Clinical Trials

Clinical Trials: A Practical Guide ■❚❙❘journal guidelines, 399–400layout, 401negative values, 403numeric data, 402–403outcomes, 401primary trial endpoints, 394P-value presentation, 403references, 402reported numbers, 402rounding, 402size, 400standard table, 393, 393–394timing in a trial, 398–399titles, 401totals, 401two-dimensional comparisons, 394–396units, 402TARGET (Do Tirofiban and ReoPro GiveSimilar Efficacy Outcomes Trial), 1163 variable graphs, 423, 424timeline, multicenter trials, 157time to maximum, repeated measurements,322, 323T max, bioequivalence trials, 122treatment-by-center, multicenter trials,313, 313–314treatment interaction, factorial design, 105–106treatments, protocol development, 31trial committees, protocol developmentsee protocol developmenttrial design, 3trial monitoring, protocol development, 33trial profile, 377–384components, 378–379, 379definition, 378examples, 380–383medical journal practice, 383, 383–384purpose, 379–3802 × 2 factorial design, 381, 381–382two-way crossover design, 382, 382–383two-way parallel design, 381trial termination, early, 360–361triple-blinded studies, 78–79t-testsone-sample, 200–204, 214critical values, 201–202, 203degrees of freedom, 200–201example, 202–204paired, 204–207, 205, 206, 214result reporting, 213standard distribution vs, 201two-sample, 207–208, 214assumptions, 209example, 208–209two-sample Wilcoxon rank-sum (Mann-Whitney)test, 210–212, 212, 214two-sample Z-test, 210, 214, 225–226type I (alpha) errors, 268significance tests, 190–191, 192type II (beta) errors, 84significance tests, 190–191, 192types, of clinical trials, 2see also specific typesUunbalanced factorial design, 109unblinding studies, 79uncontrolled trials, 15–21advantages, 18–19, 20case series studies, 18case studies, 18limitations, 19–20, 20Phase I trials, 16–17Phase II trials, 17–18rationale for use, 16–18univariate analysis, definition, 455US Food and Drug Administration (FDA)Regulations Relating to Good Clinical Practiceand Clinical Trials, 24Vvariablescategorical, 172definitions, 168–169dependent, 169independent, 169summaries of, 170–174, 171, 173, 174see also specific presentationsvitamin A supplementation trial, 260–261analysis, 261trial profile, 260Wwashout period, definition, 52Withdrawal of Inhaled Steroids Evaluation Studyin Patients with Cystic Fibrosis (CF-WISE), 71within-subject comparisons, crossover clinicaltrials, 95479