The STAR trial protocol: a randomised multi-stage phase II/III study of Sunitinib comparing temporary cessation with allowing continuation, at the time of maximal radiological response, in the first-line treatment of locally advanced/metastatic Renal Cancer

BMC Cancer

Study Protocol

Chris McCabe¹  Sandy Tubeuf¹  Peter J Selby²  Janet Brown³  DrBarbara Potrata⁴  Jenny Hewison⁵  Lucy McParland⁶  Julia Brown⁶  Helen Howard⁶  Catherine Lowe⁶  Fiona J Collinson⁶  Walter M Gregory⁶  T Wah⁷  Pat Hanlon⁸ 

[1] Academic Unit of Health Economics, Leeds Institute of Health Sciences, Charles Thackrah Building, University of Leeds, 101 Clarendon Road, LS2 9LJ, Leeds, UK;Cancer Research Building, St James’s University Hospital, LS9 7TF, Leeds, UK;Cancer Research UK Experimental Centres at Leeds and Sheffield, LS2 9TF, Leeds, UK;Charles Thackrah Building, Leeds Institute of Health Sciences, University of Leeds, LS2 9JT, Leeds, UK;Charles Thackrah Building, Leeds Institute of Health Sciences, University of Leeds, LS2 9TF, Leeds, UK;Clinical Trials Research Unit, University of Leeds, LS2 9JT, Leeds, UK;Department of Radiology St James’s University Hospital, LS9 7TF, Leeds, UK;Patient Representative National Cancer Research Institute (NCRI) Renal Cancer Clinical Studies Group, Leeds, UK;

关键词: Renal cancer;    Sunitinib;    Intermittent treatment;    Quality of life;    Quality adjusted life years;    Health economics;   

DOI  :  10.1186/1471-2407-12-598

 received in 2012-09-11, accepted in 2012-11-22,  发布年份 2012

来源: Springer

【 摘 要 】

BackgroundOver recent years a number of novel therapies have shown promise in advanced renal cell carcinoma (RCC). Internationally the standard of care of first-line therapy is sunitinib™, after a clear survival benefit was demonstrated over interferon-α. Convention dictates that sunitinib is continued until evidence of disease progression, assuming tolerability, although there is no evidence that this approach is superior to intermittent periods of treatment. The purpose of the STAR trial is to compare the standard treatment strategy (conventional continuation strategy, CCS) with a novel drug free interval strategy (DFIS) which includes planned treatment breaks.Methods/DesignThe STAR trial is an NIHR HTA-funded UK pragmatic randomised phase II/III clinical trial in the first-line treatment of advanced RCC. Participants will be randomised (1:1) to either a sunitinib CCS or a DFIS. The overall aim of the trial is to determine whether a DFIS is non-inferior, in terms of 2-year overall survival (OS) and quality adjusted life years (QALY) (averaged over treatment and follow up), compared to a CCS. The QALY primary endpoint was selected to assess whether any detriment in terms of OS could be balanced with improvements in quality of life (QoL). This is a complex trial with a number of design challenges, and to address these issues a feasibility stage is incorporated into the trial design. Predetermined recruitment (stage A) and efficacy (stage B) intermediary endpoints must be met to allow continuation to the overall phase III trial (stage C). An integral qualitative patient preference and understanding study will occur alongside the feasibility stage to investigate patients’ feelings regarding participation or non-participation in the trial.DiscussionThe optimal duration of continuing sunitinib in advanced RCC is unknown. Novel targeted therapies do not always have the same constraints to treatment duration as standard chemotherapeutic agents and currently there are no randomised data comparing different treatment durations. Incorporating planned treatment breaks has the potential to improve QoL and cost effectiveness, hopefully without significant detriment on OS, as has been demonstrated in other cancer types with other treatments.Trial RegistrationControlled-trials.com ISRCTN 06473203

【 授权许可】

Unknown   
© Collinson et al.; licensee BioMed Central Ltd. 2012. 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.