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HEALEY ALS Platform Trial - Master Protocol
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. The 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, study endpoints, schedule of assessments, trial design, the mechanism for adding and removing interventions, and the statistical methodology and recommended statistical methods for evaluating interventions.
The HEALEY ALS Platform Trial is a research trial that tests the safety and effectiveness of multiple treatments in ALS. A regimen is a specific course of treatment, each with a different study drug.The following things will happen in this research study: Blood and urine sample collection; Completion of questionnaires; Physical and neurological exams; Vital signs, current and historical review of medical information about general health and medication use review; Muscle strength testing; Measurement of the electrical activity of the heart with an electrocardiogram (ECG); and Measurement of respiratory (breathing) function.Participants will also take either the study drug, or placebo, according to the study schedule
Slow vital capacity (SVC) at least 50% predicted
Time since onset of weakness due to ALS within 36 months
Able to swallow pills and liquids
Clinically significant unstable medical condition (other than ALS) that would pose a risk to you