Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
completion around

Description

Summary

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.

Details

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.

Keywords

Amyotrophic Lateral Sclerosis, ALS, biomarkers, edaravone, Radicava®, Radicava ORS®, motor neuron disease, Edaravone (Radicava®/Radicava ORS®)

Eligibility

You can join if…

Open to people ages 18 years and up

  • 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

You CAN'T join if...

  • Participant with a contraindication to Edaravone
  • Participant is participating in an interventional clinical trial

Locations

  • UCSF
    San Francisco California 94417 United States
  • UC Davis Health
    Sacramento California 95817 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Mitsubishi Tanabe Pharma America Inc.
ID
NCT04259255
Study Type
Observational
Participants
Expecting 300 study participants
Last Updated