Evaluation of MN-166 (Ibudilast) for 12 Months Followed by an Open-label Extension for 6 Months in Patients With ALS (COMBAT-ALS)
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|ClinicalTrials.gov Identifier: NCT04057898|
Recruitment Status : Recruiting
First Posted : August 15, 2019
Last Update Posted : March 21, 2023
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|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis||Drug: MN-166 Drug: placebo||Phase 2 Phase 3|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||230 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
|Actual Study Start Date :||May 28, 2020|
|Estimated Primary Completion Date :||December 2023|
|Estimated Study Completion Date :||December 2024|
Subjects will take MN-166 10 mg capsules, up to 50 mg twice a day for 12 months.
Subjects will take MN-166 for 12 months followed by a 6-month open-label extension phase.
Other Name: ibudilast
Placebo Comparator: placebo
Subjects will take up to 5 matching placebo capsules twice a day for 12 months.
Subjects will take matching placebo for 12 months followed by a 6-month open-label extension phase.
- 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 ]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.
- Mean change from baseline of muscle strength measured by hand-held dynamometry [ Time Frame: Baseline, Treatment Phase Week 6, Months 3, 6, 9 and12 time points. ]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.
- Mean change from baseline on quality of life assessed by ALSAQ-5 at Month 12 [ Time Frame: 12 months ]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.
- Mean change from baseline of functional activity measured by ALSFRS-R at Month 12 [ Time Frame: 12 months ]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.
- Responders, measured in percent of subjects overall, whose ALSFRS-R total score was stable or improved [ Time Frame: 12 months ]Proportion of subjects in which ALSFRS-R total score was stable or improved.
- Time to survival [ Time Frame: 12 months ]Defined by death or permanent dependency to ventilator or tracheostomy.
- Number of Participants with Treatment-Related Adverse Events as Assessed by CTCAE v4.0 [ Time Frame: 12 months ]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.
- Changes from Baseline in Laboratory Values [ Time Frame: 12 months ]Incidence of out-of-normal-range values and markedly abnormal change from baseline in laboratory safety test variables by treatment group.
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|Ages Eligible for Study:||18 Years to 80 Years (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04057898
|Contact: Project Management Teamfirstname.lastname@example.org|
|Study Chair:||Kazuko Matsuda, MD PhD MPH||Medicinova Inc|
|Other Study ID Numbers:||
|First Posted:||August 15, 2019 Key Record Dates|
|Last Update Posted:||March 21, 2023|
|Last Verified:||March 2023|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
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