A Concise Guide to Clinical Trials

A Concise Guide to Clinical Trials

Volumn , Issue , 2009, Pages 1-215

  Hackshaw, Allan ^a  

^a IMPERIAL CANCER RESEARCH FUND   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 78049295541     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1002/9781444311723     Document Type: Book

References (164)

1. Laine C, Horton R, DeAngelis CD et al. Clinical Trial Registration: Looking Back and Moving Ahead. Ann Intern Med 2007; 147(4):275-277.
2. World Health Organization. International Clinical Trials Registry Platform. http://www.who.int/ictrp/about/details/en/index.html
3. http://www.jameslindlibrary.org/trialrecords/17th18thCentury/lind/lindtp.html
4. Hróbjartsson A, Gøtzsche PC, Gluud C. The controlled clinical trial turns 100 years: Fibiger's trial of serum treatment of diphtheria. BMJ 1998; 317:1243-1245.
5. Medical Research Council. Streptomycin treatment of pulmonary tuberculosis. BMJ 1948; 2:769-782.
6. Gross PA, Hermogenes H, Sacks HS, Lau J, Levandowski RA. The efficacy of influenza vaccine in elderly persons. Ann Intern Med 1995; 123:518-527.
7. Govaert TME, Thijs CTMCN, Masurel N et al. The efficacy of influenza vaccination in elderly individuals. JAMA 1994; 272(21):1661-1665.
8. EggerM, Schneider M, Davey Smith G. Meta-analysis: spurious precision?Meta-analysis of observational studies. BMJ 1998; 316:140-144.
9. Patriarca PA, Weber JA, Parker RA et al. Efficacy of influenza vaccine in nursing homes. Reduction in illness and complications during an influenza A (H3N2) epidemic. JAMA 1985; 253:1136-1139.
10. Benson K, Hartz AJ. A comparison of observational studies and randomised controlled trials. N Eng J Med 2000; 342:1878-1886.
11. Concato J, Shah N, Horwitz RI. Randomized controlled trials, observational studies, and the hierarchy of research designs. N Eng J Med 2000; 342:1887-1892.
12. Pocock SJ, Elbourne DR. Randomized trials or observational tribulations? N Eng J Med 2000; 342:1907-1909.
13. Collins R, MacMahon S. Reliable assessment of the effects of treatment on mortality and major morbidity, I: clinical trials. The Lancet 2001; 357:373-380.
14. MacMahon S, Collins R. Reliable assessment of the effects of treatment on mortality and major morbidity, II: observational studies. The Lancet 2001; 357:455-462.
15. Sacks H, Chalmers TC, Smith H. Randomized versus historical controls for clinical trials. Am J Med 1982; 72:233-240.
16. Wald NJ, Rodeck CH, Hackshaw AK et al. First and second trimester antenatal screening for Down's syndrome: the results of the Serum, Urine and Ultrasound Screening Study (SURUSS). Health Technology Assessment 2003; 7(11).
17. Moseley JB, O'Malley K, Petersen NJ et al. A Controlled Trial of Arthroscopic Surgery for Osteoarthritis of the Knee. N Eng J Med 2002; 347(2):81-88.
18. Katz R. Biomarkers and surrogate markers: an FDA perspective. NeuroRx: J Am Soc Exp Neuro Therap 2004; 1:189-195.
19. Temple R. Are surrogate markers adequate to assess cardiovascular disease drugs? JAMA 1999; 282(8):790-795.
20. Guidance for industry: clinical trial endpoints for the approval of cancer drugs and biologics. http://www.fda.gov/CbER/gdlns/clintrialend.htm.
21. Kaplan EL, Meier P. Nonparametric estimation from incomplete observations. J Am Stat Assoc 1958; 53:457-481.
22. O'Grady J, Joubert PH (Eds). Handbook of Phase 1 and 2 Clinical Drug Trials. CRC Press Inc., 1997.
23. Griffin JP, O'Grady J (Eds). The textbook of pharmaceutical medicine. BMJ Books, Blackwell Publishing, 5th edn, 2006.
