Clinical Trials

Inclusion Criteria:

• Males or females, between the ages of 18 and 80 years of age, inclusive.
• Diagnosis of familial or sporadic ALS (defined as meeting the laboratory-supported probable, probable, or definite criteria for ALS according to the World Federation of Neurology El Escorial criteria published in 2000 [Brooks 2000]).
• Patients who meet the possible criteria are eligible if they have lower motor neuron findings; those who have purely upper motor neuron findings are ineligible.
• First symptom of ALS ≤ 24 months prior to screening. The qualifying first symptoms of ALS are limited to manifestations of weakness or spasticity in extremity, bulbar, or respiratory muscles. Cramps, fasciculations, or fatigue should not be taken in isolation as a first symptom of ALS.
• ALSFRS-R total score ≤ 44 at screening (patients whose scores fall below 45 or drop by ≥ 2 points from their initial screening total score 60 days ±7 days following the original screening date and continue to meet all other inclusion/exclusion criteria will be eligible).
• Upright FVC ≥ 65.0% of predicted for age, height, sex and ethnicity at screening according to Global Lung Initiative equation.
• Able to perform reproducible pulmonary function tests defined as being able to perform FVC at screening with variability of the 2 highest raw values of less than 10% with a maximum of 5 trials permitted. Screening FVC flow volume loops must be reviewed and approved by the central review process prior to randomization. Must be either on riluzole for ≥ 30 days prior to screening or have not taken it for at least 30 days prior to screening.
• Must have completed at least 2 cycles of edaravone at the time of screening or have not received it for at least 30 days prior to screening.
• Able to swallow whole tablets.
• Clinical laboratory findings within the normal range, or if outside the normal range, not deemed clinically significant by the Investigator, except as specifically indicated as laboratory exclusion.

Exclusion Criteria:

• eGFRCysC < 45.0 mL/min/1.73 m^2 at screening.
• Urine protein/creatinine ratio > 1000 mg/g (113 mg/mmol) at screening.
• Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥ 3-times the upper limit of normal (ULN).
• Total bilirubin (TBL), direct or indirect bilirubin above the ULN.
• Cognitive impairment, related to ALS or otherwise that impairs the patient’s ability to understand and/or comply with study procedures and provide informed consent.
• Other medically significant neurological conditions that could interfere with the assessment of ALS symptoms, signs or progression.
• Presence at screening of any medically significant cardiac, pulmonary, gastrointestinal, musculoskeletal, or psychiatric illness that might interfere with the patient’s ability to comply with study procedures or that might confound the interpretation of clinical safety or efficacy data.
• Has a tracheostomy.