Phase II multi-step planning methods in oncology: Comparison, recommendations and potential applications

Médioni, Jacques; Rycke, Yann De; Facon, Caroline Tournoux; Mallet, Alain; Asselain, Bernard

doi:10.1016/j.cct.2006.08.012

Cited by 3 publications

(3 citation statements)

References 13 publications

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“…Medioni et al [11] also studied the properties of the TT as specified by Whitehead. Their numerical results were consistent with those of Bellissant et al They concluded that for p 0 values lower than 0.20 the test tended to be overpowered, at the expense of a larger than necessary ASN, and that for p 0 values above 0.60 the test had (p. 256) 'very low power, making it impossible to use in these situations, because, as reported before, the most important thing in phase II clinical trials is to control the risk of rejecting treatment.…”

Section: Introductionmentioning

confidence: 99%

An algorithm for the design of group sequential triangular tests for single‐arm clinical trials with a binary endpoint

McWilliams

2010

Statistics in Medicine

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Consider the problem of testing H(0):p ≤ p(0) vs H(1):p > p(0), where p could, for example, represent the response rate to a new drug. The group sequential TT is an efficient alternative to a single-stage test as it can provide a substantial reduction in the expected number of test subjects. Whitehead provides formulas for determining stopping boundaries for this test. Existing research shows that test designs based on these formulas (WTTs) may not meet Type I error and/or power specifications, or may be over-powered at the expense of requiring more test subjects than are necessary. We present a search algorithm, with program available from the author, which provides an alternative approach to triangular test design. The primary advantage of the algorithm is that it generates test designs that consistently meet error specifications. In tests on nearly 1000 example combinations of n (group size), p(0), p(1), α, and β the algorithm-determined triangular test (ATT) design met specified Type I error and power constraints in every case considered, whereas WTT designs met constraints in only 10 cases. Actual Type I error and power values for the ATTs tend to be close to specified values, leading to test designs with favorable average sample number performance. For cases where the WTT designs did meet Type I error and power constraints, the corresponding ATT designs also had the advantage of providing, on average, a modest reduction in average sample numbers calculated at p(0), p(1), and (p(0) + p(1))/2.

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Section: Introductionmentioning

confidence: 99%

An algorithm for the design of group sequential triangular tests for single‐arm clinical trials with a binary endpoint

McWilliams

2010

Statistics in Medicine

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“…This approach permits early study closure of the investigational therapy, conserving patients and avoiding excessive exposure. 12 Two-stage designs are useful when a primary endpoint can be rapidly evaluated (i.e. response at 3 months) such that an interim analysis could feasibly stop the trial if the treatment lacks efficacy or is particularly toxic.…”

Section: Multi-stage Phase 2 Trial Designmentioning

confidence: 99%

“…12 In multistep designs (Fleming or the Triangular test), an interval including the minimum and maximum number of responses for each step is determined. 13,14 The responses analyzed can be either for toxicity endpoints or efficacy endpoints.…”

Section: Multi-stage Phase 2 Trial Designmentioning

confidence: 99%

Are There Circumstances in Which Phase 2 Study Results Should Be Practice-Changing?

Tomblyn

Rizzo

2007

Hematology

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New pharmaceuticals, innovative combinations of approved agents, and novel treatment modalities have resulted in a marked increase in the need for clinical trials. Evidence for treatment efficacy is best derived from large phase 3 randomized, controlled clinical trials. However, phase 3 investigations are lengthy and expensive, and consume patient resources. Furthermore, some diseases and treatment indications are rare, and adequate numbers of patients for a definitive phase 3 trial do not exist. Consequently, it is imperative for clinicians to understand phase 2 trial design, since their interpretation is required to apply the findings in clinical practice appropriately. The complexity of phase 2 studies is explored, including unique designs, possible use of randomization, and other key elements necessary for interpretation of phase 2 trials. Specific examples and application of these concepts are discussed in this review.

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Evaluation of the efficacy and tolerance of a short 7 day third-generation cephalosporin treatment in the management of acute pyelonephritis in young women in the emergency department

Moustafa

Nguyen

Mathevon

et al. 2016

J. Antimicrob. Chemother.

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Phase II multi-step planning methods in oncology: Comparison, recommendations and potential applications

Cited by 3 publications

References 13 publications

An algorithm for the design of group sequential triangular tests for single‐arm clinical trials with a binary endpoint

An algorithm for the design of group sequential triangular tests for single‐arm clinical trials with a binary endpoint

Are There Circumstances in Which Phase 2 Study Results Should Be Practice-Changing?

Evaluation of the efficacy and tolerance of a short 7 day third-generation cephalosporin treatment in the management of acute pyelonephritis in young women in the emergency department

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