Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development

التفاصيل البيبلوغرافية
العنوان: Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development
المؤلفون: Sabrina, Paganoni, James D, Berry, Melanie, Quintana, Eric, Macklin, Benjamin R, Saville, Michelle A, Detry, Marianne, Chase, Alexander V, Sherman, Hong, Yu, Kristin, Drake, Jinsy, Andrews, Jeremy, Shefner, Lori B, Chibnik, Matteo, Vestrucci, Merit E, Cudkowicz, Margherita, Torti
المصدر: Annals of Neurology. 91:165-175
بيانات النشر: Wiley, 2022.
سنة النشر: 2022
مصطلحات موضوعية: Clinical Trials as Topic, Neurology, Endpoint Determination, Research Design, Amyotrophic Lateral Sclerosis, Animals, Humans, Neurology (clinical), Biomarkers
الوصف: 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.
تدمد: 1531-8249
0364-5134
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::53991fcbeb0b07905e37fb08161b3a4e
https://doi.org/10.1002/ana.26285
حقوق: OPEN
رقم الأكسشن: edsair.doi.dedup.....53991fcbeb0b07905e37fb08161b3a4e
قاعدة البيانات: OpenAIRE