Considerations for using positive controls in phase 2 clinical trials of central nervous system disorders

Drug Information Journal

Volumn 44, Issue 4, 2010, Pages 431-441

  Mallinckrodt, Craig H ^a   Detke, Michael J ^b,c,d,e   Prucka, William R ^a   Ruberg, Stephen J ^a   Molenberghs, Geert ^f,g  

^a ELI LILLY AND COMPANY   (United States)

^b MedAvante Inc   (United States)

^c HARVARD MEDICAL SCHOOL   (United States)

^d MCLEAN HOSPITAL   (United States)

^e INDIANA UNIVERSITY SCHOOL OF MEDICINE   (United States)

^f HASSELT UNIVERSITY   (Belgium)

^g UNIVERSITY OF LEUVEN   (Belgium)

Author keywords

Clinical trial; Positive controls; Power

Indexed keywords

DULOXETINE; PLACEBO;

ARTICLE; CENTRAL NERVOUS SYSTEM DISEASE; CLINICAL TRIAL; DRUG APPROVAL; DRUG EFFICACY; HUMAN; MAJOR DEPRESSION; MEDICAL DECISION MAKING; OUTCOME ASSESSMENT; PRIORITY JOURNAL; SENSITIVITY ANALYSIS; TREATMENT OUTCOME;

EID: 77954905504     PISSN: 00928615     EISSN: None     Source Type: Journal    
DOI: 10.1177/009286151004400407     Document Type: Article

References (32)

