Amyotrophic Lateral Sclerosis (ALS)Phase 3NCT01492686
Phase 3 Study of MCI-186 for Treatment of Amyotrophic Lateral Sclerosis
A Phase 3 study testing MCI-186 and MCI-186 in open label phase in people with Amyotrophic Lateral Sclerosis (ALS), run by Shionogi at 1 site in Japan.
Trial summary
The primary objective of the study is to confirm the efficacy of 60 mg of MCI-186 via intravenous drip infusion once a day in the patients with ALS based on the changes in the revised ALS functional rating scale (ALSFRS-R) scores after 24 weeks administration in double-blind, placebo-controlled manner. Read more about this study
- Who is it for?
- Ages 20 to 75 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- MCI-186 and MCI-186 in open label phase
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 137 participants
- Where
- 1 siteJapan
- Study dates
- 31 Dec 2011to 31 Oct 2014
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients whose conditions are defined as "definite ALS"or "probable ALS"diagnostic criteria El Escorial and revised Airlie House.
- Patients who can eat a meal, excrete, or move with oneself alone, and do not need assistance in everyday life.
- Patients of less than 2 years after the onset of ALS.
- Patients whose progress of the condition during 12 weeks before administration meet other requirements.
Who cannot join
- Patients with such complications as Parkinson's disease, schizophrenia, dementia, renal failure, or other severe complication, and patients who have the anamnesis of hypersensitivity to edaravone.
- Pregnant, lactating, and probably pregnant patients, and patients who want to become pregnant, and patients who can not agree to contraception.
- Patients who have participated in other trials within 12 weeks before consent, or who are participating in other clinical trials at present.
- In addition to the above exclusion criteria, patients judged to be inadequate to participate in this study by their physician.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Efficacy and Safety Study of MCI-186 for Treatment of the Patients With Amyotrophic Lateral Sclerosis (ALS) 2
The primary objective of the study is to confirm the efficacy of 60 mg of MCI-186 via intravenous drip infusion once a day in the patients with ALS based on the changes in the revised ALS functional rating scale (ALSFRS-R) scores after 24 weeks administration in double-blind, placebo-controlled manner. The study is also to examine the safety of MCI-186 to the ALS patients.
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- MCI-186, Placebo, MCI-186 in open label phase
- Keywords
- Amyotrophic Lateral Sclerosis (ALS)
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MCI-186Drug
Two ampoules (60 mg) of MCI-186 injection are intravenously administered once a day, for successive 14 days, followed by 14 days observation period (first cycle). The following treatment (10 days' administration during 14 days) - observation (14 days) cycle is repeated five times (2nd-6th cycles).
PlaceboDrug
Two ampoules of placebo injection are intravenously administered once a day, for successive 14 days, followed by 14 days observation period (first cycle). The following treatment (10 days' administration during 14 days) - observation (14 days) cycle is repeated five times (2nd-6th cycles).
MCI-186 in open label phaseDrug
All patients after the double blind phase may receive MCI-186 in 7th until 12th treatment - observation cycles at the patients' will.
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | Not described Receives: Drug: MCI-186; Drug: MCI-186 in open label phase |
| Arm 2 | Placebo comparator | Not described Receives: Drug: Placebo; Drug: MCI-186 in open label phase |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Revised ALS Functional Rating Scale (ALSFRS-R) Score in Full Analysis Set (FAS) Population at 24 Weeks
0=worst; 48=best
Measured: baseline and 24 weeks
Secondary outcomes (8)
Number of Participants With Death or a Specified State of Disease Progression
Any of "death, disability of independent ambulation, loss of upper limbs function, tracheotomy, use of respirator, use of tube feeding and loss of useful speech" was defined as an event.
Measured: 24 weeks
Change From Baseline in % Forced Vital Capacity (%FVC) in Full Analysis Set (FAS) Population at 24 Weeks
Measured: baseline and 24 weeks
Change From Baseline in Modified Norris Scale Score in Full Analysis Set (FAS) Population at 24 Weeks
The Modified Norris Scale is a measure of movement disorder for patients with ALS. 0=worst; 102=best
Measured: baseline and 24 weeks
Change From Baseline in ALS Assessment Questionnaire (40 Items) (ALSAQ40) in Full Analysis Set (FAS) Population at 24 Weeks
The ALSAQ40 score is a measure of QoL for patients with ALS. The ALSAQ40 evaluates domains that include physical mobility, Activities of daily living (ADL) and independence, eating and drinking, communication, and emotional reactions. 200=worst; 40=best
Measured: baseline and 24 weeks
Percentage of Participants With Adverse Events
Measured: 24 weeks
Percentage of Participants With Adverse Drug Reactions
Measured: 24 weeks
Laboratory Tests Percentage of Participants With Adverse Events by System Organ Class (SOC) of "Investigations" (PT, MedDRA Ver. 17.0)
Measured: 24 weeks
Percentage of Participants With Abnormal Values in Sensory Examinations
Measured: baseline and 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Dec 2011 (estimated)
- Study start31 Dec 2011
- Primary completion30 Sep 2014
- Study completion31 Oct 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral Sclerosis (ALS)Studies of the same condition, open and under way.
- More trials run by ShionogiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 137 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shionogi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Shionogi Clinical Trials Administrator Clinical Support Help Line, Study director, Shionogi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01492686
- Sponsor's protocol ID
- MCI186-19
- Status last verified
- Jun 2026
- First submitted
- 11 Dec 2011
- Last updated
- 21 Jul 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Writing Group; Edaravone (MCI-186) ALS 19 Study Group. Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2017 Jul;16(7):505-512. doi: 10.1016/S1474-4422(17)30115-1. Epub 2017 May 15.
PubMed 28522181WRITING GROUP ON BEHALF OF THE EDARAVONE (MCI-186) ALS 19 STUDY GROUP. Open-label 24-week extension study of edaravone (MCI-186) in amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2017 Oct;18(sup1):55-63. doi: 10.1080/21678421.2017.1364269.
PubMed 28872920Abrahao A, Da Silva P, Ciepielewska M, Zinman L. Real-world safety and tolerability of intravenous edaravone in patients with amyotrophic lateral sclerosis. Neurodegener Dis Manag. 2025 Dec;15(6):305-312. doi: 10.1080/17582024.2025.2552610. Epub 2025 Aug 29.
PubMed 40878665Brooks BR, Pioro EP, Sakata T, Takahashi F, Hagan M, Apple S. The effects of intervention with intravenous edaravone in Study 19 on hospitalization, tracheostomy, ventilation, and death in patients with amyotrophic lateral sclerosis. Muscle Nerve. 2023 Oct;68(4):397-403. doi: 10.1002/mus.27946. Epub 2023 Jul 31.
PubMed 37525592Takei K, Takahashi F, Liu S, Tsuda K, Palumbo J. Post-hoc analysis of randomised, placebo-controlled, double-blind study (MCI186-19) of edaravone (MCI-186) in amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2017 Oct;18(sup1):49-54. doi: 10.1080/21678421.2017.1361443.
PubMed 28872913