Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development.
Document Type
Article
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.
Medical Subject Headings
Amyotrophic Lateral Sclerosis; Animals; Biomarkers; Clinical Trials as Topic; Endpoint Determination; Humans; Research Design
Publication Date
2-1-2022
Publication Title
Annals of neurology
ISSN
1531-8249
Volume
91
Issue
2
First Page
165
Last Page
175
PubMed ID
34935174
Digital Object Identifier (DOI)
10.1002/ana.26285
Recommended Citation
Paganoni, Sabrina; Berry, James D; Quintana, Melanie; Macklin, Eric; Saville, Benjamin R; Detry, Michelle A; Chase, Marianne; Sherman, Alexander V; Yu, Hong; Drake, Kristin; Andrews, Jinsy; Shefner, Jeremy; Chibnik, Lori B; Vestrucci, Matteo; Cudkowicz, Merit E; and Ladha, Shafeeq S., "Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development." (2022). Neurology. 1263.
https://scholar.barrowneuro.org/neurology/1263