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.
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
Preview
Unable to display preview. Download preview PDF.
References
Friedman LM, Furberg CD, Demets DL (1981). Fundamentals of Clinical Trials. Boston: John Wright, PSG.
Meinert CL (1986) Clinical Trials: Design, Conduct, and Analysis. New York: Oxford University Press.
Pocock SJ (1983). Clinical Trials: A Practical Approach. Chichester: John Wiley & Sons.
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.
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.
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.
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.
Johnson FN, Johnson S (eds.) (1977). Clinical Trials. Oxford: Blackwell Scientific Publishers.
Pocock SJ (1982). Statistical aspects of clinical trial design. Statistician 31:1–18.
Peto R (1978). Clinical trial methodology. Biomedicine (special issue) 28:24–36.
Armitage P (1982). The role of randomization in clinical trials. Stat Med 1:345–352.
Armitage P (1979). The design of clinical trials. Aust J Stat 21:266–281.
Brown BW (1980). Statistical controversies in the design of clinical trials — some personal views. Controlled Clin Trials 1:13–27.
Feinstein AR (1988). Scientific standards in epidemiologic studies of the menace of daily life. Science 242:1257–1263.
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.
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.
Byar DP, Simon RM, Friederwaid WT, et al. (1976). Randomized clinical trials. Prospective on some recent ideas. N Engl J Med 295:445–459.
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.
Uberla KK (1981). Randomized clinical trials: Why not? Controlled Clin Trials 1:295–303.
Kinsey VE (1955). Etiology of retrolental fibroplasia and preliminary report of the Cooperative Study of Retrolental Fibroplasia. Trans Am Ophthalmol Otol 59:15–24.
Kinsey VE (1956). Retrolental fibroplasia. Cooperative study of retrolental fibroplasia. Arch Ophthalmol 56:481–543.
Kinsey VE, et al (1957). PaCO2 levels and retrolental fibroplasia. Report of the cooperative study. Pediatrics 60:655–667.
Silverman WA (1980). Retrolental Fibroplasia: A Modern Parable. New York: Grune & Stratton.
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.
Patz A, et al. (1955). Experimental studies. In Symposium on retrolental fibroplasia. Trans Am Acad Ophthalmol 59:25–34.
Thomas F, et al. (1955). An evaluation of the 1954 Poliomyelitis Vaccine Trials — Summary Report. Am J Pub Health 45:1–63.
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.
Brownlee KA (1955). Statistics of the 1954 polio vaccine trials. J Am Stat Assoc 50:1005–1013.
Schlesselman JJ (1982). Case-Control Studies: Design, Analysis, Conduct. New York: Oxford University Press.
Breslow NE, Day NE (1980). Statistical Methods in Cancer Research: Volume I. Analysis of Case-Control Studies. Lyon, France: IARC Scientific Publications No. 32.
Breslow NE, Day NE (1988). Statistical Methods in Cancer Research: Volume II. Analysis of Cohort Studies. Lyon, France: IARC Scientific Publications No. 82.
Kleinbaum DG, Kupper LL, Morgenstern H (1982). Epidemiologic Research: Principles and Quantitative Methods. Belmount, CA: Wadsworth.
McDowell FH (1977). The Extracranial/Intracranial Bypass Study. Stroke 8:545.
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.
Barnett HJM, McCormick CW (1980). The collaborative study on STA-MCA anastomosis: A progress report. Surg Neurol 13:409–412.
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.
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.
Miller RG (1981). Simultaneous Statistical Inference, 2nd edition. New York: Springer-Verlag.
Tukey JW (1977). Some thoughts on clinical trials, especially problems of multiplicity. Science 19:679–684.
Levy RI, Sondik EJ (1982). Initiating large-scale clinical trials. Controlled Clin Trials 3:29–46.
Gent M (1987). Single studies and overview analyses: Is aspirin of value in cerebral ischemia. Stroke 18:541–544.
Swedish Cooperative Study Group (1987). High dose acetylsalicylic acid after cerebral infarction. Stroke 18:325–334.
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.
Begg C, Berlin JA (1988). Publication bias: A problem in interpreting medical data (with discussions). J R Stat Soc A 151:419–463.
Goldring S, Zervas Z, Langfitt T (1987). The extracranial-intracranial bypass study. N Engl J Med 316:817–820.
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.
Ausman BT, Diaz FG (1986). Critique of the EC-IC bypass study. Surg Neurol 26:218–221.
Day AL, Rhoton AL, Little JR (1986). The EC-IC bypass study. Surg Neurol 26:222–226.
Barnett HJM, Fox A, Hachinski V, et al. (1986). Further conclusions from the Extracranial-Intracranial Bypass Study. Surg Neurol 26:227–235.
