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Activity Number:
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34
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Type:
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Contributed
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Date/Time:
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Sunday, August 3, 2008 : 2:00 PM to 3:50 PM
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Sponsor:
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Biopharmaceutical Section
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| Abstract - #301040 |
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Title:
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Design and Interim Monitoring of a St. Jude Clinical Trial Based on the Gompertz Distribution
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Author(s):
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Arzu Onar*+ and James M. Boyett and Robert P. Sanders and Amar Gajjar
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Companies:
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St. Jude Children's Research Hospital and St. Jude Children's Research Hospital and St. Jude Children's Research Hospital and St. Jude Children's Research Hospital
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Address:
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332 North Lauderdale St, Memphis, TN, 38105,
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Keywords:
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Clinical Trial ; Gompertz Distribution ; Sample size estimation
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Abstract:
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This talk details the statistical issues encountered in a recent single-arm trial at St Jude Children's Research Hospital for young children with embryonic brain tumors. The trial design aimed to estimate EFS distribution, which has historically been characterized by a rapid decline followed by an asymptote, representing patients who achieve long-term disease control. This pattern makes the use of conventional exponential-based designs inappropriate. So instead we used the Gompertz distribution which can accommodate increasing, decreasing and constant hazards. The parameters are easy to estimate and have intuitive interpretations. Further the log-rank type procedures can be used for testing. Sample size calculations were based on estimating the cure rate fraction and a single interim analysis was proposed. The latter however poses some additional challenges which will be detailed.
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