8 PART III: Further Topics CHAPTER 18: Genomic Targets 18.1 Introduction 18.2 Predictive markers 18.3 Enrichment design 18.4 Biomarker‐Stratified Designs 18.5 Adaptive threshold designs CHAPTER 19: Feasibility and Pilot Studies 19.1 Introduction 19.2 Feasibility studies 19.3 External‐pilot studies 19.4 Considerations across external‐pilot and main trial 19.5 Internal‐pilot studies 19.6 Other preliminary studies 19.7 Reporting CHAPTER 20: Further Topics 20.1 Introduction 20.2 Adaptive approaches 20.3 Large simple trials 20.4 Bayesian methods 20.5 Interim analyses 20.6 Zelen randomised consent designs 20.7 Systematic overviews
10 Glossary
11 References
12 Index
List of Tables
1 Chapter 3Table 2 Operational mattress definitionsTable 4 Minimisation factors
2 AppendixTable T1 Random numbersTable T2 The Normal distribution function – Probability,γ, that a Normally ...Table T3 Percentage points of the standardised Normal distribution for givenα...Table T4 The value tabulated ist1 − α/2, such that if X i...Table T5 Simon, Wittes and Ellenberg designTable T6 The multiplierκ of the estimated standard deviation, sPilot, to ob...Table T7 Total sample size for a two equal group Main trial for standardised dif...Table T8 Optimal values of Pilot, Main and hence Overall two equal‐group compara...
List of Illustrations
1 Chapter 1Figure 1.1 Individual patient reductions in disease activity (SASSAD) for th...Figure 1.2 The relative strength of evidence obtained from alternative desig...Figure 1.3 Special considerations for clinical trials in human subjectsFigure 1.4 Avicenna’s rules for the experimental use and testing of drugsFigure 1.5 Sequential phases of developing randomised controlled trials of c...
2 Chapter 2Figure 2.1 Individually randomised controlled trial of the effect of topical...
3 Chapter 3Figure 3.1 Major components of a clinical trial protocolFigure 3.2 Part of the Information Sheet utilised in the multicentre COMPLIA...Figure 3.3 Consent form designed to obtain assent from a patient to be rando...
4 Chapter 4Figure 4.1 Selected baseline, adverse event and endpoint results from a tria...Figure 4.2 Skin assessment scale used for the grading of pressure sores.Figure 4.3 Batch baseline mean and SD of estrone levels, and corresponding ZFigure 4.4 Registration form.Figure 4.5 Surgical complications form.Figure 4.6 Follow‐up form.Figure 4.7 Part of the Questionnaire (SVQ) for self‐assessment of sexual fun...Figure 4.8 General health question from SF‐36v2.
5 Chapter 5Figure 5.1 All possible permutations of block size 4 for two treatments A an...Figure 5.2 Randomised block design (RBD) for 24 units comprising two interve...Figure 5.3 Randomised block design (RBD) for 18 patients comprising three in...Figure 5.4 All possible permutations of block size 5 for two interventions SFigure 5.5 Stratification groups for a trial for alleviating urinary symptom...Figure 5.6 Distribution of patients with prostate cancer according to TURP a...Figure 5.7 Advantages and disadvantages of different types of randomisation...Figure 5.8 Key steps in preparing the randomisation list for a parallel grou...
6 Chapter 6Figure 6.1 Schematic representation of some stages involved when planning a ...Figure 6.2 Suggested details necessary for trial registration purposes.Figure 6.3 Aspects of the progress of a trial that require internal monitori...
7 Chapter 7Figure 7.1 Problems associated with missing case record forms and data items...
8 Chapter