Clinical trial designs for the early clinical development of therapeutic cancer vaccines

R. M. Simon, S. M. Steinberg, M. Hamilton, A. Hildesheim, S. Khleif, L. W. Kwak, C. L. Mackall, J. Schlom, S. L. Topalian, J. A. Berzofsky

Research output: Contribution to journalReview article

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Abstract

There are major differences between therapeutic tumor vaccines and chemotherapeutic agents that have important implications for the design of early clinical trials. Many vaccines are inherently safe and do not require phase I dose finding trials. Patients with advanced cancers and compromised immune systems are not good candidates for assessing either the toxicity or efficacy of therapeutic cancer vaccines. The rapid pace of development of new vaccine candidates and the variety of possible adjuvants and modifications in method of administration makes it important to use efficient designs for clinical screening and evaluation of vaccine regimens. We review the potential advantages of a wide range of clinical trial designs for the development of tumor vaccines. We address the role of immunological endpoints in early clinical trials of tumor vaccines, investigate the design implications of attempting to use disease stabilization as an end point and discuss the difficulties of reliably utilizing historical control data. Several conclusions for expediting the clinical development of effective cancer vaccines are proposed.

Original languageEnglish (US)
Pages (from-to)1848-1854
Number of pages7
JournalJournal of Clinical Oncology
Volume19
Issue number6
DOIs
StatePublished - Mar 15 2001

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Cancer Vaccines
Clinical Trials
Vaccines
Therapeutics
Immune System
Neoplasms

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

Cite this

Simon, R. M., Steinberg, S. M., Hamilton, M., Hildesheim, A., Khleif, S., Kwak, L. W., ... Berzofsky, J. A. (2001). Clinical trial designs for the early clinical development of therapeutic cancer vaccines. Journal of Clinical Oncology, 19(6), 1848-1854. https://doi.org/10.1200/JCO.2001.19.6.1848

Clinical trial designs for the early clinical development of therapeutic cancer vaccines. / Simon, R. M.; Steinberg, S. M.; Hamilton, M.; Hildesheim, A.; Khleif, S.; Kwak, L. W.; Mackall, C. L.; Schlom, J.; Topalian, S. L.; Berzofsky, J. A.

In: Journal of Clinical Oncology, Vol. 19, No. 6, 15.03.2001, p. 1848-1854.

Research output: Contribution to journalReview article

Simon, RM, Steinberg, SM, Hamilton, M, Hildesheim, A, Khleif, S, Kwak, LW, Mackall, CL, Schlom, J, Topalian, SL & Berzofsky, JA 2001, 'Clinical trial designs for the early clinical development of therapeutic cancer vaccines', Journal of Clinical Oncology, vol. 19, no. 6, pp. 1848-1854. https://doi.org/10.1200/JCO.2001.19.6.1848
Simon RM, Steinberg SM, Hamilton M, Hildesheim A, Khleif S, Kwak LW et al. Clinical trial designs for the early clinical development of therapeutic cancer vaccines. Journal of Clinical Oncology. 2001 Mar 15;19(6):1848-1854. https://doi.org/10.1200/JCO.2001.19.6.1848
Simon, R. M. ; Steinberg, S. M. ; Hamilton, M. ; Hildesheim, A. ; Khleif, S. ; Kwak, L. W. ; Mackall, C. L. ; Schlom, J. ; Topalian, S. L. ; Berzofsky, J. A. / Clinical trial designs for the early clinical development of therapeutic cancer vaccines. In: Journal of Clinical Oncology. 2001 ; Vol. 19, No. 6. pp. 1848-1854.
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