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Amyotrophic Lateral SclerosisPhase 2/3COMBAT-ALSNCT04057898

Evaluation of MN-166 (Ibudilast) for 12 Months Followed by an Open-label Extension for 6 Months in Patients With ALS

A Phase 2/3 study testing MN-166 in people with Amyotrophic Lateral Sclerosis, run by MediciNova at 16 sites in 2 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

A Phase 2b/3 multicenter, randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy, safety and tolerability of MN-166 given to ALS participants for 12 months followed by a 6-month open-label extension phase. Read more about this study

Who is it for?
Ages 18 to 80 years
Conditions the study is for
Amyotrophic Lateral Sclerosis
What is being tested?
MN-166
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
234 participants
Where
16 sitesin 2 countries
Study dates
28 May 2020to Apr 2028
Phase
Phase 2/3
Are there results?
Not yet: the study is under way

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 18 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Major Inclusion Criteria:

  • Male or female subjects age 18 - 80 years, inclusive;
  • Diagnosis of familial or sporadic ALS as defined by the El Escorial-Revised (2000) research diagnostic criteria for ALS [clinically definite, clinically probable, probable-laboratory-supported];
  • ALS onset of ≤18 months from first clinical signs of weakness prior to screening;
  • If currently using riluzole, subject must be on a stable dose for at least 30 days prior to initiation of study drug;
  • If currently using edaravone, subject should have completed at least 14 days of their initial treatment cycle prior to initiation of study drug;
  • Last documented pulmonary function test result (i.e., slow vital capacity or forced vital capacity) must be greater than or equal to 70% predicted;
  • Able to swallow study medication capsules;
  • No known allergies to the study drug or its excipients;
  • Received pneumococcal vaccine within 6 years prior to starting clinical trial.

Major Exclusion Criteria:

  • Confirmed hepatic insufficiency or abnormal liver function (AST and/or ALT >3 times upper limit of normal);
  • Currently diagnosed with a clinically significant psychiatric disorder or dementia that would preclude evaluation of symptoms;
  • Currently use or treated with parenteral (intramuscular or intravenous) high dose (>25 mg/week) Vitamin B12 within 30 days prior to study drug administration;
  • Poor peripheral venous access that will limit the ability to draw blood as judged by the Investigator;
  • Currently participating, or has participated in a study with an investigational or marketed compound or device within 30 days or 5 half-lives, whichever is shorter, prior to signing the informed consent;
  • Use of tracheostomy or >22/24-hour ventilatory support.

Trial locations and contacts

16 sites in 2 countries.

16 sites in 2 countries

United States10 sites

  • Lehigh Valley Health Network

    Allentown, Pennsylvania, 18103

  • Augusta University

    Augusta, Georgia, 30912

  • Johns Hopkins University

    Baltimore, Maryland, 21287

  • University of Virginia Health System

    Charlottesville, Virginia, 22908

  • Duke University

    Durham, North Carolina, 27705

  • Indiana University IU Health Neuroscience Center

    Indianapolis, Indiana, 46202

  • Mayo Clinic

    Jacksonville, Florida, 32224

  • Hennepin Healthcare Research Institute

    Minneapolis, Minnesota, 55415

  • University of California

    Orange, California, 92868

  • SUNY Upstate Medical University

    Syracuse, New York, 13210

Other trials in United States

Canada6 sites

  • University of Alberta Hospital

    Edmonton, Alberta, T6G 2G3

  • McMaster University Medical Center

    Hamilton, Ontario, L8N 3Z5

  • Montreal Neurological Institute and Hospital

    Montreal, Quebec, H3A 2B4

  • Hopital de L'Enfant-Jesus, CHU de Quebec-Universite Laval

    Québec, G1J 1Z4

  • University of Saskatchewan - Sastakoon Hospital

    Saskatoon, Saskatchwean, S7K 0M7

  • Sunnybrook Research Institute

    Toronto, Ontario, M4N 3M5

Other trials in Canada

About this study

Official title: A Phase 2b/3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, 12 Month Clinical Trial to Evaluate the Efficacy and Safety of MN-166 (Ibudilast) Followed by Open-Label Extension Phase in Subjects With Amyotrophic Lateral Sclerosis

A Phase 2b/3 multicenter, randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy, safety and tolerability of MN-166 given to ALS participants for 12 months followed by a 6-month open-label extension phase.

Read the detailed description

This is a Phase 2b/3 multicenter, randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy, safety and tolerability of MN-166 followed by an open-label extension phase compared to matching placebo in subjects diagnosed with ALS.

The study will consist of a screening phase (up to 30 days) followed by a double-blind phase (12 months). Following the screening phase, subjects who continue to meet entry criteria will be randomly assigned to one of two treatment groups: MN-166 or matching placebo in a 1:1 ratio. Upon completion of the double-blind phase, subjects will be given the option to continue to the Open-label Extension Phase for a period of six months.

Being tested
MN-166, placebo
Keywords
ALS, MN-166, ibudilast, amyotrophic lateral sclerosis
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.

