Skip to main content

Part of the book series: Foundations of Neurology ((FONY,volume 1))

Abstract

Undertaking clinical trials for the treatment or prevention of neurologic diseases presents interesting problems that provide a challenge to all who participate in this area of research. The biological and physiological processes associated with neurologic diseases have been difficult to study, and only recent advances in neuroscience and medical technology have provided the tools for probing research. For many neurologic diseases, the diagnosis is often tedious to make and may require an extended period of patient observation. Also, the etiology and natural history of neurologic diseases are often obscure. Finally, for those patients with a diagnosed neurologic disease, the clinical course of the condition, including outcome, can be variable and difficult to measure.

This is a preview of subscription content, log in via an institution to check access.

Access this chapter

Chapter
USD 29.95
Price excludes VAT (USA)
  • Available as PDF
  • Read on any device
  • Instant download
  • Own it forever
eBook
USD 259.00
Price excludes VAT (USA)
  • Available as EPUB and PDF
  • Read on any device
  • Instant download
  • Own it forever
Softcover Book
USD 329.99
Price excludes VAT (USA)
  • Compact, lightweight edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info
Hardcover Book
USD 329.99
Price excludes VAT (USA)
  • Durable hardcover edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info

Tax calculation will be finalised at checkout

Purchases are for personal use only

Institutional subscriptions

Preview

Unable to display preview. Download preview PDF.

Unable to display preview. Download preview PDF.

References

  1. Friedman LM, Furberg CD, Demets DL (1981). Fundamentals of Clinical Trials. Boston: John Wright, PSG.

    Google Scholar 

  2. Meinert CL (1986) Clinical Trials: Design, Conduct, and Analysis. New York: Oxford University Press.

    Google Scholar 

  3. Pocock SJ (1983). Clinical Trials: A Practical Approach. Chichester: John Wiley & Sons.

    Google Scholar 

  4. Kahan JP, Neu CR, Hannons GT, et al. (1985). The Decision to Initiate Clinical Trials of Current Medical Practice. Santa Monica, CA: The Rand Corporation.

    Google Scholar 

  5. Sylvester R (1984). Planning cancer clinical trials. In Buyse ME, Staquet MJ, Sylvester RJ (eds.), Cancer Clinical Trials: Methods and Practice. Oxford: Oxford University Press, pp. 47–63.

    Google Scholar 

  6. Staquet MJ, Dalesio O (1984). Design for Phase III trials. In Buyse ME, Staquet MJ, Sylvester RJ (eds.), Cancer Clinical Trials: Methods and Practice. Oxford: Oxford University Press, pp. 261–275.

    Google Scholar 

  7. Mosteller F, Gilbert JP, McPeek B (1983). Controversies in the design and analysis of clinical trials. In Shapiro SH, Louis TA (eds.), Clinical Trials: Issues and Approaches. New York: Marcel Dekker, pp. 13–64.

    Google Scholar 

  8. Johnson FN, Johnson S (eds.) (1977). Clinical Trials. Oxford: Blackwell Scientific Publishers.

    Google Scholar 

  9. Pocock SJ (1982). Statistical aspects of clinical trial design. Statistician 31:1–18.

    Article  Google Scholar 

  10. Peto R (1978). Clinical trial methodology. Biomedicine (special issue) 28:24–36.

    Google Scholar 

  11. Armitage P (1982). The role of randomization in clinical trials. Stat Med 1:345–352.

    Article  PubMed  CAS  Google Scholar 

  12. Armitage P (1979). The design of clinical trials. Aust J Stat 21:266–281.

    Article  Google Scholar 

  13. Brown BW (1980). Statistical controversies in the design of clinical trials — some personal views. Controlled Clin Trials 1:13–27.

    Article  Google Scholar 

  14. Feinstein AR (1988). Scientific standards in epidemiologic studies of the menace of daily life. Science 242:1257–1263.

    Article  PubMed  CAS  Google Scholar 

  15. Peto R, Pike MC, Armitage P, et al. (1977). Design and analysis of randomized clinical trials requiring prolonged observation of each patient. I. Introduction and Design. Br J Cancer 34:585–612.

    Article  Google Scholar 

  16. Peto R, Pike MC, Armitage P, et al. (1977). Design and analysis of randomized clinical trials requiring prolonged observation of each patient. II. Analysis and examples. Br J Cancer 35:1–39.

