Design innovations and baseline findings in a long-term parkinson’s trial: The national institute of neurological disorders and stroke exploratory trials in parkinson’s disease long-term study-1

Movement Disorders

Volumn 27, Issue 12, 2012, Pages 1513-1521

  Elm, Jordan J ^a   Hauser, Robert ^b   Tilley, Barbara C ^c   Kieburtz, Karl ^d   Aminoff, Michael J ^e   Augustine, Erika ^d   Bennett, Susan ^d   Bodis Wollner, Ivan G ^f   Cambi, Franca ^g   Carter, Julie H ^h   Chou, Kelvin ⁱ   Christine, Chadwick W ^e   Dhall, Rohit ^j   Dewey, Richard B ^k   Elble, Rodger J ^l   Fang, John ^m   Feigin, Andrew ⁿ   Galpern, Wendy ^o   Gardiner, Irenita ^d   Harman, Jennifer ^d   Goudreau, John ^p   Juncos, Jorge L ^q   Leehey, Maureen ^r   Kamp, Cornelia ^d   Lew, Mark F ^s   Lin Liang, Grace S ^t   Mari, Zoltan ^u   Martin, Wayne ^v   Nance, Martha ^w   Parashos, Sotirios ^w   Pahwa, Rajesh ^x   Lyons, Kelly E ^x   Petrovitch, Helen ^y   Racette, Brad A ^z   Ravina, Bernard ^aa   Webster Ross, G ^y   Sage, Jacob I ^ab   Shulman, Lisa ^ac   Simon, David K ^ad   Simuni, Tanya ^ae   Singer, Carlos ^af   Slevin, John T ^g   Suchowersky, Oksana ^ag   Tanner, Caroline M ^t   Videnovic, Aleksandar ^ae   Voss, Tiffini S ^ah   Walker, Harrison ^ai   Wills, Anne Marie ^aj   Zweig, Richard ^ak   more..

^a MEDICAL UNIVERSITY OF SOUTH CAROLINA   (United States)

^b UNIVERSITY OF SOUTH FLORIDA   (United States)

^c UNIVERSITY OF TEXAS   (United States)

^d UNIVERSITY OF ROCHESTER   (United States)

^e UNIVERSITY OF CALIFORNIA   (United States)

^f SUNY DOWNSTATE MEDICAL CENTER   (United States)

^g UNIVERSITY OF KENTUCKY   (United States)

^h OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

ⁱ UNIVERSITY OF MICHIGAN   (United States)

^j BARROW NEUROLOGICAL INSTITUTE   (United States)

^k UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

^l SOUTHERN ILLINOIS UNIVERSITY   (United States)

^m VANDERBILT UNIVERSITY MEDICAL CENTER   (United States)

ⁿ North Shore LIJ Health System ^*  (United States)

^o NATIONAL INSTITUTES OF HEALTH   (United States)

^p MICHIGAN STATE UNIVERSITY   (United States)

^q EMORY UNIVERSITY SCHOOL OF MEDICINE   (United States)

^r UNIVERSITY OF COLORADO   (United States)

^s UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

^t PARKINSON S INSTITUTE   (United States)

^u JOHNS HOPKINS UNIVERSITY   (United States)

^v UNIVERSITY OF ALBERTA   (Canada)

^w Struthers Parkinson's Centre   (United States)

^x UNIVERSITY OF KANSAS MEDICAL CENTER   (United States)

^y Pacific Health Research and Education Institute   (United States)

^z WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^aa BIOGEN   (United States)

^ab ROBERT WOOD JOHNSON MEDICAL SCHOOL   (United States)

^ac UNIVERSITY OF MARYLAND SCHOOL OF MEDICINE   (United States)

^ad BETH ISRAEL DEACONESS MEDICAL CENTER   (United States)

^ae NORTHWESTERN UNIVERSITY   (United States)

^af UNIVERSITY OF MIAMI   (United States)

^ag UNIVERSITY OF CALGARY   (Canada)

^ah UNIVERSITY OF VIRGINIA   (United States)

^ai UNIVERSITY OF ALABAMA AT BIRMINGHAM   (United States)

^aj BRIGHAM AND WOMEN S HOSPITAL   (United States)

^ak LOUISIANA STATE UNIVERSITY   (United States)

more..

Author keywords

Clinical trial; Creatine; Global statistical test; Neuroprotection; Parkinson s disease

Indexed keywords

CREATINE; PLACEBO;

ARTICLE; COHORT ANALYSIS; DISEASE COURSE; DOPAMINERGIC TRANSMISSION; HUMAN; PARKINSON DISEASE; PHASE 3 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); SYSTEMATIC REVIEW;

EID: 84867693278     PISSN: 08853185     EISSN: 15318257     Source Type: Journal    
DOI: 10.1002/mds.25175     Document Type: Article

References (52)

