Radicava® (Edaravone) Findings in Biomarkers From ALS (REFINE-ALS)

NCT ID: NCT04259255

Last Updated: 2023-08-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

UNKNOWN

Total Enrollment

300 participants

Study Classification

OBSERVATIONAL

Study Start Date

2019-10-21

Study Completion Date

2024-03-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

REFINE-ALS is a prospective, observational, longitudinal, multicenter study designed to identify biomarkers to serve as quantifiable biological non-clinical measures of Edaravone effects in ALS. Epigenetic and protein biomarkers will also be investigated.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Amyotrophic Lateral Sclerosis ALS

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

OTHER

Study Time Perspective

PROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

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.

Edaravone (Radicava®/Radicava ORS®)

Intervention Type DRUG

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.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Edaravone (Radicava®/Radicava ORS®)

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.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Radicava® Radicava ORS®

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Massachusetts General Hospital

OTHER

Sponsor Role collaborator

Mitsubishi Tanabe Pharma America Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

James Berry, MD, MPH

Role: PRINCIPAL_INVESTIGATOR

Massachusetts General Hospital

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Barrow Neurological Institute

Phoenix, Arizona, United States

Site Status

UCLA Als Clinic

Los Angeles, California, United States

Site Status

UC Davis Health

Sacramento, California, United States

Site Status

University of California, San Francisco

San Francisco, California, United States

Site Status

University of Colorado

Denver, Colorado, United States

Site Status

Georgetown University Medical Center

Washington D.C., District of Columbia, United States

Site Status

University of Florida, Jacksonville -Neurology Research Department

Jacksonville, Florida, United States

Site Status

Mayo Clinic Florida

Jacksonville, Florida, United States

Site Status

University of South Florida

Tampa, Florida, United States

Site Status

Johns Hopkins University

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Mercy Health

Grand Rapids, Michigan, United States

Site Status

Neurology Associates, P.C.

Lincoln, Nebraska, United States

Site Status

Las Vegas Clinic

Las Vegas, Nevada, United States

Site Status

OhioHealth

Columbus, Ohio, United States

Site Status

Jefferson Weinberg ALS Center

Philadelphia, Pennsylvania, United States

Site Status

Temple University Lewis Katz School of Medicine

Philadelphia, Pennsylvania, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

SunnyBrook Research Institute

Toronto, Ontario, Canada

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Canada

References

Explore related publications, articles, or registry entries linked to this study.

Berry J, Brooks B, Genge A, Heiman-Patterson T, Appel S, Benatar M, Bowser R, Cudkowicz M, Gooch C, Shefner J, Westra J, Agnese W, Merrill C, Nelson S, Apple S. Radicava/Edaravone Findings in Biomarkers From Amyotrophic Lateral Sclerosis (REFINE-ALS): Protocol and Study Design. Neurol Clin Pract. 2021 Aug;11(4):e472-e479. doi: 10.1212/CPJ.0000000000000968.

Reference Type DERIVED
PMID: 34476128 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

REFINE-ALS

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

ALS Reversals - Lunasin Regimen
NCT02709330 COMPLETED PHASE2
PREVENT ALL ALS Study
NCT06581861 RECRUITING
Determining the Safety of L-serine in ALS
NCT01835782 UNKNOWN PHASE1/PHASE2