| Literature DB >> 34935174 |
Sabrina Paganoni1,2, James D Berry1, Melanie Quintana3, Eric Macklin4, Benjamin R Saville3,5, Michelle A Detry3, Marianne Chase1, Alexander V Sherman1, Hong Yu1, Kristin Drake1, Jinsy Andrews6, Jeremy Shefner7, Lori B Chibnik4, Matteo Vestrucci3, Merit E Cudkowicz1.
Abstract
Current therapeutic development in amyotrophic lateral sclerosis (ALS) relies on individual randomized clinical trials to test a specific investigational product in a single patient population. This approach has intrinsic limitations, including cost, time, and lack of flexibility. Adaptive platform trials represent a novel approach to investigate several interventions for a single disease in a continuous manner. 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. ANN NEUROL 2022;91:165-175.Entities:
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Year: 2022 PMID： 34935174 DOI： 10.1002/ana.26285
Source DB: PubMed Journal: Ann Neurol ISSN： 0364-5134 Impact factor: 10.422