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Updated: Oct 21, 2025
A Protocol for Comprehensive Assessment of Bulbar Dysfunction in Amyotrophic Lateral Sclerosis ALS
Published on: February 21, 2011
Radicava/Edaravone Findings in Biomarkers From Amyotrophic Lateral Sclerosis (REFINE-ALS): Protocol and Study Design
James Berry1, Benjamin Brooks1, Angela Genge1
1Massachusetts General Hospital (JB), Boston; Atrium Health Neurosciences Institute (BB), Carolinas Medical Center, University of North Carolina School of Medicine-Charlotte Campus; Montreal Neurological Institute and Hospital (AG), QC, Canada; Lewis Katz School of Medicine (TH-P), Temple University, Philadelphia, PA; Houston Methodist (S. Appel), TX; University of Miami (MB), FL; Barrow Neurological Institute (RB, JS), Phoenix, AZ; Harvard Medical School (MC), Boston, MA; University of South Florida (CG), Tampa; Oxford BioDynamics Inc. (JW), Wilmington, DE; and Mitsubishi Tanabe Pharma America (WA, CM, SN, S. Apple), Inc., Jersey City, NJ.
This study identifies biomarkers for edaravone
Area of Science:
- Neuroscience
- Pharmacology
- Biomarker Discovery
Background:
- Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease.
- Edaravone is an approved treatment for ALS, but its precise pharmacodynamic effects require further elucidation.
- Identifying reliable biomarkers is crucial for understanding treatment efficacy and guiding future therapeutic strategies.
Purpose of the Study:
- To identify quantifiable biomarkers reflecting the pharmacodynamic effects of edaravone in ALS patients.
- To assess real-world treatment outcomes and disease progression in patients receiving edaravone.
- To explore correlations between biomarkers, clinical outcomes, and genomic factors.
Main Methods:
- Prospective, observational, longitudinal, multicenter US study (NCT04259255) with at least 200 ALS patients.
- Edaravone treatment for 24 weeks (6 cycles) in treatment-naive or recently treated patients.
- Biomarker analysis (oxidative stress, inflammation, neuronal injury, muscle injury) using EpiSwitch and SOMAscan panels; clinical efficacy assessments (ALSFRS-R, King's staging, etc.); DNA collection.
Main Results:
- The study is projected for completion in 2022, with interim analyses planned.
- Data collection includes baseline and longitudinal assessments during edaravone treatment cycles.
- Analysis will focus on biomarker changes, clinical efficacy, disease progression rates, and survival.
Conclusions:
- Study findings are expected to enhance understanding of edaravone's pharmacodynamic effects in ALS.
- Identified biomarkers may serve as objective measures of treatment response.
- Results could inform personalized treatment approaches and future clinical trial designs for ALS.
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