Journal article
Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development
Annals of neurology, Vol.91(2), pp.165-175
02/2022
DOI: 10.1002/ana.26285
PMID: 34935174
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.
Details
- Title: Subtitle
- Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development
- Creators
- Sabrina Paganoni - Massachusetts General HospitalJames D Berry - Massachusetts General HospitalMelanie Quintana - Berry Consultants Austin TXEric Macklin - Massachusetts General HospitalBenjamin R Saville - Berry Consultants Austin TXMichelle A Detry - Berry Consultants Austin TXMarianne Chase - Massachusetts General HospitalAlexander V Sherman - Sean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MAHong Yu - Massachusetts General HospitalKristin Drake - Massachusetts General HospitalJinsy Andrews - Columbia UniversityJeremy Shefner - Barrow Neurological InstituteLori B Chibnik - Massachusetts General HospitalMatteo Vestrucci - Berry Consultants Austin TXMerit E Cudkowicz - Massachusetts General HospitalHealey ALS Platform Trial Study Group
- Contributors
- Andrea Swenson (Contributor) - University of Iowa, Neurology
- Resource Type
- Journal article
- Publication Details
- Annals of neurology, Vol.91(2), pp.165-175
- DOI
- 10.1002/ana.26285
- PMID
- 34935174
- NLM abbreviation
- Ann Neurol
- ISSN
- 0364-5134
- eISSN
- 1531-8249
- Grant note
- Arthur M. Blank Family Foundation AMG Charitable Foundation ALS One ALS Finding A Cure Tambourine ALS Collaborative Muscular Dystrophy Association The ALS Association Tackle ALS I AM ALS
- Language
- English
- Date published
- 02/2022
- Academic Unit
- Neurology
- Record Identifier
- 9984302204002771
Metrics
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