Challenges of phase III trial design for novel treatments in diseases with no standard treatment: The AZA-001 myelodysplasia study model

Pierre Fenaux, John F. Seymour, Valeria Santini, Lew Silverman, Steven Gore, Alan List, Guillermo Sanz, Ghulam J. Mufti, Eli Estey, Arlene S. Swern, C. L. Beach, Eva Hellstrom-Lindberg

Research output: Contribution to journalArticle

Abstract

For cancers lacking standard treatments, comparing new agents with existing treatments is problematic. Here we discuss the study design from the AZA-001 trial, which compared azacitidine with 3 frequently used conventional care regimens (CCR) for higher-risk myelodysplastic syndromes. Before randomization, physicians preselected the most appropriate of 3 CCR for each patient, after thorough examination. Patients were then randomized to azacitidine or CCR. Patients randomized to CCR received their preselected treatment, thus including patients otherwise excluded as poor candidates for a single comparator. This design may serve as a template in other cancers lacking standard therapy.

Original languageEnglish (US)
Pages (from-to)258-262
Number of pages5
JournalLeukemia Research
Volume38
Issue number2
DOIs
StatePublished - Feb 2014

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Keywords

  • Azacitidine
  • CCR
  • Heterogenous
  • Novel treatment
  • Standard
  • Study design

ASJC Scopus subject areas

  • Cancer Research
  • Hematology
  • Oncology
  • Medicine(all)

Cite this

Fenaux, P., Seymour, J. F., Santini, V., Silverman, L., Gore, S., List, A., Sanz, G., Mufti, G. J., Estey, E., Swern, A. S., Beach, C. L., & Hellstrom-Lindberg, E. (2014). Challenges of phase III trial design for novel treatments in diseases with no standard treatment: The AZA-001 myelodysplasia study model. Leukemia Research, 38(2), 258-262. https://doi.org/10.1016/j.leukres.2013.10.014