ST 520 Statistical Principles of Clinical Trials - NCSU Statistics ...

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CHAPTER 5 ST 520, A. TSIATIS and D. Zhang jects. It contains the study background, experimental design, patient population, treatment and evaluation details, and data collection procedures. Purposes (1) To assist investigators in thinking through the research (2) To ensure that both patient and study management are considered at the planning stage (3) To provide a sounding board for external comments (4) To orient the staff for preparation of forms and processing procedures (5) To provide a document which can be used by other investigators who wish to confirm (replicate) the results I will hand out two protocols in class. One is from the Womens Health Initiative (WHI) and one from the Cancer and Leukemia Group B (CALGB) study 8541. In the WHI study, the clinical trial will evaluate the benefits and risks of Hormone Replacement Therapy (HRT), Dietary Modification, DM, and supplementation with calcium/vitamin D (CaD) on the overall health of postmenopausal women. Health will be assessed on the basis of quality of life measurements, cause-specific morbidity and mortality, and total mortality. CALGB 8541 is a study of different regimen of adjuvant CAF (combination of Cyclophosphamide, Adriamycin and 5 Fluorouracil (5-FU)) as treatment for women with pathological stage II, node positive breast cancer. Specifically, intensive CAF for four cycles versus low dose CAF for four cycles versus standard dose CAF for six cycles will be compared in a three arm randomized clinical trial. Protocols generally have the following elements: 1. Schema. Depicts the essentials of a study design. WHI: page 18 CALGB 8541: page 1 PAGE 78
CHAPTER 5 ST 520, A. TSIATIS and D. Zhang 2. Objectives The objectives should be few in number and should be based on specific quantifiable endpoints WHI: pages 14-15 and pages 22-24 CALGB 8541: page 3 3. Project background This section should give the referenced medical/historical background for therapy of these patients. WHI: pages 2-13 CALGB 8541: pages 1-3 This generally includes – standard therapy – predecessor studies (phase I and II if appropriate) – previous or concurrent studies of a similar nature – moral justification of the study 4. Patient Selection A clear definition of the patient population to be studied. This should include clear, unambiguous inclusion and exclusion criteria that are verifiable at the time of patient entry. Each item listed should be verified on the study forms. WHI: pages 24-28 CALGB 8541: pages 4-5 5. Randomization/Registration Procedures This section spells out the mechanics of entering a patient into the study WHI: pages 29-38 CALGB 8541: pages 5-7 6. Treatment Administration and Patient Management How the treatment is to be administered needs to be specified in detail. All practical eventualities should be taken into account, at least, as much as possible. Protocols should not be written with only the study participants in mind. Others may want to replicate this therapy such as community hospitals that were not able to participate in the original study. WHI: pages 18-22 and 44-49 CALGB 8541: pages 7-20 PAGE 79
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ST 520 Statistical Principles of Cl
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CHAPTER 1 ST 520, A. TSIATIS and D.
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ST 520 Statistical Principles of Clinical Trials - NCSU Statistics ...

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