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;