TY - JOUR
T1 - Reassessing Phase II Heart Failure Clinical Trials
T2 - Consensus Recommendations
AU - Butler, Javed
AU - Hamo, Carine E.
AU - Udelson, James E.
AU - O'Connor, Christopher
AU - Sabbah, Hani N.
AU - Metra, Marco
AU - Shah, Sanjiv J.
AU - Kitzman, Dalane W.
AU - Teerlink, John R.
AU - Bernstein, Harold S.
AU - Brooks, Gabriel
AU - Depre, Christophe
AU - Desouza, Mary M.
AU - Dinh, Wilfried
AU - Donovan, Mark
AU - Frische-Danielson, Regina
AU - Frost, Robert J.
AU - Garza, Dahlia
AU - Gohring, Udo Michael
AU - Hellawell, Jennifer
AU - Hsia, Judith
AU - Ishihara, Shiro
AU - Kay-Mugford, Patricia
AU - Koglin, Joerg
AU - Kozinn, Marc
AU - Larson, Christopher J.
AU - Mayo, Martha
AU - Gan, Li Ming
AU - Mugnier, Pierrre
AU - Mushonga, Sekayi
AU - Roessig, Lothar
AU - Russo, Cesare
AU - Salsali, Afshin
AU - Satler, Carol
AU - Shi, Victor
AU - Ticho, Barry
AU - Van Der Laan, Michael
AU - Yancy, Clyde
AU - Stockbridge, Norman
AU - Gheorghiade, Mihai
N1 - Publisher Copyright:
© 2003 American Heart Association.
PY - 2017/4/1
Y1 - 2017/4/1
N2 - 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.
AB - 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.
KW - United States Food and Drug Administration
KW - clinical trial
KW - heart failure
KW - mortality
KW - safety
UR - http://www.scopus.com/inward/record.url?scp=85017581011&partnerID=8YFLogxK
U2 - 10.1161/CIRCHEARTFAILURE.116.003800
DO - 10.1161/CIRCHEARTFAILURE.116.003800
M3 - Review article
C2 - 28356300
AN - SCOPUS:85017581011
SN - 1941-3289
VL - 10
JO - Circulation: Heart Failure
JF - Circulation: Heart Failure
IS - 4
M1 - e003800
ER -