Skip to main content

Advertisement

Flexible trial design in a rare condition

Article metrics

  • 589 Accesses

Running clinical trials in relapsed Ewing sarcoma (ES) is challenging due to its rarity. However randomised clinical trials are essential to evaluate the effectiveness of treatments reliably. Four chemotherapy regimens are currently most often used within Europe for relapsed disease based on small single-arm series with different eligibility criteria, short follow up and variable definitions of outcome measures. There are no comparative data on the relative efficacy, toxicity or acceptability of these regimens. As a result, the evidence base for treatment at relapse is weak and there is widespread variability in chemotherapy delivery and a lack of agreement on which chemotherapy backbone to use as a standard comparator arm for trials of novel agents. We propose a design of a multi-arm multi-stage randomised multicentre international trial to evaluate the efficacy, toxicity and acceptability of these four chemotherapy combinations. Since there is no standard of care in this setting, no standard arm can be defined and the trial will employ a drop-a-loser approach. Initially there will be a 4-way randomisation, with one arm to be dropped after the first stage and a second after the second stage (both based on response rate), with the remaining two arms progressing to a Phase III evaluation based on progression-free survival. We will present the design characteristics and planned analysis methods of this trial in this rare disease.

Author information

Correspondence to Veronica Moroz.

Rights and permissions

This article is published under license to BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

Reprints and Permissions

About this article

Cite this article

Moroz, V., Wheatley, K. & McCabe, M. Flexible trial design in a rare condition. Trials 14, P24 (2013) doi:10.1186/1745-6215-14-S1-P24

Download citation

Keywords

  • Chemotherapy Regimen
  • Rare Disease
  • Chemotherapy Combination
  • Rare Condition
  • Plan Analysis

Comments

By submitting a comment you agree to abide by our Terms and Community Guidelines. If you find something abusive or that does not comply with our terms or guidelines please flag it as inappropriate. Please note that comments may be removed without notice if they are flagged by another user or do not comply with our community guidelines.