Clinical Trial Designs for Therapeutic Vaccine Studies

  • Richard Simon
Part of the Cancer Drug Discovery and Development book series (CDD&D)


Cancer vaccines are different in many ways from cytotoxic drugs. Consequently some of the paradigms for the early clinical development of cytotoxics are not applicable to the development of therapeutic vaccines. In contrast, many of the principles of phase III clinical trials are applicable to cancer vaccines. In this chapter we will describe design strategies for the efficient early clinical development of cancer vaccines.


Cancer Vaccine Response Probability Tumor Shrinkage Vaccine Trial Peptide Vaccine 
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  1. 1.
    Salgaller ML, Marincola F, Cormier, JN, Rosenberg, SA. Immunization against epitopes in the human melanoma antigen gp100 following patient immunization with synthetic peptides. Cancer Res 1996; 56:4749–4757.PubMedGoogle Scholar
  2. 2.
    Cormier JN, Salgaller ML, Prevette T, Barracchini KC, Rivoltini L, Restifo NP, Rosenberg SA, Marincola FM. Enhancement of cellular immunity in melanoma patients immunized with a peptide form MART-1/Melan A. Cancer J Sci Am 1997; 3:37–44.PubMedGoogle Scholar
  3. 3.
    Casagrande JT, Pike MC, Smith PG. The power function of the “exact” test for comparing two binomial distributions. Appl Stat 1978; 27:176–180.CrossRefGoogle Scholar
  4. 4.
    Anderson JR, Cain KC, Gelber RD. Analysis of survival by tumor response. J Clin Oncol 1983; 1:710–719.PubMedGoogle Scholar
  5. 5.
    Simon R, Makuch RW. A non-parametric graphical representation of the relationship between survival and the occurrence of an event: application to responder versus non-responder bias. Stat Med 1984; 3:35–44.PubMedCrossRefGoogle Scholar
  6. 6.
    Toni V, Simon R, Russek-Cohen E, Midthune D, Friedman M. Relationship of response and survival in advanced ovarian cancer patients treated with chemotherapy. J Natl Cancer Inst 1992; 84:407–414.CrossRefGoogle Scholar
  7. 7.
    Fleming TR. One sample multiple testing procedure for phase II clinical trials. Biometrics 1982; 38:143.PubMedCrossRefGoogle Scholar
  8. 8.
    Simon R. Optimal two-stage designs for phase II clinical trials. Control Clin Trials 1989; 10:1–10.PubMedCrossRefGoogle Scholar
  9. 9.
    Ensign LG, Gehan EA, Kamen DS, Thall PF. An optimal three-stage design for phase II clinical trials. Stat Med 1994: 13:1727–1736.PubMedCrossRefGoogle Scholar
  10. 10.
    Bendandi M, Gocke CD, Kobrin CB, Benko FA, Sternas LA, Pennington R, et al. Complete molecular remissions induced by patient specific vaccination plus granulocyte-monocyte colony stimulating factor against lymphoma. Nature Med 1999; 5:1171–1177.PubMedCrossRefGoogle Scholar
  11. 11.
    Dixon DO, Simon R. Sample size considerations for studies comparing survival curves using historical controls. J Clin Epidemiol 1988; 41:1209–1213.PubMedCrossRefGoogle Scholar
  12. 12.
    Rubinstein LV, Gail MH, Santner TJ. Planning the duration of a comparative clinical trial with loss to follow-up and a period of continued observation. J Chronic Dis 1981; 34:469–479.PubMedCrossRefGoogle Scholar
  13. 13.
    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. J Clin Oncol 2001; 19:1848–1854.PubMedGoogle Scholar

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© Humana Press Inc. 2004

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  • Richard Simon

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