    Article  PubMed  CAS  Google Scholar 

  17. Byar DP, Simon RM, Friederwaid WT, et al. (1976). Randomized clinical trials. Prospective on some recent ideas. N Engl J Med 295:445–459.

    Article  Google Scholar 

  18. Sackett DL (1983). On some prerequisites for a successful clinical trial. In Shapiro SH, Louis TA (eds.), Clinical Trials: Issues and Approaches. New York: Marcel Dekker, pp. 65–79.

    Google Scholar 

  19. Uberla KK (1981). Randomized clinical trials: Why not? Controlled Clin Trials 1:295–303.

    Article  PubMed  CAS  Google Scholar 

  20. Kinsey VE (1955). Etiology of retrolental fibroplasia and preliminary report of the Cooperative Study of Retrolental Fibroplasia. Trans Am Ophthalmol Otol 59:15–24.

    CAS  Google Scholar 

  21. Kinsey VE (1956). Retrolental fibroplasia. Cooperative study of retrolental fibroplasia. Arch Ophthalmol 56:481–543.

    CAS  Google Scholar 

  22. Kinsey VE, et al (1957). PaCO2 levels and retrolental fibroplasia. Report of the cooperative study. Pediatrics 60:655–667.

    Google Scholar 

  23. Silverman WA (1980). Retrolental Fibroplasia: A Modern Parable. New York: Grune & Stratton.

    Google Scholar 

  24. Patz A, et al. (1952). Studies on the effect of high oxygen administration in retrolental fibroplasia. I. Nursery observations. Am J Ophthalmol 35:1248–1253.

    PubMed  CAS  Google Scholar 

  25. Patz A, et al. (1955). Experimental studies. In Symposium on retrolental fibroplasia. Trans Am Acad Ophthalmol 59:25–34.

    Google Scholar 

  26. Thomas F, et al. (1955). An evaluation of the 1954 Poliomyelitis Vaccine Trials — Summary Report. Am J Pub Health 45:1–63.

    Article  Google Scholar 

  27. Meier P (1972). The biggest health experiment ever. In Tanur JM (ed.), Statistics: A Guide to the Unknown. San Francisco: Holden-Day, pp. 2–14.

    Google Scholar 

  28. Brownlee KA (1955). Statistics of the 1954 polio vaccine trials. J Am Stat Assoc 50:1005–1013.

    Article  Google Scholar 

  29. Schlesselman JJ (1982). Case-Control Studies: Design, Analysis, Conduct. New York: Oxford University Press.

    Google Scholar 

  30. Breslow NE, Day NE (1980). Statistical Methods in Cancer Research: Volume I. Analysis of Case-Control Studies. Lyon, France: IARC Scientific Publications No. 32.

    Google Scholar 

  31. Breslow NE, Day NE (1988). Statistical Methods in Cancer Research: Volume II. Analysis of Cohort Studies. Lyon, France: IARC Scientific Publications No. 82.

    Google Scholar 

  32. Kleinbaum DG, Kupper LL, Morgenstern H (1982). Epidemiologic Research: Principles and Quantitative Methods. Belmount, CA: Wadsworth.

    Google Scholar 

  33. McDowell FH (1977). The Extracranial/Intracranial Bypass Study. Stroke 8:545.

    Article  PubMed  CAS  Google Scholar 

  34. Barnett HJM, Peerless SJ, McCormick CW (1980). In answer to the question: “As compared to what?” A progress report on the EC/IC Bypass Study. Stroke 11:137–140.

    Article  PubMed  CAS  Google Scholar 

  35. Barnett HJM, McCormick CW (1980). The collaborative study on STA-MCA anastomosis: A progress report. Surg Neurol 13:409–412.

    PubMed  CAS  Google Scholar 

  36. The EC/IC Bypass Study Group (1985). The international study of extracranial/intracranial arterial anastomosis (EC/IC Bypass Study): Methodology and entry characteristics. Stroke 16:397–406.

    Article  Google Scholar 

  37. The EC/IC Bypass Study Group (1985). Failure of extracranial/intracranial arterial bypass to reduce the risk of ischemic stroke: Results of an international randomized trial. N Engl J Med 313:1191–1200.