24. Eisenhauer EA, Twelves C, Buyse M. Phase I cancer clinical trials: a practical guide. Oxford University Press, 2006.
25. Eisenhauer EA, O'Dwyer PJ, Christian M, Humphrey JS. Phase I clinical trial design in cancer drug development. J Clin Oncol 2000; 18:684-692.
26. www.fda.gov/cder/guidance/pt1.pdf
27. http://www.fda.gov/cder/guidance/7086fnl.pdf.
28. Wensing G, Haase C, Brendel E, Bottcher MF. Pupillography as a sensitive, non-invasive biomarker in healthy volunteers: first-in-man study of BAY 63-9044, a new 5-HT1areceptor agonist with dopamine agnostic properties. Eur J Clin Pharmacol 2007; 63:1123-1128.
29. Simon R, Wittes RE, Ellenberg SS. Randomized phase II clinical trials. Cancer Treat Rep 1985; 69:1375-1381.
30. Scher HI, Heller G. Picking the winners in a sea of plenty. Clinical Cancer Research 2002; 8:400-404.
31. Steinberg SM, Venzon DJ. Early selection in a randomised phase II clinical trial. Statist Med 2002; 21:1711-1726.
32. Lee JJ, Feng L. Randomized phase II designs in cancer clinical trials: current status and future directions. J Clin Oncol 2005; 23:4450-4457.
33. Rubinstein LV, Korn EL, Friedlin B et al. Design issues of randomised phase II trials and a proposal for phase II screening trials. J Clin Oncol 2005; 23:7199-7206.
34. Wieand HS. Randomized phase II trials: what does randomisation gain? J Clin Oncol 2005; 23:1794-1795.
35. Machin D, Campbell M, Fayers P, Pinol A. Sample size tables for clinical studies. 2nd edn. Blackwell Science, 1997.
36. Tan, SB & Machin, D. Bayesian two-stage designs for phase II clinical trials. Stat Med 2002; 21:1991-2012.
37. A'Hern R. Sample size tables for exact single-stage phase II designs. Stat Med 2001; 20:859-866.
38. Fleming TR. One-sample multiple testing procedure for phase II clinical trials. Biometrics 1982; 38(1): 143-151.
39. Simon R. Optimal two-stage designs for phase II clinical trials. Controlled Clinical Trials 1989; 10:1-10.
40. PASS (Power Analysis and Sample Size software): http://www.ncss.com/pass.html
41. nQuery: http://www.statsol.ie/html/nquery/nqueryhome.html
42. Lee S-M, Buchler T, James L et al. Phase II trial of carboplatin and etoposide with thalidomide in patients with poor prognosis small-cell lung cancer. Lung Cancer 2008; 59(3):364-8.
43. Lee SM, Rudd RM, Woll PJ et al. Two randomised phase III, double blind, placebo controlled trials of thalidomide in patients with advanced non-small cell lung cancer (NSCLC) and small cell lung cancer (SCLC). J Clin Oncol 2008; 26 suppl; abstr 8045.
44. Govaert TME, Thijs CTMCN, Masurel N et al. The efficacy of influenza vaccination in elderly individuals. JAMA 1994; 272(21):1661-1665.
45. Montori VM, Permanyer-Miralda G, Ferreira-Goonzales I et al. Validity of composite end points in clinical trials. BMJ 2005; 330:594-596.
46. Machin D, CampbellM, Fayers P, Pinol A. Sample Size Tables for Clinical Studies, 2nd Edn. Blackwell Science, 1997.
47. Pocock S. Clinical Trials: A Practical Approach. John Wiley & Sons, Ltd, 1983.
48. Julious SA. Tutorial in biostatistics. Sample sizes for clinical trials with Normal data. Stat Med 2004; 23:1921-1986.