Plum F (1986). Extracranial-intracranial arterial bypass and cerebral vascular disease. N EnglJ Med 313:1221–1223.
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.
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.
Caplan LR (1984). Treatment of cerebral ischemia — where are we headed? Stroke 15 (3):571–574.
Levy D (1988). How transient are transient ischemic attacks? Neurology 38:674–677.
Werdelin L, Juhler M (1988). The course of transient ischemic attacks. Neurology 38:677–680.
Caplan LR (1988). TIAs: We need to return to the question, What is wrong with Mr. Jones? Neurology 38:791–793.
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.
Meyer LW, Ellison GW (organizers) (1988). Symposium on Rational for Immunomodula-tion Therapies of Multiple Sclerosis. Neurology 37 (suppl 2):1–89.
McFarlin DE (1983). Treatment of multiple sclerosis. N Engl J Med 308:215–217.
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.
Rose AS, Ellison GW, Myer LW, et al. (1976). Criteria for the diagnosis of multiple sclerosis. Neurology 26:20–22.
McDonald WI, Halliday AM (1977). Diagnosis and classification of multiple sclerosis. Br Med Bull 33:4–9.
Kurtzke JF (1988). Multiple sclerosis: What’s in a name? Neurology 38:309–316.
Kurtzke JF (1955) A new scale for evaluating disability in multiple sclerosis. Neurology 5:580–583.
Tindall R (1988). A closer look at plasmapheresis in multiple sclerosis: The cons. Neurology 38 (suppl):53–56.
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.
Weiner HL, Hafler DA (1988). Immunotherapy of multiple sclerosis. Ann Neurol 23:211–222.
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.
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.
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.
Hoehn NM, Yahr MD (1967). Parkinsonism: onset prognosis and mortality. Neurology 17:427–442.
Kurtzke JF (1983). Rating neurological impairment in multiple sclerosis: An expanded disability status scale (EDSS). Neurology 33:1444–1452.
Teasdale G, Jennett B (1974). Assessment of coma and impaired consciousness, a practical scale. Lancet, 81–83.
Teasdale G, Jennett B (1976). Assessment and prognosis of coma after head injury. Acta Neurochir (Wein) 34:45–55.
Oxbury JM, Greenhall RCD, Grainger KMR (1975). Predicting the outcome of stroke: acute stage after cerebral infarction. Br Med J 3:125–127.
Duvoisin RC (1970). The evaluation of extrapyramidal disease. In Monoamine, Noyaux Gris Centraux et Syndrome de Parkinson. Paris: Masson, pp. 313–325.
Kalbfleisch JD, Prentice RL (1980). The Statistical Analysis of Failure Time Data. New York: John Wiley & Sons.
Cox DR, Oakes D (1894). Analysis of Survival Data. London: Chapman and Hall.
Sackett DL (1986). Rational therapy in the neuroscience: The role of the randomized clinical trial. Stroke 18:1323–1329.
Britton M, Roden A (1985). Progression of stroke after arrival at hospital. Stroke 6:629–633.
Gautier JC (1985). Stroke-in-progression. Stroke 16:729–723
Mayeux R, Stern Y, Spanton S (1985). Heterogeneity in dementia of the Alzheimer type. Neurology 35:453–461.
Zeutsky WJ, Jankovic J, Pirozzolo FJ (1985). The heterogeneity of Parkinson’s disease: Clinical and prognostic implications. Neurology 35:522–526.
Spence JD, Donner A (1982). Problems in design of stroke treatment trials. Stroke 13:94–99.
Yatsu FM (1982). Acute medical therapy of strokes. Stroke 13:524–526.
Scheinberg P (1988). Controversies in the management of cerebral vascular disease. Neurology 38:1609–1616.
Lowenstein DH, Aminoff MJ (1988). The clinical course of spasmodic torticollis. Neurology 38:530–532.
Mitsumoto H, Hanson MR, Chad DA (1988). Amyotrophic lateral sclerosis: Recent advances in pathogenesis and therapeutic trials. Arch Neurol 45:189–202.
Kurtzke JF (1965). Further notes on disability evaluation in multiple sclerosis with scale modifications. Neurology 15:654–661.
Asplund K (1987). Clinometrics in stroke research. Stroke 18:528–530.
Chambers BR, Norris JW, Shurvell BL, et al. (1987). Prognosis for acute stroke. Neurology 37:221–225.
Kurlan R (1988). International symposium on early dopamine agonist therapy of Parkinson’s disease. Arch Neurol 45:204–208.
Mahoney FI, Barthel DW (1965). Functional evaluation: Barthel Index. Md State Med J 14:61–65.
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.
