Blinded sample size recalculation in multicentre trials with normally distributed outcome

Biometrical Journal

Volumn 52, Issue 3, 2010, Pages 377-399

  Jensen, Katrin ^a   Kieser, Meinhard ^a  

^a UNIVERSITY OF HEIDELBERG   (Germany)

Author keywords

Internal pilot study; Multicentre clinical trial; Nuisance parameter; Power; Sample size recalculation; Type I error; Variance estimate

Indexed keywords

CONTROLLED DRUG DELIVERY; MEDICAL APPLICATIONS; SAMPLING;

INTERNAL PILOT STUDY; MULTI-CENTER CLINICAL TRIALS; NUISANCE PARAMETER; PILOT STUDIES; POWER; SAMPLE SIZE RECALCULATION; SAMPLE SIZES; STUDY DESIGN; TYPE-I ERROR; VARIANCE ESTIMATE;

NORMAL DISTRIBUTION;

DOUBLE BLIND PROCEDURE; HUMAN; METHODOLOGY; MULTICENTER STUDY (TOPIC); PILOT STUDY; PROCEDURES; RANDOMIZED CONTROLLED TRIAL (TOPIC); SAMPLE SIZE; STATISTICAL MODEL; TREATMENT OUTCOME;

DOUBLE-BLIND METHOD; HUMANS; MODELS, STATISTICAL; MULTICENTER STUDIES AS TOPIC; PILOT PROJECTS; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN; SAMPLE SIZE; TREATMENT OUTCOME;

EID: 77954467678     PISSN: 03233847     EISSN: 15214036     Source Type: Journal    
DOI: 10.1002/bimj.200900114     Document Type: Article

References (28)

1. Anello, C., O'Neill, R. and Dubey, S. (2005). Multicentre trials: a US regulatory perspective. Statistical Methods in Medical Research 14, 303-318.
2. Bauer P. and Köhne K. (1994). Evaluation of experiments with adaptive interim analyses. Biometrics 50, 1029-1041.
3. Birkett, M. A. and Day, S. J. (1994). Internal pilot studies for estimating sample size. Statistics in Medicine 13, 2455-2463.
4. Center for Drug Evaluation and Research, Food and Drug Administration, U.S. Department of Health and Human Services (1988). Guideline of the Format and Content of the Clinical and Statistical Sections of New Drug Applications. U.S. Food and Drug Administration, Rockville, MD, pp. 68-71.
5. Committee for Proprietary Medicinal Products (CPMP): Points to Consider on Adjustment for Baseline Covariates (2003). Available from: http://www.emea.europa.eu/pdfs/human/ewp/286399en.pdf (accessed on 31.10.2009).
6. Dragalin, V., Fedorov, V., Jones, B. and Rockhold, F. (2001). Estimation of the combined response to treatment in multicenter trials. Journal of Biopharmaceutical Statistics 11, 275-295.
7. Fedorov, V. and Jones, B. (2005). The design of multicentre trials. Statistical Methods in Medical Research 14, 205-248.
8. Fedorov, V., Jones, B., Jones, M. and Zhigljavsky, A. (2004). Estimation of the treatment difference in multicenter trials. Journal of Biopharmaceutical Statistics 14, 1037-1063.
9. Friede, T. and Kieser, M. (2002). On the inappropriateness of an EM algorithm based procedure for blinded sample size re-estimation. Statistics in Medicine 21, 165-176.
10. Friede, T. and Kieser, M. (2006). Sample size recalculation in internal pilot study designs: a review. Biometrical Journal 48, 537-555.
11. Gallo, P. P. (1998). Practical issues in linear models analyses in multicenter clinical trials. Biopharmaceutical Report 6, 2-9.
12. Gallo, P. P. (2000). Center-weighting issues in multicenter clinical trials. Journal of Biopharmaceutical Statistics 10, 145-163.
13. Gould, A. L. and Shih, W. J. (1992). Sample size re-estimation without unblinding for normally distributed outcomes with unknown variance. Communications in Statistics (A) 21, 2833-2853.
14. International Conference on Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use (1999). ICH E9. ICH harmonized tripartite guideline: Statistical principles for clinical trials. Statisitics in Medicine 18, 1905-1942.
15. Jones, B., Teather, D., Wang, J. and Lewis, J. A. (1998). A comparison of various estimators of a treatment difference for a multi-centre clinical trial. Statistics in Medicine 17, 1767-1777.
16. Kamin, W., Maydannik, V., Malek, F. A. and Kieser, M. (2010). Efficacy and tolerability of EPs 7630 in children and adolescents with acute bronchitis. A randomized, double-blind, placebo-controlled multicenter trial with a herbal drug preparation from Pelargonium sicloicles roots. International Journal of Clinical Pharmacology and Therapeutics 48, 184-191.
17. Kieser, M. and Friede, T. (2003). Simple procedures for blinded sample size adjustment that do not affect the type I error rate. Statistics in Medicine 22, 3571-3581.
18. Lin, Z. (1999). An issue of statistical analysis in controlled multi-centre studies: how shall we weight the centers? Statistics in Medicine 18, 365-373.
19. Proschan, M. (2009). Sample size re-estimation in clinical trials. Biometrical Journal 51, 348-357.
20. Ruvuna, F. (2004). Unequal center sizes, sample size, and power in multicenter clinical trials. Drug Information Journal 38, 387-394.
21. SAS Institute (1999). SAS/STAT User's Guide, Version 9. Cary, NC: SAS Institute.
22. Senn, S. (1998). Some controversies in planning and analyzing multi-center trials. Statistics in Medicine 17, 1753-1765.
23. Senn, S. (2007). Statistical Issues in Drug Development (2nd edn) Wiley, New York.
24. Shih, W. J. and Long, J. (1998). Blinded sample size re-estimation with unequal variances and center effects in clinical trials. Communications in Statistics (A) 27, 395-408.
25. Waksman, J. (2007). Assessment of the Gould-Shih procedure for sample size re-estimation. Pharmaceutical Statistics 6, 53-65.
26. Wittes, J. and Brittain, E. (1990). The role of internal pilot studies in increasing the efficiency of clinical trials. Statistics in Medicine 9, 65-72.
27. Wittes, J., Schabenberger, O., Zucker, D., Brittain, E. and Proschan, M. (1999). Internal pilot studies I: type I error rate of the naive t-test. Statistics in Medicine 18, 3481-3491.
28. Zucker, D. M., Wittes, J. T., Schabenberger, O. and Brittain, E. (1999). Internal pilot studies II: comparison of various procedures. Statistics in Medicine 18, 3493-3509.