- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04259255
Radicava® (Edaravone) Findings in Biomarkers From ALS (REFINE-ALS)
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Treatment will be prescribed by HCPs in accordance with their clinical judgement and the prescribing information for Edaravone. The decision to prescribe Edaravone to the participants should be made separately from the decision to enroll then in the study. There will be no randomized assignments to treatment and no restrictions on the use of commercially available medications (but those participating in an experimental study, even if taking Edaravone, will be excluded). No experimental treatment is evaluated in this study. The intervention is limited to the collection of blood and urine samples for biomarker testing.
During the estimated study period, eligible patients who are prescribed Edaravone within the approved indication will be invited to participate in the study. An initial screening/baseline visit will be scheduled for participants who are considered for study participation.
Participants in this study will be followed from enrollment up to 24 weeks after treatment initiation (6 treatment cycles [each cycle consisting of 28 days], corresponding to a treatment period of approximately 24 weeks) or premature study discontinuation. Throughout the study period, the investigators will record participant baseline and follow-up information and perform clinical and biomarker assessments.
Study Type
Enrollment (Estimated)
Contacts and Locations
Study Contact
- Name: Sunny Rosenthal
- Phone Number: 617-724-2609
- Email: sjrosenthal@partners.org
Study Contact Backup
- Name: Ji Hong Lee
- Phone Number: 617-724-3867
- Email: jlee247@mgh.harvard.edu
Study Locations
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Ontario
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Toronto, Ontario, Canada, M4N 3M5
- Sunnybrook Research Institute
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Arizona
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Phoenix, Arizona, United States, 85013
- Barrow Neurological Institute
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California
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Los Angeles, California, United States, 90024
- UCLA Als Clinic
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Sacramento, California, United States, 95817
- UC Davis Health
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San Francisco, California, United States, 94417
- University of California, San Francisco
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Colorado
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Denver, Colorado, United States, 80309
- University of Colorado
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District of Columbia
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Washington, District of Columbia, United States, 20007
- Georgetown University Medical Center
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Florida
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Jacksonville, Florida, United States, 32224
- Mayo Clinic Florida
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Jacksonville, Florida, United States, 32209
- University of Florida, Jacksonville -Neurology Research Department
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Tampa, Florida, United States, 33612
- University of South Florida
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Maryland
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Baltimore, Maryland, United States, 21205
- Johns Hopkins University
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Massachusetts
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Boston, Massachusetts, United States, 02214
- Massachusetts General Hospital
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Michigan
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Grand Rapids, Michigan, United States, 49503
- Mercy Health
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Nebraska
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Lincoln, Nebraska, United States, 68506
- Neurology Associates, P.C.
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Nevada
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Las Vegas, Nevada, United States, 89145
- Las Vegas Clinic
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Ohio
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Columbus, Ohio, United States, 43215
- OhioHealth
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19140
- Temple University Lewis Katz School of Medicine
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Philadelphia, Pennsylvania, United States, 19107
- Jefferson Weinberg ALS Center
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Utah
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Salt Lake City, Utah, United States, 84112
- University of Utah
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Wisconsin
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Milwaukee, Wisconsin, United States, 53226
- Medical College of Wisconsin
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Sampling Method
Study Population
Description
Inclusion Criteria:
- Male and female aged 18 years or older at enrollment
- Sporadic or familial ALS diagnosed as possible, probable, probable-laboratory supported or definite as defined by the World Federation of Neurology revised El Escorial criteria
- Decision made to prescribe Edaravone prior to screening
- Participant will likely be able to obtain commercial Edaravone and likely to complete 6 cycles of treatment, per site investigator estimation
- Participant either naïve to Edaravone or who did not receive any Edaravone does within 1 month prior to screening
- Signed informed consent by the subject, or a witness if a subject cannot read or write or is physically unable to talk or write, obtained before any study-related activities are undertaken
Exclusion Criteria:
- Participant with a contraindication to Edaravone
- Participant is participating in an interventional clinical trial
Study Plan
How is the study designed?
Design Details
- Observational Models: Other
- Time Perspectives: Prospective
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
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Edaravone (Radicava®/Radicava ORS®)
During an estimated 12-month period, eligible participants who are prescribed Edaravone within the approved indication will be invited to participate in the study.