Coté R, Hachinski VC, Shurvell BL, et al. (1986). The Canadian Neurological Scale: A preliminary study in acute stroke. Stroke 17:731–737.
Sterman AB, Furlan AJ, Pessin M, et al. (1987). Acute stroke therapy trials: An introduction to recurring design issues. Stroke 18:524–527.
Adams RJ, Meador KJ, Sethi KD, et al. (1987). Graded neurologic scale for use in acute hemispheric stroke treatment protocols. Stroke 18:665–669.
Weiner HL, Ellison GW (1983). A working protocol to be used as a guideline for trials in multiple sclerosis. Arch Neurol 40:407–410.
Slater RJ (1983). Scoring techniques and problems in the evaluation of change in patients. Arch Neurol 40:675–677.
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.
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.
Willoughby EW, Patty DW (1988). Scales for rating impairment in multiple sclerosis: A critique. Neurology 38:1793–1798.
Schumacher GA (1974). Critique of experimental trials in multiple sclerosis. Neurology 24:1010–1014.
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.
Isaac C, Li DKB, Genton M, et al. (1988). Multiple sclerosis: A serial study using MRI in relapsing patients. Neurology 38:1511–1515.
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.
Marsden CD, Parkes JD (1976). On-off effect in patients with Parkinson’s disease on chronic levadopa therapy. Lancet 1:292–297.
Kurtzinel R, Calne DB (1976). Studies with bromocriptine, Part I. On-off phenomena. Neurology 26:508–510.
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.
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.
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.
Deyo RA (1984). Measuring functional outcome in therapeutic trials of chronic disease. Controlled Clin Trials 5:223–240.
Cox DR, Isham V (1980). Point Processes. London: Chapman and Hall.
Fleiss JL (1986). The Design and Analysis of Clinical Experiments. New York: John Wiley & Sons.
Shapiro SH, Louis TA (eds) (1983). Clinical Trials: Issues and Approaches. New York: Marcel Dekker.
Buyse ME, Staquet MJ, Sylvester RJ (eds) (1984). Cancer Clinical Trials: Methods and Practice. New York: Oxford University Press.
Tygstrup N, Lachin JM, Juhl E (eds) (1982). The Randomized Clinical Trial and Therapeutic Decisions. New York: Marcel Dekker.
Cochran WG, Cox GM (1957). Experimental Design, 2nd edition. New York: John Wiley & Sons.
Kempthorne O (1952). The Design and Analysis of Experiments. New York: John Wiley & Sons.
Brown BW (1980). The crossover experiment for clinical trials. Biometrics 36:69–79.
Hills M, Armitage P (1979). The two-period crossover clinical trial. Br J Clin Pharmacol 8:7–20.
Armitage P, Hills M (1982). The two-period crossover trial. Statistician 31:119–131.
Eisler T, Teravainen H, Nelson R, et al. (1981). Deprenyl in Parkinson’s disease. Neurology 31:19–23.
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.
Diamond S (1976). Treatment of migraine with isomethoptene, acetaminophin and dichloralphenazone combination: A double blind crossover trial. Headache 15:282–287.
Lindegaard KF, Ovrelid L, Sjaastad O (1980). Naproxen in the prevention of migraine attacks. Headache 20:96–98.
Canadian Cooperative Stroke Study Group (1978). A randomized trial of aspirin and sulphinpyrazone in threatened stroke. N Engl J Med 299:53–59.
Pocock SJ (1979). Allocation of patients to treatment in clinical trials. Biometrics 35:183–197.
Simon R (1979). Restricted randomization designs in clinical trials. Biometrics 35:503–512.
Pocock SJ, Simon R (1975). Sequential treatment assignment with balancing for prognostic factors in the controlled clinical trial. Biometrics 31:103–115.
Efron B (1971). Forcing a sequential experiment to be balanced. Biometrika 58:403–417.
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.
Hughes MD, Lee RJ (1987). Statistical problems in reporting clinical trials. N Engl J Med 317:426–432.
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.
DerSimonian R, Charlette LJ, McPeek B, et al. (1982). Reporting on methods in clinical trials. N Engl J Med 306:1332–1336.
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.
Gore SM (1981). Assessing clinical trials — Trial size. Br Med J 282:1687–1689.
Lachin JM (1981). An introduction to sample size determination and power analysis for clinical trials. Controlled Clin Trials 2:93–113.
Donner A (1984). Approaches to sample size estimation in the design of clinical trials. Controlled Clin Trials 3:199–214.
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.
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.
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.
Demets DL (1987) Practical aspects in data monitoring: A brief review. Controlled Clin Trials 6:753–760.
Lee YJ (1983). Interim recruitment goals in clinical trials. J Chron Dis 36:379–389.
Editor information
Editors and Affiliations
Rights 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