Clinical Trials Handbook

A systematic approach to all aspects of designing and
conducting clinical trials

The success or failure of clinical trials hinges on hundreds of
details that need to be developed, often under less than ideal
conditions. Written by one of the world's leading trialists,
Clinical Trials Handbook: Design and Conduct provides
clinicians with a complete guide to designing, conducting, and
evaluating clinical trials--teaching them how to simplify the
process and avoid costly mistakes.

The author draws on his extensive clinical trials experience to
outline all steps employed in setting up and running clinical
trials, from budgeting and fundraising to publishing the results.
Along the way, practical advice is offered while also addressing a
mix of logistical, ethical, psychological, behavioral, and
administrative issues inherent to clinical trials. Topics of
coverage include:

• Protocols for drug masking, controls, and treatment
  randomization

• Consent, enrollment, eligibility, and follow-up procedures

• Different types of sample size design and data collection and
  processing

• Working with study centers, research staff, and various
  committees

• Monitoring treatment effects and performance, and ensuring
  quality control

• Data analysis and access policies for study data and
  documents

Clinical Trials Handbook is invaluable for practicing
clinicians and trialists who would like to learn more about or
improve their understanding of the design and execution of clinical
trials. The book is also an excellent supplement for courses on
clinical trials at the graduate level.

Autorentext

CURTIS L. MEINERT, PhD, is Professor in the Departments of Epidemiology and Biostatistics at the Johns Hopkins Bloomberg School of Public Health, where he served as founder of the Johns Hopkins Center for Clinical Trials. He is a Fellow of the American College of Epidemiology, the American Association for the Advancement of Science, the American Heart Association, and the Society for Clinical Trials. Dr. Meinert has focused his research on the methodological issues in clinical trials, including their design, conduct, policy, and practice. He is the author of Clinical Trials Dictionary: Terminology and Usage Recommendations, Second Edition (Wiley).

Zusammenfassung

A systematic approach to all aspects of designing and conducting clinical trials

The success or failure of clinical trials hinges on hundreds of details that need to be developed, often under less than ideal conditions. Written by one of the world's leading trialists, Clinical Trials Handbook: Design and Conduct provides clinicians with a complete guide to designing, conducting, and evaluating clinical trialsteaching them how to simplify the process and avoid costly mistakes.

The author draws on his extensive clinical trials experience to outline all steps employed in setting up and running clinical trials, from budgeting and fundraising to publishing the results. Along the way, practical advice is offered while also addressing a mix of logistical, ethical, psychological, behavioral, and administrative issues inherent to clinical trials. Topics of coverage include:

• Protocols for drug masking, controls, and treatment randomization
• Consent, enrollment, eligibility, and follow-up procedures
• Different types of sample size design and data collection and processing
• Working with study centers, research staff, and various committees
• Monitoring treatment effects and performance, and ensuring quality control
• Data analysis and access policies for study data and documents
  Clinical Trials Handbook is invaluable for practicing clinicians and trialists who would like to learn more about or improve their understanding of the design and execution of clinical trials. The book is also an excellent supplement for courses on clinical trials at the graduate level.

Inhalt

Acknowledgments xi

Preface xiii

On planning xv

Explanatory notes, focus, and conventions xvii

Abbreviations and designations xxi

I. General1

1. Terminology 3

2. Definitions 5

3. Measurement units 7

4. Trial type 9

5. Design and flow schematics 13

6. Design and operating principles 15

7. Counting and analysis rules 17

8. Multi-study umbrella name 19

9. Study name 21

II. Design Specifications25

10. Objective 27

11. Specific aims 29

12. Experimental variable 31

13. Treatment unit 33

14. Primary outcome 35

15. Outcome measures 39

16. Design synopsis 41

III. Funding45

17. Type of funding initiative 47

18. Funding: Specifications 49

19. Funding: Terminology 51

20. Funding: Type 59

21. Funding: Initiative 61

22. Funding: Period 63

23. Funding: Budget 65

24. Funding: Mode 67

IV. Treatment Groups/Treatment Administration69

25. Study groups 71

26. Comparison group 73

27. Study treatments 75

28. Test treatments 77

29. Control/comparison treatment 79

30. Placebo treatment 87

31. Sham treatment 91

32. Treatment modality 93

33. Treatment schedule 95

34. Treatment compliance measures 97

35. Protocol overrides 101

36. Protocol bailouts 103

V. Masking 105

37. Mask/masking: Definitions 107

38. Masking principles 111

39. Masking, censoring, and shielding specifications 113

40. Drug masking procedure 115

41. Drug packaging and labeling 117

42. Drug supply 121

43. Masking safeguards 123

44. Unmasking treatment assignment 125

45. Results blackouts 127

VI. Bias and Variance Control129

46. Bias control procedures 131

47. Stratification 135

48. Variance control procedures 139

49. Separations 141

VII. Treatment Assignment/Randomization 143

50. Assignment methods: Fixed vs. adaptive 145

51. Treatment assignment: Random vs. nonrandom 147

52. Randomization: Complete vs. restricted 151

53. Randomization unit 155

54. Randomization: Procedures 157

VIII. IRBs and Consents159

55. IRBs 161

56. IRBs: Models and procedures 163

57. Consent 169

58. Consent: Checklist 177

59. Consent: Disclaimers and notifications 181

60. Consent: Principles and purpose 183

61. Consent: Process 185

62. Consent: Types 189

63. Consent: Questions and answers 191

IX. Enrollment and Followup195

64. Notation 197

65. Timing conventions 199

66. Required approvals, permissions, accesses, and supplies 201

67. Start-up design 203

68. Start-up checklist 207

69. Recruitment design 209

70. Enrollment goals 211

71. Enrollment quotas 213

72. Followup: Terminology 217

73. Followup: Method 221

74. Followup: Length 223

75. Closeout design 225

76. Missed visit 229

77. Dropout 231

78. Loss to followup 235

79. Study timetable 239

80. Critical event path analysis 241

81. Eligibility criteria 243

82. Exclusions from enrollment 245

83. Eligibility and exclusions by reason 249

X. Sample Size251

84. Sample size: Desi...