49. Jones B, Jarvis P, Lewis JA, Ebbutt AF. Trials to assess equivalence: the importance of rigorous methods. BMJ 1996; 13:36-39.
50. Power and Sample Size calculation. Du Pont WD, Plummer WD. http://medipe.psu.ac.th/episoft/pssamplesize/
51. PASS (Power Analysis and Sample Size software): http://www.ncss.com/pass.html
52. nQuery: http://www.statsol.ie/html/nquery/nqueryhome.html
53. STATA: http://www.stata.com
54. MINITAB: http://www.minitab.com/
55. SAS (Statistical Analysis Software): http://www.sas.com/
56. SPSS (Statistical Package for the Social Sciences): http://www.spss.com
57. Senn SJ. Testing for baseline balance in clinical trials. Stat Med 1994; 13:1715-1726.
58. Averley PA, Girdler NM, Bond S, Steen N, Steele J. A randomised controlled trial of paediatric conscious sedation for dental treatment using intravenous midazolam combined with inhaled nitrous oxide or nitrous oxide/sevoflurane. Anaesthesia, 2004; 59:844-852.
59. Govaert TME, Thijs CTMCN, Masurel N et al. The efficacy of influenza vaccination in elderly individuals. JAMA 1994; 272(21): 1661-1665.
60. Foster GD, Wyatt HR, Hill JO et al. A randomized trial of a low carbohydrate diet for obesity. N Engl J Med 2003; 348:2082-2090.
61. Vickers AJ, Altman DG. Analysing controlled trials with baseline and follow-up measurements. BMJ 2001; 323:1123-1124.
62. Romond EH, Perez EA, Bryant J et al. Trastuzumab plus Adjuvant Chemotherapy for Operable HER2-Positive Breast Cancer. N Eng J Med 2005; 353:1673-1684.
63. Pocock S, Wang D, Wilhelmsen L, Hennekens C. The data monitoring experience in the Candesartan in Heart Failure Assessment of Reduction in Mortality and morbidity (CHARM) program. Am Heart J 2005; 149:939-943.
64. MRC Vitamin Study Research Group. Prevention of neural tube defects: results of the MRC vitamin study. The Lancet 1991; 338:132-137.
65. Lovell K, Cox D, Haddock G et al. Telephone administered cognitive behaviour therapy for treatment of obsessive compulsive disorder: randomised controlled non-inferiority trial. BMJ 2006; 333:883-887.
66. Kerry SM, Bland JM. Analysis of a trial randomised in clusters. BMJ 1998; 316:54.
67. Bland JM, Kerry SM. The intracluster correlation coefficient in cluster randomisation. BMJ 1998; 316:1455-1460.
68. Cuzick J. Forest plots and the interpretation of subgroups. The Lancet 2005; 365:1308.
69. Collins R, MacMahon S. Reliable assessment of the effects of treatment on mortality and major morbidity, I: clinical trials. The Lancet 2001; 357:373-380.
70. Silverstein FE, Faich G, Goldstein JL et al. Gastrointestinal toxicity with Celecoxib vs nonsteroidal anti-inflammatory drugs for osteoarthritis and rheumatoid arthritis. The CLASS Study: a randomised controlled trial. JAMA 2000; 284:1247-1255.
71. O'Brien PC, Fleming TR. A multiple testing procedure for clinical trials. Biometrics 1979; 35:549-556.
72. Pocock SJ. When to stop a clinical trial. BMJ 1992; 305:235-240.
73. Tang JL, Law M, Wald N. How effective is nicotine replacement therapy in helping people to stop smoking? BMJ 1994; 308:21-26.
74. DerSimonian R, Laird N. Meta-analysis in clinical trials. Controlled Clinical Trials 1986; 7:177-188.
75. RevMan Analyses (Computer program). Version 1.0 for Windows. In: Review Manager (RevMan) 4.2. Copenhagen: The Nordic Cochrane Centre, The Cochrane Collaboration, 2003. http://www.cc-ims.net/RevMan
76. Higgins JPT, Thompson SG, Deeks JJ, Altman DG. Measuring inconsistency in metaanalyses. BMJ 2003; 327:557-560.
77. Sterne JAC, EggerM, Davey Smith G. Systematic reviews in health care: Investigating and dealing with publication and other biases in meta-analysis. BMJ 2001; 323:101-105.
