Study Purpose:
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.Study Status:
Recruiting
Disease:
Amyotrophic Lateral Sclerosis , ALS
Study Type:
Observational
Type of Intervention:
N/A
Intervention Name:
N/A
Placebo:
N/A
Phase:
N/A
Study Chair(s)/Principal Investigator(s):
James Berry, MD, MPH, Massachusetts General Hospital
Clinicaltrials.gov ID:
Neals Affiliated?
Yes
Coordinating Center Contact Information
Massachusetts General Hospital
Sunny Rosenthal / email hidden; JavaScript is required / 617-724-2609 / Jingqi Zhu / email hidden; JavaScript is required / 617-643-2522
Neurological Clinical Research Institute
Boston, Massachusetts, 02114 United States
Full Study Summary:
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.
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 Sponsor:
Mitsubishi Tanabe Pharma America Inc.
Participant Duration:
24 weeks
Estimated Enrollment:
300
Estimated Study Start Date:
10 / 21 / 2019
Estimated Study Completion Date:
03 / 01 / 2023
Posting Last Modified Date:
01 / 23 / 2023
Date Study Added to neals.org:
02 / 06 / 2020
Minimum Age:
18 Years
Maximum Age:
N/A
Can participants use Riluzole?
Yes
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
Barrow Neurological Institute | Active, not recruiting
Phoenix, Arizona
85013
United States
UCLA Als Clinic | Recruiting
Suchi Tiwari / email hidden; JavaScript is required
Los Angeles, California
90024
United States
UC Davis Health | Recruiting
Omaid Sarwary / email hidden; JavaScript is required
Erica Goude / email hidden; JavaScript is required
Sacramento, California
95817
United States
University of California, San Francisco | Recruiting
Neha Madugala / email hidden; JavaScript is required
San Francisco, California
94417
United States
University of Colorado | Recruiting
Neurology Research Group at UC Denver / email hidden; JavaScript is required
Denver, Colorado
80309
United States
Georgetown University Medical Center | Recruiting
Eli Wolfgang / email hidden; JavaScript is required
Principal Investigator : Shakti Nayar, MD
Washington, District of Columbia
20007
United States
University of Florida, Jacksonville -Neurology Research Department | Recruiting
Yasmeen Shabbir / email hidden; JavaScript is required
Jacksonville, Florida
32209
United States
Mayo Clinic Florida | Recruiting
Lisa Thuro / email hidden; JavaScript is required
Jacksonville, Florida
32224
United States
University of South Florida | Recruiting
Beverly Brooks / email hidden; JavaScript is required
Tampa, Florida
33612
United States
Johns Hopkins University | Recruiting
Betsy Mosmiller / email hidden; JavaScript is required
Baltimore, Maryland
21205
United States
Massachusetts General Hospital | Recruiting
Ryan Gifford / email hidden; JavaScript is required
Henry Rutherford / email hidden; JavaScript is required
Boston, Massachusetts
02214
United States
Mercy Health | Recruiting
Stacy Smith / 616-685-5597 / email hidden; JavaScript is required
Grand Rapids, Michigan
49503
United States
Neurology Associates, P.C. | Recruiting
Wendy D Bradford / email hidden; JavaScript is required
Lincoln, Nebraska
68506
United States
Las Vegas Clinic | Recruiting
Melanie Hjalmervik / email hidden; JavaScript is required
Las Vegas, Nevada
89145
United States
OhioHealth | Recruiting
Christina Fischer / email hidden; JavaScript is required
Principal Investigator : John Novak, MD
Columbus, Ohio
43215
United States
Jefferson Weinberg ALS Center | Recruiting
Mattison Worthy / 215-503-3213 / email hidden; JavaScript is required
Philadelphia, Pennsylvania
19107
United States
Temple University Lewis Katz School of Medicine | Recruiting
John Furey / email hidden; JavaScript is required
Kathleen Hatala / email hidden; JavaScript is required
Philadelphia, Pennsylvania
19140
United States
University of Utah | Recruiting
Michael Papadakis / email hidden; JavaScript is required
Salt Lake City, Utah
84112
United States
Medical College of Wisconsin | Recruiting
Marie Mejaki / email hidden; JavaScript is required
Milwaukee, Wisconsin
53226
United States
SunnyBrook Research Institute | Recruiting
Shirley Pham / 416-480-6100 / email hidden; JavaScript is required
Toronto, Ontario
M4N 3M5
Canada