1. Ravina BM, Fagan SC, Hart RG, et al. Neuroprotective agents for clinical trials in Parkinson’s disease: a systematic assessment. Neurology 2003;60:1234-1240.
2. NINDS NET-PD Investigators. A randomized, double-blind, futility clinical trial of creatine and minocycline in early Parkinson disease. Neurology 2006;66:664-671.
3. NINDS NET-PD Investigators. A randomized clinical trial of coenzyme Q10 and GPI-1485 in early Parkinson disease. Neurology 2007;68:20-28.
4. NINDS NET-PD Investigators. A pilot clinical trial of creatine and minocycline in early Parkinson disease: 18-month results. Clin Neuropharmacol 2008;31:141-150.
5. Jenner P, Schapira AH, Marsden CD. New insights into the cause of Parkinson’s disease. Neurology 1992;42:2241-2250.
6. Zhang Y, Dawson V, Dawson T. Oxidative stress and genetics in the pathogenesis of Parkinson’s disease. Neurobiol Dis 2000;7:240-250.
7. Marsden C, Olanow C. The causes of Parkinson’s disease are being unraveled and rational neuroprotective therapy is close to reality. Ann Neurol 1998;44:S189-S196.
8. Tarnopolsky MA, Beal MF. Potential for creatine and other therapies targeting cellular energy dysfunction in neurological disorders. Ann.Neurol 2001;49:561-574.
9. Matthews RT, Ferrante RJ, Klivenyi P, et al. Creatine and cyclocreatine attenuate MPTP neurotoxicity. Exp Neurol 1999;157: 142-149.
10. McCall W, Persky AM. Pharmacokinetics of creatine. Subcell Biochem 2007;46:261-273.
11. Williams M, Branch J. Creatine supplementation and exercise performance: an update [see comments]. J Am Coll Nutr 1998;17: 216-234.
12. Gordon A, Hultman E, Kaijser L, et al. Creatine supplementation in chronic heart failure increases skeletal muscle creatine phosphate and muscle performance. Cardiovasc Res 1995;30:413-438.
13. Clark J. Creatine: a review of its nutritional applications in sport. Nutrition 1998;14:322-324.
14. Hauser RA, Auinger P, Oakes D; Parkinson Study Group. Levodopa response in early Parkinson’s disease. Mov Disord 2009;24: 2328-2336.
15. Guimaraes P, Kieburtz K, Goetz CG, et al. Non-linearity of Parkinson’s disease progression: implications for sample size calculations in clinical trials. Clin Trials 2005;2:509-518.
16. Fahn S, Elton RL; the UPDRS Development Committee. Unified Parkinson’s Disease Rating Scale. In Fahn S, Marsden C, Calne D, Goldstein M, eds. Recent Developments in Parkinson’s Disease. Florham Park, NJ: Macmillan Healthcare Information; 1987:153-163.
17. Huang P, Goetz CG, Woolson RF, et al. Using global statistical tests in long-term Parkinson’s disease clinical trials. Mov Disord 2009;24:1732-1739.
18. Huang P, Woolson RF, Granholm A. The use of a global statistical approach for the design and data analysis of clinical trials with multiple primary outcomes. Exp Stroke 2009;1:100-109.
19. Schwab R, England A, Jr. Projection technique for evaluating surgery in Parkinson’s disease. In Gillingham F, Donaldson I, eds. Third Symposium on Parkinson’s Disease. Edinburgh: E. & S. Livingstone; 1969:152-157.
20. Smith A. Symbol Digit Modalities Test Manual. Los Angeles, CA: Western Psychological Services; 2002.
21. Jenkinson C, Fitzpatrick R, Norquist J, Findley L, Hughes K. Cross-cultural evaluation of the Parkinson’s Disease Questionnaire: tests of data quality, score reliability, response rate, and scaling assumptions in the United States, Canada, Japan, Italy, and Spain. J Clin Epidemiol 2003;56:843-847.
22. van Swieten JC, Koudstaal PJ, Visser MC, Schouten HJ, van Gijn J. Interobserver agreement for the assessment of handicap in stroke patients. Stroke 1988;19:604-607.
23. Beck A, Steer R, Ball R, Ranieri W. Comparison of Beck Depression Inventories-IA and-II in psychiatric outpatients. J Pers Assess 1996;67:588-597.
24. Shoulson I, Kurlan R, Rubin A, et al. Assessment of functional capacity in neurodegenerative movement disorders: Huntington’s disease as a prototype. In Munsat T, ed. Quantification of Neurologic Deficit. Boston, MA: Butterworths; 1989:271-283.
25. Marinus J, Visser M, Verwey NA, et al. Assessment of cognition in Parkinson’s disease. Neurology 2003;61:1222-1228.
26. EuroQol Group. EuroQol—a new facility for the measurement of health-related quality of life. Health Policy 1990;16:199-208.
27. O’Brien P. Procedures for comparing samples with multiple endpoints. Biometrics 1984;40:1079-1087.
28. Huang P, Tilley BC, Woolson RF, Lipsitz S. Adjusting O’Brien’s test to control type I error for the generalized nonparametric Behrens-Fisher problem. Biometrics 2005;61:532-539.
