Structure of proof of concept studies that precede a nonalcoholic steatohepatitis development program.
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ABSTRACT:
SUBMITTER: Williams RN
PROVIDER: S-EPMC5367378 | biostudies-literature | 2017 Apr
REPOSITORIES: biostudies-literature
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Structure of proof of concept studies that precede a nonalcoholic steatohepatitis development program.
Williams R N RN Filozof C C Goldstein B J BJ Cusi K K
Clinical pharmacology and therapeutics 20161229 4
Surrogate endpoints for clinical proof of concept (POC) trials in nonalcoholic steatohepatitis (NASH) are based upon expert pathological review of liver biopsies. During early development, these long-term POC studies (≥48 weeks) add cost and time to the "Go/No Go" decision process. However, it is possible to conduct short-term noninvasive POC studies utilizing biomarkers and magnetic resonance imaging. Here, we discuss the use of shorter noninvasive POC studies relative to biopsy-driven studies ...[more]
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