Design and conduct of phase II studies of targeted anticancer therapy: Recommendations from the task force on methodology for the development of innovative cancer therapies (MDICT)

Booth, Christopher M.; Calvert, A. Hilary; Giaccone, Giuseppe; Lobbezoo, Marinus W.; Eisenhauer, Elizabeth A.; Seymour, Lesley K.; On behalf of the Task Force on Methodology for the Development of Innovative Cancer Therapies,

doi:10.1016/j.ejca.2007.07.031

« Previous Next »European Journal of Cancer
Volume 44, Issue 1 , Pages 25-29, January 2008

Design and conduct of phase II studies of targeted anticancer therapy: Recommendations from the task force on methodology for the development of innovative cancer therapies (MDICT)

Christopher M. Booth
- National Cancer Institute of Canada Clinical Trials Group, Queen’s University, 10 Stuart Street, Kingston, ON, Canada K7L 3N6
- Research Fellow, NCIC Clinical Trials Group.
A. Hilary Calvert
- Northern Institute for Cancer Research, Medical School, Newcastle upon Tyne, UK
Giuseppe Giaccone
- Medical Oncology Branch, National Cancer Institute, Bethesda, USA
Marinus W. Lobbezoo
- NDDO Research Foundation, Amsterdam, The Netherlands
Elizabeth A. Eisenhauer
- National Cancer Institute of Canada Clinical Trials Group, Queen’s University, 10 Stuart Street, Kingston, ON, Canada K7L 3N6
Lesley K. Seymour
- National Cancer Institute of Canada Clinical Trials Group, Queen’s University, 10 Stuart Street, Kingston, ON, Canada K7L 3N6
- Corresponding author: Tel.: +1 613 533 6430; fax: +1 613 533 2941.
On behalf of the Task Force on Methodology for the Development of Innovative Cancer Therapies

Received 26 July 2007; accepted 31 July 2007. published online 10 September 2007.

Abstract

The Methodology for the Development of Innovative Cancer Therapies (MDICT) task force considered aspects of the design and conduct of phase II studies for molecular targeted agents during their 2007 meeting. The task force recommended that multinomial endpoints and designs should be considered for phase II studies of targeted agents, that both single arm as well as randomised designs remain appropriate in certain settings, and that further assessment of novel endpoints (tumour growth kinetic assessment, biomarker or functional imaging) and designs (randomised discontinuation or Bayesian adaptive design) be encouraged. The MDICT cautioned on the use of small randomised trials which have a number of statistical pitfalls and dangers and strongly encouraged the complete reporting, including negative trials, in the scientific literature.

Keywords: Clinical trials/phase II, Drug therapy, Neoplasms, Receptors, Growth factors/antagonists, Inhibitors