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

NCT ID: NCT04057898

Last Updated: 2025-09-16

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 234 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-05-28
• Study Completion Date: 2026-12-31

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.

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.

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

placebo

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

Group Type: PLACEBO_COMPARATOR

placebo

Intervention Type: DRUG

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

MN-166

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

Group Type: EXPERIMENTAL

MN-166

Intervention Type: DRUG

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

Interventions

MN-166

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

Intervention Type: DRUG

placebo

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

Intervention Type: DRUG

Other Intervention Names

ibudilast

Eligibility Criteria

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.

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

MediciNova

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Project Management Team

Role: STUDY_CHAIR

Medicinova Inc

Locations

University of California

Orange, California, United States

Mayo Clinic

Jacksonville, Florida, United States

Augusta University

Augusta, Georgia, United States

Indiana University IU Health Neuroscience Center

Indianapolis, Indiana, United States

Johns Hopkins University

Baltimore, Maryland, United States

Hennepin Healthcare Research Institute

Minneapolis, Minnesota, United States

SUNY Upstate Medical University

Syracuse, New York, United States

Duke University

Durham, North Carolina, United States

Lehigh Valley Health Network

Allentown, Pennsylvania, United States

University of Virginia Health System

Charlottesville, Virginia, United States

University of Alberta Hospital

Edmonton, Alberta, Canada

McMaster University Medical Center

Hamilton, Ontario, Canada

Sunnybrook Research Institute

Toronto, Ontario, Canada

Montreal Neurological Institute and Hospital

Montreal, Quebec, Canada

University of Saskatchewan - Sastakoon Hospital

Saskatoon, Saskatchwean, Canada

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

Québec, , Canada

Countries

United States; Canada

References

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.

Reference Type: DERIVED

PMID: 36008843 (View on PubMed)

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.

Reference Type: DERIVED

PMID: 34816762 (View on PubMed)

Related Links

https://youtu.be/dayfOj08_3s

This webinar explains the COMBAT-ALS study design and background of MN-166 (ibudilast) as a potential treatment for ALS.

Other Identifiers

MN-166-ALS-2301

Identifier Type: -

Identifier Source: org_study_id