Efficacy and Safety Study of Oral Edaravone Administered in Subjects With ALS

Phase 3 Terminated 384 enrolled Shionogi

RandomizedParallel-groupQuadruple-blindTreatment

Summary

To evaluate and compare the efficacy of two dosing regimens of oral edaravone in subjects with amyotrophic lateral sclerosis (ALS) based on the change in ALS Functional Rating Scale- Revised (ALSFRS-R) score from baseline up to Week 48:

Timeline

Start: 2020-11-13
Primary completion: 2023-09-29
Completion: 2023-09-29

Outcome

Missed primary endpoint

Stopped: “Futility was met.”

registry analysis (superiority test); Edaravone 105 mg (Once Daily) vs Edaravone 105mg (2 Weeks On/Off ); p = 0.777; ANCOVA NCT04569084 ↗

Publications

Results Rothstein J, Genge A, De Silva S, Zinman L, Chum M, Chio A, Sobue G, Aoki M, Yoshino H, Doyu M, Selness D, Todorovic V, Sasson N, Hirai M, Takahashi F, Salah A, Wamil A, Apple S. 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). Muscle Nerve. 2025 Sep;72(3):433-442. doi: 10.1002/mus.28448. Epub 2025 Jun 6.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Edaravone	Unknown	105 mg	Oral