78. Moher D, Cook DJ, Eastwood S et al. for the QUORUM Group. Improving the quality of reports of meta-analyses of randomized controlled trials: the QUORUM statement. The Lancet 1999; 354:1896-1900.
79. Mulrow CD. Systematic Reviews: Rationale for systematic reviews. BMJ 1994; 309:597-599.
80. Davey Smith G, Egger M, Phillips AN. Meta-analysis: beyond the grand mean? BMJ 1997; 315:1610-1614.
81. Egger M, Davey Smith G. Meta-analysis: potentials and promise. BMJ 1997; 315:1371-1374.
82. Egger M, Davey Smith G. Meta-analysis: bias in location and selection of studies. BMJ 1998; 316:61-66.
83. Egger M, Davey Smith G, Phillips AN. Meta-analysis: principles and procedures. BMJ 1997; 315:1533-1537.
84. Egger M, Davey Smith G, Schneider M, Minder CE. Bias in meta-analysis detected by a simple graphical test. BMJ 1997; 315: 629-634.
85. Egger M, Schneider M, Davey Smith G. Meta-analysis: spurious precision? Meta-analysis of observational studies. BMJ 1998; 316:140-144.
86. Glasziou P, Irwig L, Bain C, Colditz G. Systematic Reviews in Health Care: A Practical Guide. Cambridge University Press, 2001.
87. EggerM, Davey Smith G, Altman D. (Eds). Systematic Reviews in Health Care: Meta-analysis in Context, 2nd edn. BMJ Books, 2001.
88. Khan KS, Kunz R, Kleijnen J, Antes G. Systematic Reviews to Support Evidence-based Medicine: How to Review and Apply Findings of Healthcare Research. Royal Society of Medicine Press Ltd, 2003.
89. Ware JE, Kosinski M, Turner-Bowker DM, Gandek B. How to score Version 2 of the SF-12 Health Survey. Quality Metric Inc., Lincoln, RI 2002. http://www.qualitymetric.com/
90. The EuroQol Group. EuroQol - a new facility for the measurement of health-related quality of life. Health Policy 1990; 16:199-208. http://www.euroqol.org/
91. Torrance GW, Feeny DH, Furlong W et al. Multi-attribute preference functions for a comprehensive health status classification syste. Health Utilities Index Mark 2. Med Care 1996; 34:702-722.
92. Feeny DH, Furlong W, Torrance GW et al. Multi-attribute preference functions for a comprehensive health status classification syste. Health Utilities Index Mark 3. Med Care 2002; 40:113-128.
93. Aaronson NK, Ahmedzai S, Bergman B et al. The European Organisation for Research and Treatment of Cancer QLQ-C30: A quality-of-life instrument for use in international clinical trials in oncology. J Nat Cancer Inst 1993; 85:365-376. http://www.eortc.be/home/qol/
94. National Institute for Health and Clinical Excellence. http://www.nice.org.uk/
95. Addington-Hall J, Kalra L. Who should measure quality of life? BMJ 2001; 322:1417-1420.
96. Carr AJ, Gibson B, Robinson PG. Is quality of life determined by expectations or experience? BMJ 2001; 322:1240-1243.
97. Carr AJ, Higginson IJ. Are quality of lifemeasures patient centred? BMJ 2001; 322:1357-1360.
98. Farsides B, Dunlop RJ. Is there such a thing as a life not worth living? BMJ 2001; 322:1481-1483.
99. Fayers P, Hays R. Assessing quality of life in clinical trials. 2nd edn. Oxford University Press, 2005.
100. Fayers PM, Machin D. Quality of life: assessment, analysis and interpretation. John Wiley & Sons, Ltd, 2000.
101. Higginson IJ, Carr AJ. Using quality of life measures in the clinical setting. BMJ 2001; 322:1297-1300.
102. Spiegelhalter DJ, Gore SM, Fitzpatrick R et al. Quality of life measures in health care. III: resource allocation. BMJ 1992; 305:1205-1209.
