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

NCT04057898 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: MN-166-ALS-2301
• Study Type: interventional
• Target: 234
• Locations: 2 countries
• Sites: 16

Brief 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.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS; MN-166; ibudilast; amyotrophic lateral sclerosis

Outcome Measures

Primary Outcomes (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.

  12 months

Secondary Outcomes (7)

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

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

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

  12 months

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

  12 months

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

  12 months

• Time to survival

  12 months

Study Arms (2)

MN-166

EXPERIMENTAL

Subjects will take MN-166 10 mg capsules, up to 50 mg twice a day for 12 months.

Drug: MN-166

placebo

PLACEBO COMPARATOR

Subjects will take up to 5 matching placebo capsules twice a day for 12 months.

Drug: placebo

Interventions

MN-166 DRUG

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

Also known as: ibudilast

MN-166

placebo DRUG

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

placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• MediciNova: lead

Study Sites (16)

University of California

Orange, California, 92868, United States

Location

Mayo Clinic

Jacksonville, Florida, 32224, United States

Location

Augusta University

Augusta, Georgia, 30912, United States

Location

Indiana University IU Health Neuroscience Center

Indianapolis, Indiana, 46202, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21287, United States

Location

Hennepin Healthcare Research Institute

Minneapolis, Minnesota, 55415, United States

Location

SUNY Upstate Medical University

Syracuse, New York, 13210, United States

Location

Duke University

Durham, North Carolina, 27705, United States

Location

Lehigh Valley Health Network

Allentown, Pennsylvania, 18103, United States

Location

University of Virginia Health System

Charlottesville, Virginia, 22908, United States

Location

University of Alberta Hospital

Edmonton, Alberta, T6G 2G3, Canada

Location

McMaster University Medical Center

Hamilton, Ontario, L8N 3Z5, Canada

Location

Sunnybrook Research Institute

Toronto, Ontario, M4N 3M5, Canada

Location

Montreal Neurological Institute and Hospital

Montreal, Quebec, H3A 2B4, Canada

Location

University of Saskatchewan - Sastakoon Hospital

Saskatoon, Saskatchwean, S7K 0M7, Canada

Location

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

Québec, G1J 1Z4, Canada

Location

Related Publications (2)

• 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.

  PMID: 36008843 DERIVED

• 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.

  PMID: 34816762 DERIVED

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

ibudilast

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Project Management Team

  Medicinova Inc

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 6, 2019
• First Posted: August 15, 2019
• Study Start: May 28, 2020
• Primary Completion (Estimated): April 1, 2027
• Study Completion (Estimated): April 1, 2028
• Last Updated: March 6, 2026

Record last verified: 2026-03

Locations

CA (6); US (10)