Derisking Phase II Clinical Trials in Heart Failure

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.

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Publication Details

Journal
JACC Basic to Translational Science
Published
2025-05-01
DOI
https://doi.org/10.1016/j.jacbts.2025.04.003
Citations
1
Primary Topic
Heart Failure Treatment and Management
Type
article
Field-Weighted Citation Impact
1.39
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article

Derisking Phase II Clinical Trials in Heart Failure

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1 citations
JACC Basic to Translational Science
Heart Failure Treatment and Management
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Derisking Phase II Clinical Trials in Heart Failure

Ellis F. Unger, Janet Wittes, Biykem Bozkurt, G. Michael Felker, Scott David Solomon, Mona Fiuzat, Fady I. Malik, Benjamin R. Saville, Douglas L. Mann, Christopher M. O’Connor, Norman Stockbridge
article en
1 citations

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.

JACC Basic to Translational ScienceVol. 10(5)
Brigham and Women's Hospital (US), Michael E. DeBakey VA Medical Center (US), Baylor College of Medicine (US), Washington University in St. Louis (US), Duke Medical Center (US), Cytokinetics (United States) (US), Washington Center (US), Alaska Heart and Vascular Institute (US), Oldham Council (GB), Florida Atlantic University (US), Vanderbilt University Medical Center (US)
Good health and well-being
Openalex Percentile: Top 19%
Heart Failure Treatment and Management
1.39
DOIOpen Access PDFOpenAlex
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