Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development.
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.
Duke Scholars
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- Research Design
- Neurology & Neurosurgery
- Humans
- Endpoint Determination
- Clinical Trials as Topic
- Biomarkers
- Animals
- Amyotrophic Lateral Sclerosis
- 3209 Neurosciences
- 3202 Clinical sciences
Citation
Published In
DOI
EISSN
Publication Date
Volume
Issue
Start / End Page
Location
Related Subject Headings
- Research Design
- Neurology & Neurosurgery
- Humans
- Endpoint Determination
- Clinical Trials as Topic
- Biomarkers
- Animals
- Amyotrophic Lateral Sclerosis
- 3209 Neurosciences
- 3202 Clinical sciences