Language

English

Publication Date

5-1-2025

Journal

JACC: Basic to Translational Science

DOI

10.1016/j.jacbts.2025.04.003

PMID

40436529

PMCID

PMC12235394

PubMedCentral® Posted Date

5-26-2025

PubMedCentral® Full Text Version

Post-print

Abstract

Phase II clinical trials play an important role in drug development, providing key data that guide decision-making for promising therapeutic candidates. An important objective of phase II is to establish proof of concept by demonstrating that the drug produces its intended biological effects in the target population. Phase II trials also evaluate pharmacokinetics, pharmacodynamics, safety, and dose-response relationships. Ultimately, the goal is to generate the evidence needed to inform go/no-go decisions for further development. However, as we will discuss, phase II studies have inherent limitations and cannot fully predict phase III outcomes. In this JACC: Basic to Translational Science and Heart Failure Collaboratory position paper, we examine the value and constraints of phase II programs, using prior examples of heart failure trials, with the goal of providing insights that will help investigators and sponsors to derisk go/no-go decisions.

Keywords

clinical trials, heart failure, phase II

Published Open-Access

yes

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