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Abstract: The HEALEY ALS Platform Trial is simultaneously testing multiple novel treatment regimens in persons with ALS with the goal of rapidly finding novel treatments. Within ALS, regulatory guidance recommends an integrated analysis of survival and function to assess efficacy of novel therapies. The traditional approach is a joint rank.
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The HEALEY ALS Platform Trial is enrolling people living with Amyotrophic Lateral Sclerosis (ALS) to test the efficacy of multiple investigational products. The platform trial is designed to decrease the time it takes to test new potential therapies and increase access to research for people living with ALS. One of the innovative features of.

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  • The HEALEY ALS Platform Trial, a national study led by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, allows researchers at 54 sites across the country to test multiple ...
  • The HEALEY ALS Platform Trial is being conducted by leading ALS experts from the Healey Center in collaboration with the Northeast ALS Consortium (NEALS) clinical trial network. In contrast with traditional trials that typically evaluate only one drug at a time, platform trials create an infrastructure for the ongoing study of multiple ...
  • The HEALEY ALS Platform Trial will study approximately 160 adults with ALS for each treatment. Participants will be randomized in a 3-to-1 ratio to be treated with verdiperstat 600 mg oral tablet ...
  • Already in use in oncology, this approach is now being employed more often in neurology. Here, we describe a newly launched platform trial for ALS. The Healey ALS Platform Trial is testing multiple investigational products concurrently in people with ALS, with the goal of rapidly identifying novel treatments, biomarkers, and trial endpoints.
  • Here, we describe a newly launched platform trial for ALS. The Healey ALS Platform Trial is testing multiple investigational products concurrently in people with ALS, with the goal of rapidly identifying novel treatments, biomarkers, and trial endpoints. ANN NEUROL 2022;91:165–175 Urgent Need for Novel Trial Designs in Neurology