a Period of 48 Weeks in Subjects With Amyotrophic Lateral Sclerosis), which is not on this map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT04569084 phase3terminatednot on this map

A Phase 3b, Multicenter, Randomized, Double-Blind Study to Evaluate Efficacy and Safety of Oral Edaravone Administered for a Period of 48 Weeks in Subjects With Amyotrophic Lateral Sclerosis (ALS)

TypeinterventionalSponsorShionogiRan2020 to 2023Enrolled384ConditionsALSArmsMT-1186, Placebo
3 · Its place in the literature

Who cites it

7 citing papers in PubMed.

  1. Trial
  2. Trial
  3. Review
  4. Review
  5. Review
  6. Review
  7. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

18 authors.

Jeffrey RothsteinDepartment of Neurology, School of Medicine, Johns Hopkins University, Baltimore, Maryland, USA.
Angela GengeMontreal Neurological Institute and Hospital, Montreal, Canada.
Shari De SilvaWoodland Research Northwest, Rogers, Arkansas, USA.
Lorne ZinmanSunnybrook Health Sciences Centre, University of Toronto, Toronto, Ontario, Canada.
Marvin ChumMcMaster University Health Sciences Centre, Hamilton, Ontario, Canada.
Adriano ChioUniversità Degli Studi di Torino, Centro Regionale Esperto per la Sclerosi Laterale Amiotrofica (CRESLA), Turino, Italy.
Gen SobueNagoya University Graduate School of Medicine, Nagoya, Aichi, Japan.
Masashi AokiTohoku University School of Medicine, Sendai, Miyagi, Japan.
Hiide YoshinoNeurology Clinic, Chiba, Japan.
Manabu DoyuDepartment of Neurology, Aichi Medical University, Nagakute, Aichi, Japan.
Daniel SelnessMitsubishi Tanabe Pharma America, Inc., Jersey City, New Jersey, USA.
Vesna TodorovicMitsubishi Tanabe Pharma Europe, Ltd., London, UK.
Nissim SassonNStat Solutions, Biostatistical Services, Rehovot, Israel.
Manabu HiraiMitsubishi Tanabe Pharma America, Inc., Jersey City, New Jersey, USA.
Fumihiro TakahashiMitsubishi Tanabe Pharma America, Inc., Jersey City, New Jersey, USA.ORCID https://orcid.org/0000-0001-5701-5332
Alejandro SalahMitsubishi Tanabe Pharma America, Inc., Jersey City, New Jersey, USA.
Art WamilMitsubishi Tanabe Pharma America, Inc., Jersey City, New Jersey, USA.
Stephen AppleMitsubishi Tanabe Pharma America, Inc., Jersey City, New Jersey, USA.ORCID https://orcid.org/0000-0002-3051-3953

Funding

Mitsubishi Tanabe Pharma America Inc.
6 · The paper itself

Abstract

INTRODUCTION/

aimsAn On/Off dosing regimen of intravenous (IV) edaravone and edaravone oral suspension is approved in the US for the treatment of amyotrophic lateral sclerosis (ALS). Placebo-controlled clinical trials showed IV edaravone slows the rate of physical functional decline. This study evaluated whether investigational daily dosing displayed superior efficacy vs. approved on/off dosing of edaravone oral suspension, and assessed safety and tolerability, over 48 weeks in patients with ALS.

methodsStudy MT-1186-A02 (NCT04569084) was a multicenter, phase 3b, double-blind, parallel group, superiority study that randomized patients to edaravone oral suspension (105-mg dose) administered Once Daily or the same edaravone oral suspension dose administered according to the approved On/Off regimen including placebo to mimic daily drug dosing. Patients had definite or probable ALS, baseline forced vital capacity ≥ 70%, and baseline disease duration ≤ 2 years. The primary endpoint was a combined assessment of function and survival (CAFS) at week 48, which included change in ALS Functional Rating Scale-Revised (ALSFRS-R) and time to death.

resultsCAFS at week 48 indicated Once Daily dosing did not show a statistically significant difference vs. approved on/off dosing (p = 0.777). Both dosing regimens provided comparable change from baseline ALSFRS-R total score to week 48 (least squares mean difference: 0.27 [95% CI -1.43 to 1.97]). Edaravone oral suspension was well tolerated, and no new safety concerns were identified in either group. DISCUSSION: Daily edaravone oral suspension did not show superiority and had equivalent safety and tolerability vs. the approved On/Off regimen, reinforcing the appropriateness of the approved dosing regimen.

Indexed as

Amyotrophic Lateral SclerosisEdaravoneFree Radical ScavengersAdministration, OralAdultAgedDouble-Blind MethodDrug Administration ScheduleFemaleHumansMaleMiddle AgedSuspensionsTreatment OutcomeEdaravoneFree Radical ScavengersSuspensionsamyotrophic lateral sclerosisclinical trialedaravoneefficacysafety

Identifiers

PMID40474686
PMCPMC12338024

What OpenQuestion holds

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.