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-  2016 

Proof of concept demonstration of optimal composite MRI endpoints for clinical trials

DOI: 10.1016/j.trci.2016.05.002

Keywords: Primary progressive aphasia, PPA, Clinical trial, Sample size, Power calculations, Composite endpoint, Alzheimer's disease, Structural magnetic resonance imaging, MRI, Region of interest

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Abstract:

Atrophy measures derived from structural MRI are promising outcome measures for early phase clinical trials, especially for rare diseases such as primary progressive aphasia (PPA), where the small available subject pool limits our ability to perform meaningfully powered trials with traditional cognitive and functional outcome measures

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