Study to Investigate the Efficacy and Safety of FAB122 (Daily Oral Edaravone) in Patients With Amyotrophic Lateral Sclerosis

Study Overview
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Brief Summary
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Multicenter, multinational, double-blind, randomized (2:1), placebo-controlled Phase III study to investigate the efficacy and safety of 100 mg FAB122 once daily as oral formulation in ALS patients.

Official Title
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A Multicenter, Randomized, Double-blind, Placebo-controlled Study to Investigate the Efficacy and Safety of FAB122 in Patients With Amyotrophic Lateral Sclerosis

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Drug: FAB122
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Main Inclusion Criteria: Age 18 - 80 years (both inclusive), male or female; Diagnosis of definite, probable, probable laboratory supported or possible ALS as based on the El Escorial and the revised Airlie House diagnostic criteria for ALS; Onset of first symptoms* no longer than 24 months prior to randomization; *Date of onset is the date the patient reported one or more of the following symptoms: Muscle weakness in limbs Speech/swallowing difficulties Respiratory symptoms: dyspnea was noticed SVC equal to or more than 70% of the predicted normal value for gender, height and age at screening visit; Change in ALSFRS-R score between 0.35 points and 1.5 points per month (both inclusive) in the period from onset of first symptoms to the Screening visit; Capable of providing informed consent and complying with trial procedures. Main Exclusion Criteria: Diagnosis of Primary Lateral Sclerosis; Diagnosis of Frontotemporal Dementia; Diagnosis of other neurodegenerative diseases (e.g. Parkinson disease, Alzheimer disease); Diagnosis of polyneuropathy; Other causes of neuromuscular weakness; Have a significant pulmonary disorder not attributed to ALS and/or require treatment interfering with the evaluation of ALS on respiratory function; Use of intravenous (IV) edaravone within 6 months of the screening visit; Depend on mechanical ventilation (invasive or non-invasive) or require tracheostomy at Screening; Renal impairment as indicated by a creatinine clearance of less than 50 mL/min as calculated by the Cockcroft Gault equation; Subject has a history of clinically significant hepatic disease, hepatitis or biliary tract disease, or subject has a positive screening test for HIV, hepatitis B or C;

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: FAB122<br> | Drug: FAB122<br>* Daily dose 100 mg<br>|
| Placebo Comparator: Placebo<br> | Drug: Placebo<br>* Daily dose<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) | The total score ranges from 48 (no limitation in daily activities) to 0 (total inability). | 48 weeks |