    Article  Google Scholar 

  38. Miller RG (1981). Simultaneous Statistical Inference, 2nd edition. New York: Springer-Verlag.

    Google Scholar 

  39. Tukey JW (1977). Some thoughts on clinical trials, especially problems of multiplicity. Science 19:679–684.

    Article  Google Scholar 

  40. Levy RI, Sondik EJ (1982). Initiating large-scale clinical trials. Controlled Clin Trials 3:29–46.

    Article  PubMed  CAS  Google Scholar 

  41. Gent M (1987). Single studies and overview analyses: Is aspirin of value in cerebral ischemia. Stroke 18:541–544.

    Article  PubMed  CAS  Google Scholar 

  42. Swedish Cooperative Study Group (1987). High dose acetylsalicylic acid after cerebral infarction. Stroke 18:325–334.

    Article  Google Scholar 

  43. Yusef S, Simon R, Ellenberg S (eds.) (1987). Proceedings of the workshop on methodologic issues in overviews of randomized clinical trials, May 1986. Stat Med 6:217–409.

    Google Scholar 

  44. Begg C, Berlin JA (1988). Publication bias: A problem in interpreting medical data (with discussions). J R Stat Soc A 151:419–463.

    Article  Google Scholar 

  45. Goldring S, Zervas Z, Langfitt T (1987). The extracranial-intracranial bypass study. N Engl J Med 316:817–820.

    Article  PubMed  CAS  Google Scholar 

  46. Barnett JHM, Sackett D, Haynes B, et al. (1987). Are the results of the extracranial-intracranial bypass study generalizable? N Engl J Med 316:820–824.

    Article  PubMed  CAS  Google Scholar 

  47. Ausman BT, Diaz FG (1986). Critique of the EC-IC bypass study. Surg Neurol 26:218–221.

    Article  PubMed  CAS  Google Scholar 

  48. Day AL, Rhoton AL, Little JR (1986). The EC-IC bypass study. Surg Neurol 26:222–226.

    Article  PubMed  CAS  Google Scholar 

  49. Barnett HJM, Fox A, Hachinski V, et al. (1986). Further conclusions from the Extracranial-Intracranial Bypass Study. Surg Neurol 26:227–235.

    Article  PubMed  CAS  Google Scholar 

  50. Plum F (1986). Extracranial-intracranial arterial bypass and cerebral vascular disease. N EnglJ Med 313:1221–1223.

    Article  Google Scholar 

  51. Sundt TM (1987). Was the international randomized trial of extracranial-intracranial arterial bypass representative of the population at risk. N EnglJ Med 316:814–816.

    Article  Google Scholar 

  52. Orgogozo JM, Capildeo R (1988). Methodology of clinical trials in stroke. Part II: Future trials — recommendations. In Capildeo R, Orgogozo JM (eds.), Methods in Clinical Trials in Neurology: Vascular and Degenerative Brain Disease. London: The MacMillan Press, pp. 207–221.

    Google Scholar 

  53. Caplan LR (1984). Treatment of cerebral ischemia — where are we headed? Stroke 15 (3):571–574.

    Article  PubMed  CAS  Google Scholar 

  54. Levy D (1988). How transient are transient ischemic attacks? Neurology 38:674–677.

    PubMed  CAS  Google Scholar 

  55. Werdelin L, Juhler M (1988). The course of transient ischemic attacks. Neurology 38:677–680.

    PubMed  CAS  Google Scholar 

  56. Caplan LR (1988). TIAs: We need to return to the question, What is wrong with Mr. Jones? Neurology 38:791–793.

    PubMed  CAS  Google Scholar 

  57. Herndon RM, Murray TJ (organizers) (1983). Proceedings of the International Conference on Therapeutic Trials in Multiple Sclerosis, Grand Island, NY, April 23–24, 1982. Arch Neurol (special issue) 40:663–710.

    Google Scholar 

  58. Meyer LW, Ellison GW (organizers) (1988). Symposium on Rational for Immunomodula-tion Therapies of Multiple Sclerosis. Neurology 37 (suppl 2):1–89.

    Google Scholar 

  59. McFarlin DE (1983). Treatment of multiple sclerosis. N Engl J Med 308:215–217.

    Article  PubMed  CAS  Google Scholar 

  60. Schumacher G, Beebe G, Kibler RF, et al. (1965). Problems of experimental trials of therapy in multiple sclerosis. Ann NY Acad Sci 122:552–568.

