Efficacy and Safety Study of Oral Edaravone Administered in Subjects With ALS

Brief Title

Efficacy and Safety Study of Oral Edaravone Administered in Subjects With ALS

Official Title

A Phase 3b, Multicenter, Randomized, Double-Blind Study to Evaluate Efficacy and Safety of Oral Edaravone Administered for a Period of 48 Weeks in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Brief Summary

      To evaluate and compare the efficacy of two dosing regimens of oral edaravone in subjects
      with amyotrophic lateral sclerosis (ALS) based on the change in ALS Functional Rating Scale-
      Revised (ALSFRS-R) score from baseline up to Week 48:
    


Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Change in ALS Functional Rating Scale- Revised (ALSFRS-R) score from baseline to Week 48 of treatment

Secondary Outcome

 Change from baseline in % slow vital capacity (SVC) at Week 48

Condition

ALS

Intervention

MT-1186

Study Arms / Comparison Groups

 MT-1186
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

380

Start Date

November 13, 2020

Completion Date

March 2022

Primary Completion Date

March 2022

Eligibility Criteria

        Inclusion Criteria:

          1. Subjects must provide a signed and dated informed consent form (ICF) to participate in
             the study. Subjects must be able (in the judgment of the Investigator) to understand
             the nature of the study and all risks involved with participation in the study.
             Subjects must be willing to cooperate and comply with all protocol restrictions and
             requirements.

          2. Subjects will be male or female, ≥ 18 to 75 years of age at the time the ICF is
             signed.

          3. Subjects will be diagnosed with Definite ALS or Probable ALS according to the El
             Escorial revised criteria for the diagnosis of ALS.

          4. Subjects with a baseline score ≥ 2 points on each individual item of the ALSFRS- R at
             screening and baseline visits.

          5. Subjects have a screening and baseline %forced vital capacity (FVC) ≥ 70%.

          6. Subjects with 1 to 4 points decline for 8 weeks in ALSFRS-R total score between
             screening and baseline visits.

          7. Subjects whose first symptom of ALS has occurred within 2 years of providing written
             informed consent.

        Exclusion Criteria:

        Exclusions Related to Primary Diagnosis

          1. Subjects with a history of spinal surgery after the onset of ALS, such as surgery for
             cervical spondylosis or a herniated disc, or plans for such surgery during the study
             period.

             Exclusions Related to Other Neurological Disorders (including, but not limited to the
             following)

          2. Subjects with the possibility that the current symptoms may be symptoms of a disease
             requiring differential diagnosis, such as cervical spondylosis and multifocal motor
             neuropathy, cannot be ruled out.

             Exclusions Related to General Health or Concomitant Conditions

          3. Subjects undergoing treatment for a malignancy.

          4. Subjects with a complication that could have a significant effect on efficacy
             evaluations, such as Parkinson's disease or syndrome, schizophrenia, bipolar disorder,
             and dementia.

          5. Subjects who have the presence or history of any clinically significant (CS) disease
             (except ALS) that could interfere with the objectives of the study (the assessment of
             safety and efficacy) or the safety of the subject, as judged by the Investigator.

          6. Subjects who are female and pregnant (a positive pregnancy test) or lactating at the
             screening visit (Visit 1).

          7. Subjects of childbearing potential unwilling to use acceptable method of contraception
             from the screening visit until 3 months after the last dose of study medication.
             Subjects who are sexually active who do not agree to use contraception during the
             study period.

          8. Subjects who have a significant risk of suicidality. Subjects with any suicidal
             behavior or suicidal ideation of type 4 (active suicidal ideation with some intent to
             act, without a specific plan) or type 5 (active suicidal ideation with specific plan
             and intent) based on the Columbia-Suicide Severity Rating Scale (C-SSRS) within the 3
             months before the screening visit.

          9. Subjects who have alanine aminotransferase (ALT) or aspartate aminotransferase (AST)
             elevations greater than 2 times the upper limit of normal (ULN) at screening.

         10. Subjects with a Glomerular Filtration Rate (GFR) < 30 mL/Min Per 1.73 m2 at screening,
             using the Larsson Equation.

             Exclusions Related to Medications

         11. Subjects with history of hypersensitivity to edaravone, any of the additives or
             inactive ingredients of edaravone, or sulfites.

         12. Subjects with hereditary problems of fructose intolerance (eg, fructose, sucrose,
             invert sugar, and sorbitol).

         13. Subjects who participated in another study and were administered an investigational
             product within 1 month or 5 half-lives of the investigational agent, whichever is
             longer, before providing informed consent for the present study.

         14. Subjects who have received any previous treatment with edaravone.

         15. Subjects who have received stem cell therapy.

         16. Subjects who are unable to take their medications orally at baseline (Visit 2).
      

Gender

All

Ages

18 Years - 75 Years

Accepts Healthy Volunteers

No

Contacts

Head of Medical Science, +1-800-313-9381, [email protected]

Location Countries

Canada

Location Countries

Canada

Administrative Informations


NCT ID

NCT04569084

Organization ID

MT-1186-A02


Responsible Party

Sponsor

Study Sponsor

Mitsubishi Tanabe Pharma Development America, Inc.


Study Sponsor

Head of Medical Science, Study Director, Mitsubishi Tanabe Pharma Development America, Inc.


Verification Date

April 2021