103. Streiner DL, Norman GR. Health measurement scales. 3rd edn. Oxford University Press, 2003.
104. Byford S, Raftery J. Perspectives in economic evaluation. BMJ 1998; 316:1529-1530.
105. Byford S, Torgerson DJ, Raftery J. Cost of illness studies. BMJ 2000; 320:1335.
106. Glick HA, Doshi JA, Sonnad SS, Polsky D. Economic evaluation in clinical trials. Oxford University Press, 2007.
107. Palmer S, Byford S, Raftery J. Types of economic evaluation. BMJ 1999; 318:1349.
108. Palmer S, Torgerson DJ. Definitions of efficiency. BMJ 1999; 318:1136.
109. Raftery J. Economic evaluation: an introduction. BMJ 1998; 316:1013-1014.
110. Raftery J. Costing in economic evaluation. BMJ 2000; 320:1597.
111. Robinson R. Economic evaluation and health care: What does it mean? BMJ 1993; 307:670-673.
112. Robinson R. Economic evaluation and health care: Costs and cost-minimisation analysis. BMJ 1993; 307:726-728.
113. Robinson R. Economic evaluation and health care: Cost-effectiveness analysis. BMJ 1993; 307:793-795.
114. Robinson R. Economic evaluation and health care: Cost-utility analysis. BMJ 1993; 307: 859-862.
115. Robinson R. Economic evaluation and health care: Cost-benefit analysis. BMJ 1993; 307: 924-926.
116. Robinson R. Economic evaluation and health care: The policy context. BMJ 1993; 307:994-996.
117. Torgerson DJ, Byford S. Economic modelling before clinical trials. BMJ 2002; 325:98.
118. Torgerson DJ, Campbell MK. Use of unequal randomisation to aid the economic efficiency of clinical trials. BMJ 2000; 321:759.
119. Torgerson DJ, Campbell MK. Cost effectiveness calculations and sample size. BMJ 2000; 321:1697.
120. Torgerson D, Raftery J. Measuring outcomes in economic evaluations. BMJ 1999; 318:1413.
121. Torgerson DJ, Raftery J. Discounting. BMJ 1999; 319:914-915.
122. http://eudract.emea.europa.eu/
123. http://www.fda.gov/oc/initiatives/advance/fdaaa.html#actions
124. http://www.fda.gov/cder/guidance/2125fnl.htm
125. http://www.ich.org/LOB/media/MEDIA482.pdf
126. Baigent C, Harrell FE, Buyse M, Emberson JR, Altman DG. Ensuring trial validity by data quality assurance and diversification of monitoring methods. Clin Trials 2008; 5:49-55.
127. Damocles Study Group. A proposed charter for clinical trial data monitoring committees: helping them to do their job well. The Lancet 2005; 365:711-722.
128. http://www.mhra.gov.uk/Howweregulate/Medicines/Licensingofmedicines/Clinicaltrials/Safetyreporting-SUSARSandASRs/index.htm
129. Moher D, Schulz KF, Altman DG. The CONSORT Statement: Revised recommendations for improving the quality of reports of parallel-group randomized trials. Ann Intern Med 2001; 134:657-662.
130. CampbellMK, Elbourne DR, Altman DG. CONSORT statement: extension to cluster randomised trials. BMJ 2004; 328(7441):702-708.
131. Piaggio G, Elbourne DR, Altman DG, Pocock SJ, Evans SJW. Reporting of noninferiority and equivalence randomized trials: An extension of the CONSORT statement. JAMA 2006; 295:1152-1160.