    Article  Google Scholar 

  61. Rose AS, Ellison GW, Myer LW, et al. (1976). Criteria for the diagnosis of multiple sclerosis. Neurology 26:20–22.

    PubMed  CAS  Google Scholar 

  62. McDonald WI, Halliday AM (1977). Diagnosis and classification of multiple sclerosis. Br Med Bull 33:4–9.

    PubMed  CAS  Google Scholar 

  63. Kurtzke JF (1988). Multiple sclerosis: What’s in a name? Neurology 38:309–316.

    PubMed  CAS  Google Scholar 

  64. Kurtzke JF (1955) A new scale for evaluating disability in multiple sclerosis. Neurology 5:580–583.

    PubMed  CAS  Google Scholar 

  65. Tindall R (1988). A closer look at plasmapheresis in multiple sclerosis: The cons. Neurology 38 (suppl):53–56.

    PubMed  CAS  Google Scholar 

  66. Bornstein MB, Miller A, Slagle S, et al. (1987). A pilot study of COP 1 in exacerbating-remitting multiple sclerosis. N Engl J Med 317:404–413.

    Article  Google Scholar 

  67. Weiner HL, Hafler DA (1988). Immunotherapy of multiple sclerosis. Ann Neurol 23:211–222.

    Article  PubMed  CAS  Google Scholar 

  68. Kapos L, Patzold U, Dommasch D, et al. (1988). Cyclosporine versus azathioprine in the long-term treatment of multiple sclerosis — results of the German multicenter study. Ann Neurol 23:56–68.

    Article  Google Scholar 

  69. Lataste X, Findley LJ (1988). Methodology of clinical trials in Parkinson’s disease. Part I: Analysis of previous clinical trials. In Capildeo R, Orgogozo JM (eds.), Methods in Clinical Trials in Nurology: Vascular and Degenerative Brain Disease, London: The MacMillan Press, pp. 223–246.

    Google Scholar 

  70. Findley LJ, Lataste X (1988). Methodology of clinical trials in Parkinson’s disease. Part II: Future trials — recommendations. In Capildeo R, Orgogozo JM (eds.), Methods in Clinical Trials in Neurology: Vascular and Degeneratve Brain Disease, London: The MacMillan Press, pp. 247–258.

    Google Scholar 

  71. Hoehn NM, Yahr MD (1967). Parkinsonism: onset prognosis and mortality. Neurology 17:427–442.

    PubMed  CAS  Google Scholar 

  72. Kurtzke JF (1983). Rating neurological impairment in multiple sclerosis: An expanded disability status scale (EDSS). Neurology 33:1444–1452.

    PubMed  CAS  Google Scholar 

  73. Teasdale G, Jennett B (1974). Assessment of coma and impaired consciousness, a practical scale. Lancet, 81–83.

    Google Scholar 

  74. Teasdale G, Jennett B (1976). Assessment and prognosis of coma after head injury. Acta Neurochir (Wein) 34:45–55.

    Article  CAS  Google Scholar 

  75. Oxbury JM, Greenhall RCD, Grainger KMR (1975). Predicting the outcome of stroke: acute stage after cerebral infarction. Br Med J 3:125–127.

    Article  PubMed  CAS  Google Scholar 

  76. Duvoisin RC (1970). The evaluation of extrapyramidal disease. In Monoamine, Noyaux Gris Centraux et Syndrome de Parkinson. Paris: Masson, pp. 313–325.

    Google Scholar 

  77. Kalbfleisch JD, Prentice RL (1980). The Statistical Analysis of Failure Time Data. New York: John Wiley & Sons.

    Google Scholar 

  78. Cox DR, Oakes D (1894). Analysis of Survival Data. London: Chapman and Hall.

    Google Scholar 

  79. Sackett DL (1986). Rational therapy in the neuroscience: The role of the randomized clinical trial. Stroke 18:1323–1329.

    Article  Google Scholar 

  80. Britton M, Roden A (1985). Progression of stroke after arrival at hospital. Stroke 6:629–633.

    Article  Google Scholar 

  81. Gautier JC (1985). Stroke-in-progression. Stroke 16:729–723

    Article  PubMed  CAS  Google Scholar 

  82. Mayeux R, Stern Y, Spanton S (1985). Heterogeneity in dementia of the Alzheimer type. Neurology 35:453–461.

    PubMed  CAS  Google Scholar 

  83. Zeutsky WJ, Jankovic J, Pirozzolo FJ (1985). The heterogeneity of Parkinson’s disease: Clinical and prognostic implications. Neurology 35:522–526.

