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

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

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.

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;