29. Luo S, He B, Lawson AB, Elm JJ, Tilley BC. Multivariate longitudinal Bayesian imputation for Global Statistical Test, with applications to Parkinson’s disease. Presentation at the Joint Statistical Meetings (JSM), July 31-August 5, 2010, Vancouver, British Columbia, Canada.
30. Parkinson Study Group. Pramipexole vs levodopa as initial treatment for Parkinson disease: a 4-year randomized controlled trial. Arch Neurol 2004;61:1044-1053.
31. Parkinson Study Group. Impact of deprenyl and tocopherol treatment on Parkinson’s disease in DATATOP patients requiring levodopa. Ann Neurol 1996;39:37-45.
32. Shannon KM, Elm JJ, Goetz CG, Tilley BC. The Modified Rankin Scale as an outcome measure in Parkinson’s disease patients on dopaminergic therapy. American Academy of Neurology 58th Annual Meeting, April 1-April 7, 2006, San Diego, CA, 2006 Scientific Program. 2006;S43.002.
33. Kieburtz K, McDermott M, Como P, et al. The effect of deprenyl and tocopherol on cognitive performance in early untreated Parkinson’s disease. Neurology 1994;44:1756-1759.
34. Sheridan LK, Fitzgerald HE, Adams KM, et al. Normative Symbol Digit Modalities Test performance in a community-based sample. Arch Clin Neuropsychol 2006;21:23-28.
35. Olanow CW, Kieburtz K, Stern M, et al. Double-blind, placebocontrolled study of entacapone in levodopa-treated patients with stable Parkinson disease. Arch Neurol 2004;61:1563-1568.
36. Huang P, Woolson RF, O’Brien PC. A rank-based sample size method for multiple outcomes in clinical trials. Stat Med 2008;27:3084-3104.
37. Friedman L, Furberg C, DeMets D. Fundamentals of Clinical Trials. Littleton, MA: PSG; 1985.
38. Mayeux R, Marder K, Cote LJ, et al. The frequency of idiopathic Parkinson’s disease by age, ethnic group, and sex in northern Manhattan, 1988-1993. Am J Epidemiol 1995;142:820-827.
39. Van Den Eeden SK, Tanner CM, Bernstein AL, et al. Incidence of Parkinson’s disease: variation by age, gender, and race/ethnicity. Am J Epidemiol 2003;157:1015-1022.
40. Morens D, Davis J, Grandinetti A, Ross G, Popper J, White L. Epidemiologic observations on Parkinson’s disease: incidence and mortality in a prospective study of middle-aged men. Neurology 1996;46:1044-1050.
41. Schneider M, Swearingen C, Ye J, Baumgerten M, Tilley BC. Minority enrollment in Parkinson’s disease clinical trials. Parkinsonism Relat Disord 2009;15:258-262.
42. Hauser RA, Holford NH. Quantitative description of loss of clinical benefit following withdrawal of levodopa-carbidopa and bromocriptine in early Parkinson’s disease. Mov Disord 2002;17:961-968.
43. Hauser R, Panisset M, Abbruzzese G, Mancione L, Dronamraju N, Kakarieka A. Double-blind trial of levodopa/carbidopa/entacapone versus levodopa/carbidopa in early Parkinson’s disease. Mov Disord 2009;24:541-550.
44. Parkinson Study Group.A controlled trial of rasagiline in early Parkinson disease: the TEMPO Study. Arch Neurol 2002;59:1937-1943.
45. Fahn S, Oakes D, Shoulson I, et al. Levodopa and the progression of Parkinson’s disease. N Engl J Med 2004;351:2498-2508.
46. Olanow CW, Rascol O, Hauser R, et al. A double-blind, delayedstart trial of rasagiline in Parkinson’s disease. N Engl J Med 2009; 361:1268-1278.
47. Tilley BC, Mainous A, Elm JJ, et al. A randomized recruitment intervention trial in Parkinson’s disease to increase participant diversity: early stopping for lack of efficacy. Clin Trials 2012;9:188-197.
48. Koller WC, Hutton JT, Tolosa E, Capilldeo R. Immediate-release and controlled-release carbidopa/levodopa in PD: a 5-year randomized multicenter study. Carbidopa/Levodopa Study Group. Neurology 1999;53:1012-1019.
49. Lees AJ, Katzenschlager R, Head J, Ben Shlomo Y. Ten-year follow-up of three different initial treatments in de-novo PD: a randomized trial. Neurology 2001;57:1687-1694.
50. Rascol O, Brooks DJ, Korczyn AD, De Deyn PP, Clarke CE, Lang AE. A five-year study of the incidence of dyskinesia in patients with early Parkinson’s disease who were treated with ropinirole or levodopa. 056 Study Group. N Engl J Med 2000;342:1484-1491.
51. Capildeo R. Implications of the 5-year CR FIRST trial. Sinemet CR Five-Year International Response Fluctuation Study. Neurology 1998;50:S15-S17.
52. Tomlinson C, Stowe R, Patel S, Rick C, Gray R, Clarke C. Systematic review of levodopa dose equivalency reporting in Parkinson’s disease. Mov Disord 2010;25:2649-2653.