    Google Scholar 

  84. Spence JD, Donner A (1982). Problems in design of stroke treatment trials. Stroke 13:94–99.

    Article  PubMed  CAS  Google Scholar 

  85. Yatsu FM (1982). Acute medical therapy of strokes. Stroke 13:524–526.

    Article  PubMed  CAS  Google Scholar 

  86. Scheinberg P (1988). Controversies in the management of cerebral vascular disease. Neurology 38:1609–1616.

    PubMed  CAS  Google Scholar 

  87. Lowenstein DH, Aminoff MJ (1988). The clinical course of spasmodic torticollis. Neurology 38:530–532.

    PubMed  CAS  Google Scholar 

  88. Mitsumoto H, Hanson MR, Chad DA (1988). Amyotrophic lateral sclerosis: Recent advances in pathogenesis and therapeutic trials. Arch Neurol 45:189–202.

    PubMed  CAS  Google Scholar 

  89. Kurtzke JF (1965). Further notes on disability evaluation in multiple sclerosis with scale modifications. Neurology 15:654–661.

    PubMed  CAS  Google Scholar 

  90. Asplund K (1987). Clinometrics in stroke research. Stroke 18:528–530.

    Article  PubMed  CAS  Google Scholar 

  91. Chambers BR, Norris JW, Shurvell BL, et al. (1987). Prognosis for acute stroke. Neurology 37:221–225.

    PubMed  CAS  Google Scholar 

  92. Kurlan R (1988). International symposium on early dopamine agonist therapy of Parkinson’s disease. Arch Neurol 45:204–208.

    Google Scholar 

  93. Mahoney FI, Barthel DW (1965). Functional evaluation: Barthel Index. Md State Med J 14:61–65.

    PubMed  CAS  Google Scholar 

  94. Capildeo R, Orgogozo JM (1988). Methodology of clinical trials in stroke. Part I: Analysis of previous clinical trials. In Capildeo R, Orgogozo JM (eds.) Methods in Clinical Trials in Neurology: Vascular and Degenerative Brain Disease. London: The MacMillan Press, pp. 175–205.

    Google Scholar 

  95. Coté R, Hachinski VC, Shurvell BL, et al. (1986). The Canadian Neurological Scale: A preliminary study in acute stroke. Stroke 17:731–737.

    Article  PubMed  Google Scholar 

  96. Sterman AB, Furlan AJ, Pessin M, et al. (1987). Acute stroke therapy trials: An introduction to recurring design issues. Stroke 18:524–527.

    Article  PubMed  CAS  Google Scholar 

  97. Adams RJ, Meador KJ, Sethi KD, et al. (1987). Graded neurologic scale for use in acute hemispheric stroke treatment protocols. Stroke 18:665–669.

    Article  PubMed  CAS  Google Scholar 

  98. Weiner HL, Ellison GW (1983). A working protocol to be used as a guideline for trials in multiple sclerosis. Arch Neurol 40:407–410.

    Google Scholar 

  99. Slater RJ (1983). Scoring techniques and problems in the evaluation of change in patients. Arch Neurol 40:675–677.

    PubMed  CAS  Google Scholar 

  100. Tourtellotte WW, Syndulko K, Baumhefner RW, et al. (1988). A comprehensive protocol for clinical trials in multiple sclerosis which favored azathioprine and corticosteriods as a type of treatment for the chronic progressive phase. Neurology 38 (suppl):83–86.

    PubMed  CAS  Google Scholar 

  101. Paty DW (1988). Trial measures in multiple sclerosis: The use of magnetic resonance imaging in the evaluation of clinical trials. Neurology 38 (suppl):82–83.

    PubMed  CAS  Google Scholar 

  102. Willoughby EW, Patty DW (1988). Scales for rating impairment in multiple sclerosis: A critique. Neurology 38:1793–1798.

    PubMed  CAS  Google Scholar 

  103. Schumacher GA (1974). Critique of experimental trials in multiple sclerosis. Neurology 24:1010–1014.

    PubMed  CAS  Google Scholar 

  104. Poser CM, Paty DW, Scheinberg LC, et al. (1983). New diagnostic criteria for multiple sclerosis: Guidelines for clinical research protocols. Ann Neurol 13:227–231.

