Doubly randomized delayed-start design for enrichment studies with responders or nonresponders

Journal of Biopharmaceutical Statistics

Volumn 22, Issue 4, 2012, Pages 737-757

  Liu, Qing ^a   Lim, Pilar ^a   Singh, Jaskaran ^a   Lewin, David ^a   Schwab, Barry ^a   Kent, Justine ^a  

^a JANSSEN RESEARCH AND DEVELOPMENT   (United States)

Author keywords

Adaptive design; Enrichment design; Maintenance effect; Proof of concept trials; Randomized start design; Sequential parallel design

Indexed keywords

PLACEBO;

ALTERNATIVE HYPOTHESIS; ANALYSIS OF COVARIANCE; ARTICLE; CLINICAL TRIAL (TOPIC); CORRELATION ANALYSIS; CORRELATION COEFFICIENT; FOLLOW UP; HAMILTON SCALE; HUMAN; MAJOR DEPRESSION; MONTE CARLO METHOD; NORMAL DISTRIBUTION; NULL HYPOTHESIS; PRIORITY JOURNAL; PROBABILITY; PROPORTIONAL HAZARDS MODEL; RANDOMIZATION; SAMPLE SIZE; SCORING SYSTEM; SENSITIVITY ANALYSIS; STATISTICAL ANALYSIS; STUDY DESIGN; SYSTEMATIC ERROR; TREATMENT RESPONSE;

ALGORITHMS; BIAS (EPIDEMIOLOGY); CENTRAL NERVOUS SYSTEM DISEASES; CROSS-OVER STUDIES; HUMANS; NEOPLASMS; PLACEBOS; PROBABILITY; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN; SAMPLE SIZE;

EID: 84863448922     PISSN: 10543406     EISSN: 15205711     Source Type: Journal    
DOI: 10.1080/10543406.2012.678234     Document Type: Article

References (22)

1. Capizzi, T., Survill, T. T., Heyes, J. F. (1992). An empirical and simulated comparison of some tests for detecting progressiveness of response with increasing doses of a compound. Biometrical Journal 3:275-289.
2. Chen, Y. F., Yang, Y., Hung, H. M. J., Wang, S. J. (2011). Evaluation of performance of some enrichment designs dealing with high placebo response in psychiatric clinical trials. Contemporary Clinical Trials 32:592-604.
3. Chi, Y. G., Liu, Q. (1999). The attractiveness of the concept of a prospectively designed two-stage clinical trial. Journal of Biopharmaceutical Statistics 9(4):537-547. (Pubitemid 29535946)
4. Cui, L., Hung, H. M. J., Wang, S. J. (1999). Modification of sample size in group sequential clinical trials. Biometrics 55:853-857. (Pubitemid 29427088)
5. Fava, M., Evins, A. E., Dorer, D. J., Schoenfeld, D. A. (2003). The problem of the placebo response in clinical trials for psychiatric disorders: culprits, possible remedies, and a novel study design approach. Psychotherapy and Psychosomatics 72:115-127. (Pubitemid 36469860)
6. Habermann, T. M., Weller, E. A., Morrison, V. A., et al. (2006). Rituximab-CHOP versus CHOP alone or with maintenance rituximab in older patients with diffuse large B-cell lymphoma. Journal of Clinical Oncology 24:3121-3127. (Pubitemid 46638950)
7. Heyn, R. M., Joo, P., Karon, M., et al. (1974). BCG in the treatment of acute lymphocytic leukemia. Blood 46:431-442.
8. Jennison, C., Turnbull, B. W. (2000). Group Sequential Methods with Applications to Clinical Trials Boca Raton, FL: Chapman & Hall.
9. Khin, N. A., Chen, Y. (2011). Exploratory analyses of efficacy data from major depressive disorder trials submitted to the US Food and Drug Administration in support of new drug applications. Journal of Clinical Psychiatry 72:464-472.
10. Leber, P. (1996). Observations and suggestions for antidementia drug development. Alzheimer Disease and Associated Disorders 10(suppl. 1):31-34.
11. Leber, P. (1997). Slowing the progression of Alzheimer disease: Methodological issues. Alzheimer Disease and Associated Disorders 11(suppl. 5):10-21.
12. Liu, Q., Chi, G. Y. H. (2010a). Fundamental theory of adaptive designs with unplanned design change in clinical trials with blinded data. In: Pong, A., Chow, S. C., eds. Handbook of Adaptive Designs in Pharmaceutical and Clinical Development. Boca Raton, FL: Chapman & Hall, pp.2-1-2-8.
13. Liu, Q., Chi, G. Y. H. (2010b). Understanding the FDA guidance on adaptive designs: historical, legal and statistical perspectives. Journal of Biopharmaceutical Statistics 20(special issue):1178-1219.
14. Liu, Q., Pledger, G. W. (2005). Interim analysis and bias in clinical trials: The adaptive design perspective. In: Buncher, R., Tsay, J.-Y., eds. Statistics in the Pharmaceutical Industry. 3rd ed. Revised and Expanded, New York: Taylor & Francis, pp. 231-244.
15. Liu, Q., Proschan, M. A., Pledger, G. W. (2002). A unified theory of two-stage adaptive designs. Journal of the American Statistical Association 97:1034-1041. (Pubitemid 36136563)
16. McDermott, M. P., Hall, W. J., Oakes, D., Eberly, S. (2002). Design and analysis of twoperiod studies of potentially disease-modifying treatments. Controlled Clinical Trials 23:635-649. (Pubitemid 36005474)
17. Mills, E. J., Kelly, S., Wu P., Guyatt, G. H. (2007). Epidemiology and reporting of randomized trials employing rerandomization of patient groups: a systematic survey. Contemporary Clinical Trials 28:268-275. (Pubitemid 46330170)
18. Papakostas, G. I., Fava, M. (2009). Does the probability of receiving placebo influence clinical trial outcome? A meta-regression of double-blind, randomized clinical trials in MDD. European Neuropsychopharmacology 19:34-40.
19. Sinyor, M., Levitt, A. J., et al. (2010). Does inclusion of a placebo arm influence response to active antidepressant treatment in randomized controlled trials? Results from pooled and meta-analyses. Journal of Clinical Psychiatry 71:270-279.
20. Tamura, R., Huang, X. (2007). An examination of the efficiency of the sequential parallel design in psychiatric clinical trials. Clinical Trials 4:309-317. (Pubitemid 47572263)
21. Tedeschini, E., Fava, M., Goodness, T. M., Papakostas, G. I. (2010). Relationship between probability of receiving placebo and probability of prematurely discontinuing treatment in double-blind, randomized clinical trials for MDD: a meta-analysis. European Neuropsychopharmacology 20:562-567.
22. Temple, R. J. (1994). Special study designs: early escape, enrichment, studies in nonresponders. Communications in Statistics - Theory and Methods 23:499-531.