  • MN-166Drug

    Also called ibudilast

    Subjects will take MN-166 for 12 months followed by a 6-month open-label extension phase.

  • placeboDrug

    Subjects will take matching placebo for 12 months followed by a 6-month open-label extension phase.

Study groups in NCT04057898
Study groupRoleWhat they receive
MN-166ExperimentalSubjects will take MN-166 10 mg capsules, up to 50 mg twice a day for 12 months.
Receives: Drug: MN-166
placeboPlacebo comparatorSubjects will take up to 5 matching placebo capsules twice a day for 12 months.
Receives: Drug: placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change from baseline in ALSFRS-R score at Month 12 (or last measurement before death in case of censoring) and survival time.

    The amyotrophic lateral sclerosis functional rating scale-revised, or ALSFRS-R, measures the functional status of subjects with ALS. It is based on 12 items, each of which is rated on a 5-point scale (0 to 4). The rate of total functional disability thus ranges from 0 (maximum disability) to 48 (normal function) points.

    Measured: 12 months

Secondary outcomes (7)

  1. Mean change from baseline of muscle strength measured by hand-held dynamometry

    Hand-held dynamometry, or HHD, is used to measure the force generated by each muscle. The scale ranges from 0 (no visible movement of the part) to 10 (holds test position against strong pressure). Thus, the higher the total score, the higher muscle strength is observed.

    Measured: Baseline, Treatment Phase Week 6, Months 3, 6, 9 and12 time points.

  2. Mean change from baseline on quality of life assessed by ALSAQ-5 at Month 12

    The Amyotrophic Lateral Sclerosis Assessment Questionnaire, or ALSAQ-5, is a patient self-report questionnaire specifically designed to measure 5 areas of health: physical mobility, activities of daily living and independence, eating and drinking, communication and emotional functioning. The subject is asked about 5 different areas of difficulties in their daily lives: ability to stand up, use of limbs, consuming solid food, level of speech coherence, and degree of hope about the future.Each question provides 5 choices from which to choose: Never, Rarely, Sometimes, Often, and Always or cannot do at all.

    Measured: 12 months

  3. Mean change from baseline of functional activity measured by ALSFRS-R at Month 12

    The ALSFRS-R assessment tool measures the functional status of subjects with ALS. It is based on 12 items, each of which is rated on a 5-point scale (0 to 4). The rate of total functional disability thus ranges from 0 (maximum disability) to 48 (normal function) points. In this context, the ALSFRS-R total score change (lower, same, higher) is documented.

    Measured: 12 months

  4. Responders, measured in percent of subjects overall, whose ALSFRS-R total score was stable or improved

    Proportion of subjects in which ALSFRS-R total score was stable or improved.

    Measured: 12 months

  5. Time to survival

    Defined by death or permanent dependency to ventilator or tracheostomy.

    Measured: 12 months

  6. Number of Participants with Treatment-Related Adverse Events as Assessed by CTCAE v4.0

    The incidence of treatment-emergent adverse events (TEAEs), severity (mild, moderate, severe), as well as relationship to study treatment (not related, possibly related, probably related) and whether they are considered serious.

    Measured: 12 months

  7. Changes from Baseline in Laboratory Values

    Incidence of out-of-normal-range values and markedly abnormal change from baseline in laboratory safety test variables by treatment group.

    Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Aug 2019
  2. Study start28 May 2020
  3. Primary completionApr 2027 (estimated)
  4. Study completionApr 2028 (estimated)

Resources

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 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
234 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04057898
Sponsor's protocol ID
MN-166-ALS-2301
Acronym
COMBAT-ALS
Status last verified
Mar 2026
First submitted
6 Aug 2019
Last updated
6 Mar 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Trist BG, Fifita JA, Hogan A, Grima N, Smith B, Troakes C, Vance C, Shaw C, Al-Sarraj S, Blair IP, Double KL. Co-deposition of SOD1, TDP-43 and p62 proteinopathies in ALS: evidence for multifaceted pathways underlying neurodegeneration. Acta Neuropathol Commun. 2022 Aug 25;10(1):122. doi: 10.1186/s40478-022-01421-9.

    PubMed 36008843

  • Oskarsson B, Maragakis N, Bedlack RS, Goyal N, Meyer JA, Genge A, Bodkin C, Maiser S, Staff N, Zinman L, Olney N, Turnbull J, Brooks BR, Klonowski E, Makhay M, Yasui S, Matsuda K. MN-166 (ibudilast) in amyotrophic lateral sclerosis in a Phase IIb/III study: COMBAT-ALS study design. Neurodegener Dis Manag. 2021 Dec;11(6):431-443. doi: 10.2217/nmt-2021-0042. Epub 2021 Nov 24.

    PubMed 34816762

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing MN-166 in people with Amyotrophic Lateral Sclerosis, run by MediciNova at 16 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 6 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 16 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change from baseline in ALSFRS-R score at Month 12 (or last measurement before death in case of censoring) and survival time. (time frame: 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04057898, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Mar 2026.

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