    Article  PubMed  CAS  Google Scholar 

  105. Isaac C, Li DKB, Genton M, et al. (1988). Multiple sclerosis: A serial study using MRI in relapsing patients. Neurology 38:1511–1515.

    PubMed  CAS  Google Scholar 

  106. Paty DW, Oger JJF, Kastrukoff LF, et al. (1988). MRI in the diagnosis of MS: A prospective study with comparison of clinical evaluation, evoked potentials, oligoclonal bands and CT. Neurology 38:180–184.

    PubMed  CAS  Google Scholar 

  107. Marsden CD, Parkes JD (1976). On-off effect in patients with Parkinson’s disease on chronic levadopa therapy. Lancet 1:292–297.

    Article  PubMed  CAS  Google Scholar 

  108. Kurtzinel R, Calne DB (1976). Studies with bromocriptine, Part I. On-off phenomena. Neurology 26:508–510.

    Google Scholar 

  109. Pearce JMS, Wallace MG, Capildeo R (1988). Methodology of clinical trials in dementia. Part I: Analysis of previous trials. In Capildeo R, Orgogozo JM (eds.), Methods in Clinical Trials in Neurology: Vascular and Degenerative Brain Disease. London: The MacMillan Press, pp. 265–294.

    Google Scholar 

  110. Andres PL, Finison LJ, Conlon T, et al. (1988). Use of composite scores (megascores) to measure deficit in amyotrophic lateral sclerosis. Neurology 38:405–408.

    PubMed  CAS  Google Scholar 

  111. Olesen J, Tfelt-Hansen P (1988). Methodology of clinical trials in migraine. In Capildeo R, Orgogozo JM (eds.), Methods in Clinical Trials in Neurology: Vascular and Degenerative Brain Disease. London: The MacMillan Press, pp. 85–109.

    Google Scholar 

  112. Deyo RA (1984). Measuring functional outcome in therapeutic trials of chronic disease. Controlled Clin Trials 5:223–240.

    Article  PubMed  CAS  Google Scholar 

  113. Cox DR, Isham V (1980). Point Processes. London: Chapman and Hall.

    Google Scholar 

  114. Fleiss JL (1986). The Design and Analysis of Clinical Experiments. New York: John Wiley & Sons.

    Google Scholar 

  115. Shapiro SH, Louis TA (eds) (1983). Clinical Trials: Issues and Approaches. New York: Marcel Dekker.

    Google Scholar 

  116. Buyse ME, Staquet MJ, Sylvester RJ (eds) (1984). Cancer Clinical Trials: Methods and Practice. New York: Oxford University Press.

    Google Scholar 

  117. Tygstrup N, Lachin JM, Juhl E (eds) (1982). The Randomized Clinical Trial and Therapeutic Decisions. New York: Marcel Dekker.

    Google Scholar 

  118. Cochran WG, Cox GM (1957). Experimental Design, 2nd edition. New York: John Wiley & Sons.

    Google Scholar 

  119. Kempthorne O (1952). The Design and Analysis of Experiments. New York: John Wiley & Sons.

    Google Scholar 

  120. Brown BW (1980). The crossover experiment for clinical trials. Biometrics 36:69–79.

    Article  PubMed  Google Scholar 

  121. Hills M, Armitage P (1979). The two-period crossover clinical trial. Br J Clin Pharmacol 8:7–20.

    PubMed  CAS  Google Scholar 

  122. Armitage P, Hills M (1982). The two-period crossover trial. Statistician 31:119–131.

    Article  Google Scholar 

  123. Eisler T, Teravainen H, Nelson R, et al. (1981). Deprenyl in Parkinson’s disease. Neurology 31:19–23.

    PubMed  CAS  Google Scholar 

  124. LeWitt PA, Gopinathan G, Ward CD, et al. (1982). Lisuride versus bromocriptine treatment in Parkinson’s disease: A double blind study. Neurology 32:69–72.

    PubMed  CAS  Google Scholar 

  125. Diamond S (1976). Treatment of migraine with isomethoptene, acetaminophin and dichloralphenazone combination: A double blind crossover trial. Headache 15:282–287.

    Article  PubMed  CAS  Google Scholar 

  126. Lindegaard KF, Ovrelid L, Sjaastad O (1980). Naproxen in the prevention of migraine attacks. Headache 20:96–98.

    Article  PubMed  CAS  Google Scholar 

  127. Canadian Cooperative Stroke Study Group (1978). A randomized trial of aspirin and sulphinpyrazone in threatened stroke. N Engl J Med 299:53–59.

