Amyotrophic Lateral Sclerosis MedDRA version: 21.1 Level: PT Classification code 10002026 Term: Amyotrophic lateral sclerosis System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Product Name: Ibudilast
Product Code: MN-166
Pharmaceutical Form: Capsule
INN or Proposed INN: IBUDILAST
CAS Number: 50847-11-5
Current Sponsor code: MN-166
Concentration unit: mg milligram(s)
Concent
Sponsors
MediciNova, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Written or verbal informed consent is obtained and willing and able to comply with the protocol in the opinion of the Investigator; 2.Male or female subjects ages 18 to 80 years, inclusive; 3.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]; 4.ALS onset of = 18 months from first clinical signs of weakness prior to Screening; 5.Documented ALS history of location of disease onset (i.e. bulbar onset, limb onset) 6.A total ALSFRS-R score of at least 35 overall at screening and: a.No more than one of the 12 ALSFRS-R individual component items has a score of 1 or less at screening; b.For limb onset subjects, ALSFRS-R score of = 3 on item #1 (speech), # 2 (salivation) and # 3 (swallowing); 7.ALSFRS-R score progression from onset of the first symptom of weakness to the ALSFRS-R score at Screening of > 0.3 points and =65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: 1.Confirmed hepatic insufficiency or abnormal liver function (AST and/or ALT > 3 times the upper limit of normal); 2.Currently has a clinically significant psychiatric disorder or dementia which would preclude evaluation of symptoms; 3.Has a clinically significant medical condition (other than ALS) including the following: neurological, metabolic, hepatic, renal, hematological, pulmonary, cardiovascular, gastrointestinal, urological disorder, or central nervous system infection that would pose a risk to the subject if they were to participate in the study or that might confound the results of the study; 4.Female subject is lactating, pregnant or planning pregnancy at Screening or Baseline; 5.History of malignancy 450 ms; 8.History of HIV (human immunodeficiency virus), clinically significant chronic hepatitis, or other active infection; 9.Current use or treated with Edaravone® = 3 months prior to signing consent; 10.Current use or treated with Nuedexta® = 3 months prior to signing consent; 11.Current use or treated with Methylcobalamin Vitamin B12 = 3 months prior to signing consent 12.History of stomach or intestinal surgery or any other condition that could interfere with or is judged by the Investigator to interfere with absorption, distribution, metabolism, or excretion of study drug; 13.History of alcohol or substance abuse (DSM-5 criteria) = 3 months prior to screening or alcohol or substance dependence (DSM-5 criteria) = 12 months prior to screening; 14.Poor peripheral venous access that will limit the ability to draw blood as judged by the Investigator; 15.Currently participating, or has participated in, a study with an investigational or marketed compound or device = 3 months prior to signing the informed consent; 16.Unable to cooperate with any study procedures, unlikely to adhere to the study procedures and keep appointments, in the opinion of the Investigator; 17.Use of tracheostomy, tracheostomy invasive mechanical ventilation [TIMV].
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to evaluate the efficacy of MN-166 versus placebo on patient’s functional activity measured by ALSFRS-R score and time to survival in ALS subjects.;Secondary Objective: The secondary objective of the study is to evaluate the efficacy of MN-166 on muscle strength measured by hand-held dynamometry (HHD), the efficacy of MN-166 on quality of life measured by ALSAQ-5, the efficacy of MN-166 on functional activity measured by ALSFRS-R, the safety and tolerability of MN-166, to characterize the pharmacokinetics (PK) of MN-166 using population PK modeling and to measure survival. ;Primary end point(s): The primary endpoint is the change from baseline in ALSFRS-R score at Month 12 (or last measurement before death in case of censoring) and survival time. ;Timepoint(s) of evaluation of this end point: According to the protocol | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): •Mean change from baseline of muscle strength measured by hand-held dynamometry (HHD) at Month 12. •Mean change from baseline on quality of life assessed by ALSAQ-5 at Month 12. •Mean change from baseline of functional activity measured by ALSFRS-R at Month 12. •Responder analysis (%) defined as subjects whose ALSFRS-R was stable or improved over the 12-month Treatment Phase •Time to survival as defined by death or permanent dependency to ventilator or tracheostomy. •Safety and tolerability assessed by monitoring and recording all treatment-emergent adverse events (TEAEs) including serious adverse events (SAEs) and discontinuations due to TEAEs. Additional assessments will include monitoring of hematology, blood chemistry, and urine values, measurement of vital signs, ECGs, and medical history, physical and neurological examinations. •Relationship between MN-166 plasma levels and ALSFRS-R and adverse events. ;Timepoint(s) of evaluation of this end point: According to the protocol | — |
Countries
Germany, Greece, Hungary, Italy, Poland, Spain, United States
Contacts
Public ContactRegulatory Unit
Accelsiors CRO and Consultancy Services Ltd
Outcome results
None listed