The HEALEY ALS Platform Trial is a research trial that tests the safety and effectiveness of multiple treatment courses on Amyotrophic Lateral Sclerosis (ALS). Different courses of treatments may be ongoing at the same time and additional treatment courses may be added to the study as time goes by. Qualified participants will have a 3 in 4 chance of being randomly assigned to an active drug or a 1 in 4 chance of being randomly assigned to an inactive drug. After a treatment course is completed, participants may participate in another treatment course, or if available, continue in an optional extension period of the treatment.
Myasthenia gravis (MG) is a serious, sometimes life threatening, debilitating condition associated with numerous symptoms including muscular weakness and fatigue. This study is to see how effective and safe rozanolixizumab is in adult patients experiencing moderate to severe symptoms of generalized myasthenia gravis (gMG).
The study consists of a Screening Period of up to 4 weeks, followed by a 6-week double-blind Treatment Period and an Observation Period of 8 weeks. During this 18 weeks period there will be 14 visits to the study clinic.
Adult patients with ALS that have been recently perscribed Edaravone may qualify to participate in this observational study. Blood and urine samples will be collected to evaluate the effects of Edaravone on ALS and the severity of ALS. During an estimated 12-month period, eligible participants will have approximately 15 clinic visits.