    Article  Google Scholar 

  128. Pocock SJ (1979). Allocation of patients to treatment in clinical trials. Biometrics 35:183–197.

    Article  PubMed  CAS  Google Scholar 

  129. Simon R (1979). Restricted randomization designs in clinical trials. Biometrics 35:503–512.

    Article  PubMed  CAS  Google Scholar 

  130. Pocock SJ, Simon R (1975). Sequential treatment assignment with balancing for prognostic factors in the controlled clinical trial. Biometrics 31:103–115.

    Article  PubMed  CAS  Google Scholar 

  131. Efron B (1971). Forcing a sequential experiment to be balanced. Biometrika 58:403–417.

    Article  Google Scholar 

  132. Chalmers TC (1983). The control of bias in clinical trials. In Shapiro SH, Louis TA (eds), Clinical Trials: Issues and Approaches. New York: Marcel Dekker, pp. 115–127.

    Google Scholar 

  133. Hughes MD, Lee RJ (1987). Statistical problems in reporting clinical trials. N Engl J Med 317:426–432.

    Article  PubMed  Google Scholar 

  134. Gore SM, Jones IG, Rytter EC (1977). Misuse of statistical methods: Critical assessment of articles in BMJ from January to March 1976. Br Med J 85–87.

    Google Scholar 

  135. DerSimonian R, Charlette LJ, McPeek B, et al. (1982). Reporting on methods in clinical trials. N Engl J Med 306:1332–1336.

    Article  PubMed  CAS  Google Scholar 

  136. Freiman JA, Chalmers TC, Smith H, et al. (1978). The importance of beta, the type II error, and sample size in the design and interpretation of the randomized control trial. N Engl J Med 299:690–694.

    Article  PubMed  CAS  Google Scholar 

  137. Gore SM (1981). Assessing clinical trials — Trial size. Br Med J 282:1687–1689.

    Article  CAS  Google Scholar 

  138. Lachin JM (1981). An introduction to sample size determination and power analysis for clinical trials. Controlled Clin Trials 2:93–113.

    Article  PubMed  CAS  Google Scholar 

  139. Donner A (1984). Approaches to sample size estimation in the design of clinical trials. Controlled Clin Trials 3:199–214.

    CAS  Google Scholar 

  140. George SL (1984). The required size and length of a Phase III clinical trial. In Buyse ME, Staquet MJ, Sylvester RJ (eds.), Cancer Clinical Trials: Methods and Practice. Oxford: Oxford University Press, pp. 287–310.

    Google Scholar 

  141. Rubinstein LV, Gail MH, Santner TJ (1981). Planning the duration of a comparative clinical trial with loss to follow-up and a period of continued observation. J Chron Dis 34:469–479.

    Article  PubMed  CAS  Google Scholar 

  142. Armitage P (1983). Exclusions, losses to follow-up, and withdrawals in clinical trials. In Shapiro SH, Louis TA (eds.), Clinical Trials: Issues and Approaches. New York: Marcel Dekker, pp. 99–114.

    Google Scholar 

  143. Demets DL (1987) Practical aspects in data monitoring: A brief review. Controlled Clin Trials 6:753–760.

    CAS  Google Scholar 

  144. Lee YJ (1983). Interim recruitment goals in clinical trials. J Chron Dis 36:379–389.

    Article  PubMed  CAS  Google Scholar 

Download references

Authors

Editor information

Editors and Affiliations

Rights and permissions

Reprints and permissions

Copyright information

© 1990 Kluwer Academic Publishers

About this chapter

Cite this chapter

Dambrosia, J.M. (1990). Statistical and Epidemiological Considerations. In: Porter, R.J., Schoenberg, B.S. (eds) Controlled Clinical Trials in Neurological Disease. Foundations of Neurology, vol 1. Springer, Boston, MA. https://doi.org/10.1007/978-1-4613-1495-0_3

Download citation

  • DOI: https://doi.org/10.1007/978-1-4613-1495-0_3

  • Publisher Name: Springer, Boston, MA

  • Print ISBN: 978-1-4612-8804-6

  • Online ISBN: 978-1-4613-1495-0

  • eBook Packages: Springer Book Archive

Publish with us

Policies and ethics