HEALEY ALS Platform Trial: Start-Up

This trial is designed as a perpetual platform trial. This means that there is a single Master Protocol dictating the conduct of the trial. The Master Protocol describes the overall framework of the platform trial, including the target population, inclusion and exclusion criteria, randomization scheme, primary, secondary and exploratory endpoints, schedule of assessments, trial design, the mechanism for adding and for removing specific interventions for success or failure at interim analyses, and the statistical methodology and recommended statistical methods for evaluating interventions. Interventions (i.e., investigational products) are tested in trial regimens. Each trial regimen is described in its own Regimen-Specific Appendix (RSA) to the Master Protocol. The RSA will describe the nature of the intervention and its mechanism of action (MoA) including the mode and frequency of administration, number of doses, the specific target population (to be selected within the pre-defined subsets of the Master Protocol), additional enrollment criteria (if any), sample size, and other specific intervention-related information and assessments (safety or other assessments that may be in addition to those outlined in the Master Protocol).