Comparison of error rates in single-arm versus randomized phase II cancer clinical trials

Journal of Clinical Oncology

Volumn 28, Issue 11, 2010, Pages 1936-1941

  Tang, Hui ^a   Foster, Nathan R ^a   Grothey, Axel ^a   Ansell, Stephen M ^a   Goldberg, Richard M ^a   Sargent, Daniel J ^a  

^a NONE

Author keywords

[No Author keywords available]

Indexed keywords

FLUOROURACIL; FOLINIC ACID; IRINOTECAN; ANTINEOPLASTIC AGENT;

ADULT; ARTICLE; COLORECTAL CANCER; COMPARATIVE STUDY; ERROR; FALSE POSITIVE RESULT; FEMALE; HUMAN; MAJOR CLINICAL STUDY; MALE; METHODOLOGY; NULL HYPOTHESIS; OUTCOME ASSESSMENT; PATIENT SELECTION; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); SAMPLE SIZE; SIMULATION; STATISTICAL MODEL; AGED; BIOASSAY; CASE CONTROL STUDY; COLORECTAL TUMOR; COMPUTER SIMULATION; LABORATORY DIAGNOSIS; PHASE 2 CLINICAL TRIAL; PROGNOSIS; RANDOMIZED CONTROLLED TRIAL; STANDARD; SURVIVAL RATE;

AGED; ANTINEOPLASTIC AGENTS; CASE-CONTROL STUDIES; CLINICAL TRIALS, PHASE II AS TOPIC; COLORECTAL NEOPLASMS; COMPUTER SIMULATION; ENDPOINT DETERMINATION; FALSE NEGATIVE REACTIONS; FALSE POSITIVE REACTIONS; FEMALE; HUMANS; MALE; PROGNOSIS; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN; SAMPLE SIZE; SURVIVAL RATE;

EID: 77951639219     PISSN: 0732183X     EISSN: 15277755     Source Type: Journal    
DOI: 10.1200/JCO.2009.25.5489     Document Type: Article

References (26)

1. Simon R: Optimal two-stage designs for phase II clinical trials. Control Clin Trials 10:1-10, 1989
2. Green SJ, Dahlberg S: Planned versus attained design in phase II clinical trials. Stat Med 11:853-862, 1992
3. Chalmers TC: When should randomisation begin? Lancet 291:858, 1968
4. Senn S: Statistical Issues in Drug Development (ed 2). Malden, MA, Wiley-Interscience, 2008
5. Kindler HL, Friberg G, Singh DA, et al: Phase II trial of bevacizumab plus gemcitabine in patients with advanced pancreatic cancer. J Clin Oncol 23:8033-8040, 2005 (Pubitemid 46657405)
6. Kindler HL, Niedzwiecki D, Hollis D, et al: A double-blind, placebo-controlled, randomized phase III trial of gemcitabine plus bevacizumab versus gemcitabine plus placebo in patients with advanced pancreatic cancer: A preliminary analysis of Cancer and Leukemia Group B. J Clin Oncol 25:199s, 2007 (suppl; abstr 4508)
7. Xiong HQ, Rosenberg A, LoBuglio A, et al: Cetuximab, a monoclonal antibody targeting the epidermal growth factor receptor, in combination with gemcitabine for advanced pancreatic cancer: A multicenter phase II trial. J Clin Oncol 22:2610-2616, 2004 (Pubitemid 41103748)
8. Philip PA, Benedetti J, Fenoglio-Preiser C, et al: Phase III study of gemcitabine [G] plus cetuximab [C] versus gemcitabine in patients [pts] with locally advanced or metastatic pancreatic adenocarcinoma [PC]: SWOG S0205 study. J Clin Oncol 25:199s, 2007 (suppl; abstr LBA4509)
9. Thall PF, Simon R: Incorporating historical control data in planning phase II clinical trials. Stat Med 9:215-228, 1990 (Pubitemid 20252730)
10. Williams DA: Extra-binomial variation in logistic linear models. Appl Statist 31:144-148, 1982
11. Estey EH, Thall PF: New designs for phase 2 clinical trials. Blood 102:442-448, 2003 (Pubitemid 36841959)
12. Rubinstein LV, Korn EL, Freidlin B, et al: Design issues of randomized phase II trials and a proposal for phase II screening trials. J Clin Oncol 23:7199-7206, 2005
13. Taylor JM, Braun TM, Li Z: Comparing an experimental agent to a standard agent: Relative merits of a one-arm or randomized two-arm phase II design. Clinical Trials 3:335-348, 2006 (Pubitemid 44645824)
14. Sanoff HK, Sargent DJ, Campbell ME, et al: Five-year data and prognostic factor analysis of oxaliplatin and irinotecan combinations for advanced colorectal cancer: N9741. J Clin Oncol 28:5721-5727, 2008
15. Saltz LB, Cox JV, Blanke C, et al: Irinotecan plus fluorouracil and leucovorin for metastatic colorectal cancer. N Engl J Med 343:905-914, 2000
16. Ratain MJ, Sargent DJ: Optimizing the design of phase II oncology trials: The importance of randomization. Eur J Cancer 45:275-280, 2009
17. Rubinstein L, Crowley J, Ivy P, et al: Randomized phase II designs. Clinical Cancer Res 15:1883-1890, 2009
18. McShane LM, Hunsberger S, Adjei AA: Effective incorporation of biomarkers into phase II trials. Clin Cancer Res 15:1898-1905, 2009
19. Chan JK, Ueda SM, Sugiyama VE, et al: Analysis of phase II studies on targeted agents and subsequent phase II trials: What are the predictors for success? J Clin Oncol 26:1511-1518, 2008
20. EI-Maraghi RH, Eisenhauer EA: Review of phase II trial designs used in studies of molecular targeted agents: Outcomes and predictors of success in phase III. J Clin Oncol 26:1346-1354, 2008
21. Korn EL, Liu PY, Lee SJ, et al: Meta-analysis of phase II cooperative group trials in metastatic stage IV melanoma to determine progression-free and overall survival benchmarks for future phase II trials. J Clin Oncol 26:527-534, 2008 (Pubitemid 351264344)
22. London WB, Chang MN: One- and two-stage designs for stratified phase II clinical trials. Stat Med 24:2597-2611, 2005 (Pubitemid 41300797)
23. Parmer MKB, Barthel FMS, Sydes M, et al: Speeding up the evaluation of new agents in cancer. J Natl cancer Inst 100:1204-1214, 2008
24. Vickers AJ, Ballen V, Scher HI: Setting the bar in phase II trials: The use of historical data for determining "Go/No Go" decision for definitive phase III testing. Clin Cancer Res 13:972-976, 2007
25. Fazzari M, Heller G, Scher HI: The phase II/III transition: Toward the proof of efficacy in cancer clinical trials. Control Clin Trials 21:360-368, 2000 (Pubitemid 30456295)
26. Mazumdar M, Fazzari M, Panageas KS: A standardization method to adjust for the effect of patient selection in phase II clinical trials. Stat Med 20:883-892, 2001 (Pubitemid 32249087)