Reassessing Phase II Heart Failure Clinical Trials: Consensus Recommendations.

Journal Article (Journal Article)

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.

Full Text

Duke Authors

Cited Authors

  • Butler, J; Hamo, CE; Udelson, JE; O'Connor, C; Sabbah, HN; Metra, M; Shah, SJ; Kitzman, DW; Teerlink, JR; Bernstein, HS; Brooks, G; Depre, C; DeSouza, MM; Dinh, W; Donovan, M; Frische-Danielson, R; Frost, RJ; Garza, D; Gohring, U-M; Hellawell, J; Hsia, J; Ishihara, S; Kay-Mugford, P; Koglin, J; Kozinn, M; Larson, CJ; Mayo, M; Gan, L-M; Mugnier, P; Mushonga, S; Roessig, L; Russo, C; Salsali, A; Satler, C; Shi, V; Ticho, B; van der Laan, M; Yancy, C; Stockbridge, N; Gheorghiade, M

Published Date

  • April 2017

Published In

Volume / Issue

  • 10 / 4

PubMed ID

  • 28356300

Pubmed Central ID

  • PMC5400283

Electronic International Standard Serial Number (EISSN)

  • 1941-3297

Digital Object Identifier (DOI)

  • 10.1161/CIRCHEARTFAILURE.116.003800


  • eng

Conference Location

  • United States