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Leuk Res. 2014 Feb;38(2):258-62. doi: 10.1016/j.leukres.2013.10.014. Epub 2013 Oct 27.

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

Author information

1
Hôpital St Louis, Paris 7 University, Paris, France. Electronic address: pierre.fenaux@sls.aphp.fr.
2
Peter MacCallum Cancer Centre, East Melbourne, & University of Melbourne, Australia.
3
University of Florence, Florence, Italy.
4
Mount Sinai School of Medicine, New York, NY, USA.
5
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, MD, USA.
6
H Lee Moffitt Cancer Center and Research Institute, Tampa, FL, USA.
7
Hospital Universitario y Politecnico La Fe, Valencia, Spain.
8
King's College London, London, UK.
9
Division of Hematology, University of Washington School of Medicine, Seattle, WA, USA.
10
Celgene Corporation, Summit, NJ, USA.
11
Karolinska Institute, Department of Hematology, Karolinska University Hospital, Stockholm, Sweden.

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.

KEYWORDS:

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

PMID:
24360612
DOI:
10.1016/j.leukres.2013.10.014
[Indexed for MEDLINE]
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