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Participants will be followed from enrollment up to 24 weeks after treatment initiation (6 treatment cycles - 28 days per cycle, corresponding to a treatment period of approximately 24 weeks) or premature study discontinuation. Biomarker testing and clinical assessments will be performed at baseline (at enrollment or before the start of cycle 1), and at cycles 1, 3, and 6. Dosing is 60 mg daily by intravenous infusion for 14 days for the initial treatment cycle, followed by daily dosing on 10 out of 14 days in subsequent treatment cycles.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Changes in levels of 8-F2 isoprostanes as a potential biomarker of oxidative stress, inflammation or neurodegeneration.
Time Frame: Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Collection of blood and/or urine samples for 8-F2 isoprostanes.
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Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Change in levels of 3-nitrotyrosine (3-NT) as a potential biomarker of oxidative stress, inflammation or neurodegeneration.
Time Frame: Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Collection of blood and/or urine samples for 3-NT.
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Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Change in levels of 8-hydroxy-2'-deoxyguanosine (8-OHdG) as a potential biomarker of oxidative stress, inflammation or neurodegeneration.
Time Frame: Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Collection of blood and/or urine samples 8-OHdG.
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Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Change in levels of urate as a potential biomarker of oxidative stress, inflammation or neurodegeneration.
Time Frame: Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Collection of blood and/or urine samples for urate.
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Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Change in levels of matrix metalloproteinase-9 (MMP-9) as a potential biomarker of oxidative stress, inflammation or neurodegeneration.
Time Frame: Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Collection of blood and/or urine samples for MMP-9.
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Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Change in levels of urinary neutrophin receptor p75 as a potential biomarker of oxidative stress, inflammation or neurodegeneration.
Time Frame: Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Collection of blood and/or urine samples for urinary neutrophin receptor p75.
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Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Change in levels of neurofilaments (Nf) (Heavy and Light) as a potential biomarker of oxidative stress, inflammation or neurodegeneration.
Time Frame: Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Collection of blood and/or urine samples for neurofilaments (Nf) (Heavy and Light).
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Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Change in levels of creatinine as a potential biomarkers of oxidative stress, inflammation or neurodegeneration.
Time Frame: Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Collection of blood and/or urine samples for creatinine.
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Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Change from baseline in the ALSFRS-R (ALS Functional Rating Scale .Revised) Score
Time Frame: Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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The ALSFRS-R is a quickly administered ordinal rating scale (ratings 0-4) used to determine participants' assessment of their capability and independence in 12 functional activities.
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Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Change from baseline in the King's Clinical Staging.
Time Frame: Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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The King's clinical staging is based on the number of body regions affected by ALS and the presence of respiratory or nutritional failure.
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Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Change from baseline in the ALSAQ-40 (ALS Assessment Questionnaire).
Time Frame: Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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The ALSAQ-40 is a questionnaire that consists of 40 questions/items with 5 discrete scales: physical mobility, activities of daily living and independence, eating and drinking, communication, emotional reactions.
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Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Change from baseline in the Appel ALS Score.
Time Frame: Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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The Appel ALS score consists of five sub-scores: bulbar, respiratory, muscle strength, and lower extremity and upper extremity function.
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Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Change from baseline in slow vital capacity.
Time Frame: Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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The vital capacity (VC) (percent of predicted normal) will be determined, using the upright slow VC method.
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Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Change from baseline in hand-held dynamometry.
Time Frame: Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Hand-held dynamometry (HHD) will be used as a quantitative measure of muscle strength for this study.
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Cycles 1, 3, and 6 of each Edaravone cycle (each cycle is 28 days).
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Collaborators and Investigators
Collaborators
Investigators
- Principal Investigator: James Berry, MD, MPH, Massachusetts General Hospital
Publications and helpful links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Metabolic Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Neuromuscular Diseases
- Neurodegenerative Diseases
- Spinal Cord Diseases
- TDP-43 Proteinopathies
- Proteostasis Deficiencies
- Motor Neuron Disease
- Amyotrophic Lateral Sclerosis
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Neuroprotective Agents
- Protective Agents
- Antioxidants
- Free Radical Scavengers
- Edaravone
Other Study ID Numbers
- REFINE-ALS
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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