A Double-blind Randomised, Placebo-controlled Clinical Trial to Test Ambroxol Treatment in ALS

Inclusion Criteria

• •1. Must have given written informed consent before any study related assessments are performed and must be able to understand purpose of the study, including any possible risks and adverse events.
• •2. ALS as diagnosed according to the recently proposed Gold Coast diagnostic criteria.
• •3. First symptom of ALS less than or equal to 18 months prior to screening. The qualifying first symptoms of ALS are limited to manifestations of weakness in extremity, bulbar, or respiratory muscles. Cramps, fasciculations, or fatigue should not be taken in isolation as a first symptom of ALS.
• •4. Forced vital capacity (FVC) greater than or equal to 60% of predicted value as adjusted for gender, height and age at the Screening Visit.
• •5. Male or female patients aged 18 years or greater (inclusive) and less than 85 years at the time of ALS diagnosis.
• •6. Able to swallow liquid.
• •7. Able to perform reproducible pulmonary function tests
• •8. Female patients must be post-menopausal or sterilized or must not be breastfeeding, have no intention to become pregnant during the study, and use acceptable methods of contraception or abstain from intercourse.
• •9. Male patients who have not had a vasectomy and confirmed zero sperm count must agree after receiving the first dose of study drug either to use acceptable methods of contraception or abstain from intercourse.
• •10. If on riluzole, stable dosing for 30-days prior to screening.
• •11. Pre-study ALSFRS-R progression between disease onset and screening of greater than or equal to 0.5 points/month (calculated by ALSFRS-R total score decline from 48 divided by the months since onset of ALS symptoms).