2009
DOI: 10.1158/1078-0432.ccr-08-2031
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Randomized Phase II Designs

Abstract: As the use of molecularly targeted agents, which are anticipated to increase overall and progression-free survival (OS and PFS), but not necessarily tumor response, has increased in oncology, there has been a corresponding increase in the recommendation and use of randomized phase II designs. Such designs reduce the potential for bias, existent in comparisons with historical controls, but also substantially increase the sample size requirements. We review the principal statistical designs for historically cont… Show more

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Cited by 125 publications
(101 citation statements)
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“…We used what some have referred to as a "selection" or "pick the winner" randomized phase 2 trial design, wherein each arm is regarded as experimental and the efficacy of each arm is evaluated independently and compared with historical data with standard therapy. 27 We sought to determine whether either experimental arm was associated with a CR rate of at least 75%, substantially higher than the approximately 50% CR rate typically observed for standard regimens, including CHOP or R-CHOP therapy in a previous AMC trial (AMC 010) performed in the HAART era. 8 Although this design does not allow a direct efficacy comparison between the 2 arms, it does facilitate a direct comparison of toxicity in the 2 arms.…”
Section: Discussionmentioning
confidence: 99%
“…We used what some have referred to as a "selection" or "pick the winner" randomized phase 2 trial design, wherein each arm is regarded as experimental and the efficacy of each arm is evaluated independently and compared with historical data with standard therapy. 27 We sought to determine whether either experimental arm was associated with a CR rate of at least 75%, substantially higher than the approximately 50% CR rate typically observed for standard regimens, including CHOP or R-CHOP therapy in a previous AMC trial (AMC 010) performed in the HAART era. 8 Although this design does not allow a direct efficacy comparison between the 2 arms, it does facilitate a direct comparison of toxicity in the 2 arms.…”
Section: Discussionmentioning
confidence: 99%
“…Because a usual randomized study, designed to detect a small difference in outcomes between the two arms, would have needed a large number of patients (not compatible with a phase II study nor a rare disease), no formal statistical comparison between the two arms was planned. 19,20 The decision rules were the same for our reference (etoposide-carboplatin) and the new arm (vincristinecarboplatin), and were defined according to the singlestage Fleming design. 21,22 The number of eyes to include in each arm is 33 (17 to 33 patients).…”
Section: Methodsmentioning
confidence: 99%
“…16,17 First, the outcomes associated with patients and the methods used to assess the outcomes will change over time due to factors, such as improved supportive care, efficacy of later-line therapies, earlier disease detection, better equipment and technology, and new versions of response assessment criteria (eg, WHO -Ͼ RECIST 1.0 -Ͼ RECIST 1.1). Second, patient baseline prognostic factors (Eastern Cooperative Oncology Group performance status, previous treatments, tolerance of adverse events, and unrecognized and therefore unaccountable factors) will inevitably fluctuate between trials, so that the patient population in the new trial differs from the historical controls.…”
Section: Discussionmentioning
confidence: 99%