One- and two-stage design proposals for a Phase II trial comparing three active treatments with control using an ordered categorical endpoint

Statistics in Medicine

Volumn 28, Issue 5, 2009, Pages 828-847

  Whitehead, John ^a   Jaki, Thomas ^a  

^a LANCASTER UNIVERSITY   (United Kingdom)

Author keywords

Interim analysis; One stage design; Ordinal data; Phase II trial; Two stage design

Indexed keywords

PLACEBO;

ARTICLE; BARTHEL INDEX; CEREBROVASCULAR ACCIDENT; CLASSIFICATION; CLINICAL RESEARCH; CLINICAL TRIAL; CLUSTER ANALYSIS; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; EXPERIMENTAL DESIGN; HUMAN; PHASE 2 CLINICAL TRIAL; PROBABILITY; RANDOMIZED CONTROLLED TRIAL; SAMPLE SIZE; SIMULATION; STATISTICAL ANALYSIS; ALGORITHM; COMPUTER SIMULATION; EPIDEMIOLOGY; METHODOLOGY; NONPARAMETRIC TEST; STATISTICAL MODEL; STROKE; TREATMENT OUTCOME;

ALGORITHMS; CLINICAL TRIALS, PHASE II AS TOPIC; COMPUTER SIMULATION; EPIDEMIOLOGIC RESEARCH DESIGN; HUMANS; MEDICAL FUTILITY; MODELS, STATISTICAL; PROBABILITY; RANDOMIZED CONTROLLED TRIALS AS TOPIC; SAMPLE SIZE; STATISTICS, NONPARAMETRIC; STROKE; TREATMENT OUTCOME;

EID: 64549124373     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.3508     Document Type: Article

References (20)

1. Pocock SJ. Clinical Trials: A Practical Approach. Wiley: New York, 1983.
2. Dunnett CW. Selection of the best treatment in comparison to a control with an application to a medical trial. In Design of Experiments: Ranking and Selection, Santner TJ, Tamhane AC (eds). Marcel Dekker: New York, 1984; 47-66.
3. Thall PF, Simon R, Ellenberg SS. Two-stage selection and testing designs for comparative clinical trials. Biometrika 1988; 75:303-310.
4. Stallard N, Todd S. Sequential designs for phase III clinical trials incorporating treatment selection. Statistics in Medicine 2003; 22:689-703.
5. Bretz F, Schmidli H, König F, Racine A, Maurer W. Confirmatory seamless phase II/III clinical trials with hypotheses selection at interim: general concepts. Biometrical Journal 2006; 48:623-634.
6. Schmidli H, Bretz F, König F, Racine A, Maurer W. Confirmatory seamless phase II/III clinical trials with hypotheses selection at interim: applications and practical considerations. Biometrical Journal 2006; 48:635-643.
7. Jennison C, Turnbull BW. Confirmatory seamless phase II/III clinical trials with hypotheses selection at interim: opportunities and limitations. Biometrical Journal 2006; 48:650-655.
8. Koenig F, Brannath W, Bretz F, Posch M. Adaptive Dunnett tests for treatment selection. Statistics in Medicine 2008; 27:1612-1625.
9. Whitehead J. Application of sequential methods to a phase III clinical trial in stroke. Drug Information Journal 1993; 27:733-740.
10. Mahoney FI, Barthel DW. Functional evaluation: the Barthel index. Maryland State Medical Journal 1965; 14:61-65.
11. Jones DR, Whitehead J. Sequential forms of the log rank and modified Wilcoxon tests for censored data. Biometrika 1979; 66:105-113.
12. McCullagh P. Regression models for ordinal data (with Discussion). Journal of the Royal Statistical Society, Series B 1980; 42:109-142.
13. Dark R, Bolland K, Whitehead J. Statistical methods for ordered categorical data based on a constrained odds model. Biometrical Journal 2003; 45:453-470.
14. Scharfstein DO, Tsiatis AA, Robins JM. Semiparametric efficiency and its implication on the design and analysis of group-sequential studies. Journal of the American Statistical Association 1997; 92:1342-1350.
15. R Development Core Team. R: A Language and Environment for Statistical Computing. R Foundation for Statistical Computing, Vienna, Austria, 2008.
16. Todd S, Stallard N. A new clinical trial design combining phases II and III: sequential designs with treatment selection and a change of endpoint. Drug Information Journal 2005; 39:109-118.
17. Kelly PJ, Stallard N, Todd S. An adaptive group sequential design for phase II/III clinical trials that select a single treatment from several. Journal of Biopharmaceutical Statistics 2005; 15:641-658.
18. MPS Research Unit. PEST4: Operating Manual. Lancaster University, 2000.
19. Lesaffre E, Scheys I, Fröhlich J, Bluhmki E. Calculation of power and sample size with bounded outcome scores. Statistics in Medicine 1993; 12:1063-1078.
20. Lees KR, Asplund K, Carolei A, Davis SM, Diener H-C, Kaste M, Orgogozo J-M, Whitehead J. Glycine antagonist (gavestinel) in neuroprotection (GAIN International) in patients with acute stroke: a randomised controlled trial. Lancet 2000; 355:1949-1954.