132. Ioannidis JP, Evans SJ, Gotzsche PC et al. Better reporting of harms in randomized trials: an extension of the CONSORT statement. Ann Intern Med 2004; 141(10):781-788.
133. http://ohsr.od.nih.gov/guidelines/nuremberg.html
134. http://www.wma.net/e/policy/b3.htm
135. http://www.access.gpo.gov/nara/cfr/waisidx00/45cfr4600.html
136. http://www.ich.org/cache/compo/276-254-1.html
137. http://www.ich.org/LOB/media/MEDIA482.pdf (see page 8).
138. www.ich.org
139. http://www.fda.gov/oc/gcp/default.htm
140. http://www.mhra.gov.uk/Howweregulate/Medicines/Licensingofmedicines/Clinicaltrials/Isaclinicaltrialauthorisationrequired/index.htm
141. http://ec.europa.eu/enterprise/pharmaceuticals/eudralex/vol-1/dir200120/dir200120en.pdf#
142. http://eudravigilance.emea.europa.eu/veterinary/evDbms01.asp
143. http://www.ich.org/LOB/media/MEDIA433.pdf
144. http://www.access.gpo.gov/nara/cfr/cfr-table-search.html#page1
145. http://www.hc-sc.gc.ca/dhp-mps/prodpharma/applic-demande/guideld/clini/indexe.html
146. Griffin JP, O'Grady J (Eds). The Textbook of Pharmaceutical Medicine. 5th edn. BMJ Books, Blackwell Publishing, 2006.
147. http://www.qctn.com.au/ConductingTrials/HowtostartatrialinAustralia/tabid/67/Default.aspx
148. http://www.qctn.com.au/Portals/0/Australian%20Clinical%20Trials-%20Handbook.pdf
149. http://www.icmr.nic.in/
150. http://www.iscr.org/ClinicalTrialsRegulation.aspx
151. Altman D. Practical Statistics for Medical Research. CRC Press, 1990.
152. Altman D, Machin D, Bryant TN, Gardner MJ. StatisticsWith Confidence. 2nd edn. BMJ Books, 2000.
153. Bland JM. An Introduction to Medical Statistics. 3rd edn. Oxford University Press, 2000.
154. Clive C. Handbook of SOPs for Good Clinical Practice. 2nd edn. Interpharm Press Inc., 2004.
155. Ellenbery S, Fleming TR, DeMets DL. Data Monitoring Committees in Clinical Trials: A Practical Perspective (Statistics in Practice). John Wiley & Sons, Ltd, 2002.
156. Friedman L, Furberg CD, DeMets DL. Fundamentals of Clinical Trials. 3rd Rev. edn. Springer-Verlag New York Inc., 2006.
157. Girling D, Parmar M, Stenning S, Stephens R, Stewart, L. Clinical Trials in Cancer: Principles and Practice. Oxford University Press, 2003.
158. Griffin JP, O'Grady J (Eds). The Textbook of Pharmaceutical Medicine. 5th edn. BMJ Books, Blackwell Publishing, 2006.
159. Guyatt G, Rennie D, Meade M, Cook D. Users' Guides to the Medical Literature: A Manual for Evidence-Based Clinical Practice. 2nd edn. McGraw-Hill Medical, 2008.
160. Kirkwood B, Sterne J. Medical Statistics. 2nd edn. Blackwell, 2003.
161. Machin D, Day S, Green S, Everitt B, George S (Eds). Textbook of Clinical Trials. John Wiley & Sons, Ltd, 2004.
162. Petrie A, Sabin C. Medical Statistics at a Glance. 2nd edn. BMJ Books, 2005.
163. Pocock S. Clinical Trials: A Practical Approach. JohnWiley & Sons, Ltd, 1983.
164. Sackett DL, Straus SE, Richardson WS, Rosenberg W, Haynes RB. Evidence-Based Medicine: How to Practice and Teach EBM. 2nd Rev. edn. Churchill Livingstone, 2000.