Reassessing Phase II Heart Failure Clinical Trials: Consensus Recommendations

Javed Butler*, Carine E. Hamo, James E. Udelson, Christopher O'Connor, Hani N. Sabbah, Marco Metra, Sanjiv J. Shah, Dalane W. Kitzman, John R. Teerlink, Harold S. Bernstein, Gabriel Brooks, Christophe Depre, Mary M. Desouza, Wilfried Dinh, Mark Donovan, Regina Frische-Danielson, Robert J. Frost, Dahlia Garza, Udo Michael Gohring, Jennifer HellawellJudith Hsia, Shiro Ishihara, Patricia Kay-Mugford, Joerg Koglin, Marc Kozinn, Christopher J. Larson, Martha Mayo, Li Ming Gan, Pierrre Mugnier, Sekayi Mushonga, Lothar Roessig, Cesare Russo, Afshin Salsali, Carol Satler, Victor Shi, Barry Ticho, Michael Van Der Laan, Clyde Yancy, Norman Stockbridge, Mihai Gheorghiade

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

11 Scopus citations


The increasing burden and the continued suboptimal outcomes for patients with heart failure underlines the importance of continued research to develop novel therapeutics for this disorder. This can only be accomplished with successful translation of basic science discoveries into direct human application through effective clinical trial design and execution that results in a substantially improved clinical course and outcomes. In this respect, phase II clinical trials play a pivotal role in determining which of the multitude of potential basic science discoveries should move to the large and expansive registration trials in humans. A critical examination of the phase II trials in heart failure reveals multiple shortcomings in their concept, design, execution, and interpretation. To further a dialogue on the challenges and potential for improvement and the role of phase II trials in patients with heart failure, the Food and Drug Administration facilitated a meeting on October 17, 2016, represented by clinicians, researchers, industry members, and regulators. This document summarizes the discussion from this meeting and provides key recommendations for future directions.

Original languageEnglish (US)
Article numbere003800
JournalCirculation: Heart Failure
Issue number4
StatePublished - Apr 1 2017


  • United States Food and Drug Administration
  • clinical trial
  • heart failure
  • mortality
  • safety

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine


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