1. Hurko O, Ryan JL. Translational research in central nervous system drug discovery. NeuroRx. 2005;2:671-682.
2. Kola I, Landis J. Can the pharmaceutical industry reduce attrition rates? Nat Rev Drug Discov. 2004;3:711-715.
3. Kemp AS, Schooler NR, Kalali AH, et al. What is causing the reduced drug-placebo difference in recent schizophrenia clinical trials and what can be done about it? Schizophr Bull. Epub ahead of print, August 22, 2008. doi:10.1093/schbul/sbn110.
4. Temple R, Ellenberg SS. Placebo-controlled trials and active-control trials in the evaluation of new treatments. Part 1: ethical and scientific issues. Ann Intern Med. 2000;133:455-463.
5. Tsong Y, Zhang JJ. Testing superiority and non-inferiority hypotheses in active controlled active trials. Biom J. 2005;47(1):62-74.
6. Hung HMJ, Wang SJ, Tsong Y, Lawrence J, O'Neil RT. Some fundamental issues with non-inferiority testing in active controlled trials. Stat Med. 2003;22(2):213-225.
7. International Conference on Harmonization. ICH Efficacy Document E-9. Statistical principles of clinical trials. http://www.fda.gov/cder/guidance.
8. DerSimonian R, Laird N. Meta-analysis in clinical trials. Control Clin Trials. 1986;7(3):177-188.
9. Sankoh AJ, Huque MF. Impact of multiple endpoints on type I error rate and power of test statistic in non-superiority clinical trials. Far East J Theor Stat. 2004;13(1):47-65.
10. Sankoh AJ, Al-Osh M, Huque FM. On the utility of the Dirichlet distribution for meta-analysis of clinical studies. J Biopharm Stat. 1999;9(2):289-306.
11. Rohmel J. Therapeutic equivalence investigations: statistical consideration. Stat Med. 1998;17(15-16):1703-1714.
12. Holmgren EB. Establishing equivalence by showing that a specified percentage of the effect of the active control over placebo is maintained. J Biopharm Stat. 1999;9:651-659.
13. Khan A, Kolts RL, Thase ME, Krishnan KR, Brown W. Research design features and patient characteristics associated with the outcome of antidepressant clinical trials. Am J Psychiatry. 2004;161:2045-2049.
14. Goldstein DJ, Mallinckrodt C, Lu Y, Demitrack MA. Duloxetine in the treatment of major depressive disorder: a double-blind clinical trial. J Clin Psychiatry. 2002;63(3):225-231.
15. Goldstein DJ, Lu Y, Detke MJ, Wiltse C, Mallinckrodt C, Demitrack MA. Duloxetine in the treatment of depression: a double-blind placebo-controlled comparison with paroxetine. J Clin Psychopharmacol. 2004;24:389-399.
16. Detke MJ, Wiltse CG, Mallinckrodt CH, McNamara RK, Demitrack MA, Bitter I. Duloxetine in the acute and long-term treatment of major depressive disorder: a placebo and paroxetine-controlled trial. Eur Neuropsychopharmacol. 2004;14:457-470.
17. Perahia DG, Wang F, Mallinckrodt CH, Walker DJ, Detke MJ. Duloxetine in the treatment of major depressive disorder: a placebo- and paroxetine-controlled trial. Eur Psychiatry. 2006;21:367-378.
18. Nierenberg AA, Greist JH, Mallinckrodt CH, et al. Duloxetine versus escitalopram and placebo in the treatment of patients with major depressive disorder: onset of antidepressant action, a non-inferiority study. Curr Med Res Opin. 2007;23:401-416.
19. Detke MJ, Lu Y, Goldstein DJ, Hayes JR, Demitrack MA. Duloxetine, 60 mg once daily for major depressive disorder: a randomized double-blind placebo-controlled trial. J Clin Psychiatry. 2002;63:308-315.
20. Detke MJ, Lu Y, Goldstein DJ, McNamara RK, Demitrack MA. Duloxetine 60 mg once daily dosing versus placebo in the acute treatment of major depression. J Psychiatr Res. 2002;36:383-390.
21. Raskin J, Wiltse CG, Siegal A, et al. Efficacy of duloxetine on cognition, depression, and pain in elderly patients with major depressive disorder: an 8-week, double-blind, placebo-controlled trial. Am J Psychiatry. 2007;164:900-909.
22. Nemeroff CB, Schatzberg AF, Goldstein DJ, et al. Duloxetine for the treatment of major depressive disorder. Psychopharmacol Bull. 2002;36:106-132.
23. Mallinckrodt CH, Prakash A, Houston JP, Swindle R, Detke MJ, Fava M. Differential antidepressant symptom efficacy: placebo-controlled comparisons of duloxetine and SSRIs (fluoxetine, paroxetine, escitalopram). Neuropsychobiology. 2007;56(2-3):73-85.
24. Eli Lilly and Company. A study comparing duloxetine and placebo in assessing energy and vitality in MDD patients. ClinicalTrials.gov. Bethesda, MD: National Library of Medicine; 2000. http://clinicaltrials.gov/show/NCT00536471.
25. Bech P, Kajdasz DK, Porsdal V. Dose-response relationship of duloxetine in placebo-controlled clinical trials in patients with major depressive disorder. Psychopharmacology (Berl). 2006;188:273-280.
26. Entsuah R, Shaffer M, Zhang J. A critical examination of the sensitivity of unidimensional subscales derived from the Hamilton Depression Rating Scale to antidepressant drug effects. J Psychiatr Res. 2002;36:437-448.
27. Faries D, Herrera J, Rayamajhi J, DeBrota D, Demitrack M, Potter WZ. The responsiveness of the Hamilton Depression Rating Scale. J Psychiatr Res. 2000;34(1):3-10.
28. Mallinckrodt CH, Meyers AL, Prakash A, Faries DE, Detke MJ. Simple options for improving signal detection in antidepressant clinical trials. Psychopharmcol Bull. 2007;40(2):101-114.
29. Maier W, Philipp M. Improving the assessment of severity of depressive states: a reduction of the Hamilton Depression Scale. Pharmacopsychiatry. 1985;18:114-115.
30. Papakostas GI, Fava M. Does the probability of receiving placebo influence clinical trial outcome? A meta-regression of double-blind, randomized clinical trials in MDD. Eur Neuropsychopharmacol. 2009;19(1):34-40.
31. Khan A, Detke M, Khan SR, Mallinckrodt C. Placebo response and antidepressant clinical trial outcome. J Nerv Ment Dis. 2003;191(4):211-218.
32. Khan A, Khan SR, Walens G, Kolts R, Giller EL. Frequency of positive studies among fixed and flexible dose antidepressant clinical trials: an analysis of the Food and Drug Administration summary basis of approval reports. Neuropsychopharmacology. 2003;28:552-557.