Is response rate relevant to the phase II trial design of targeted agents?

Journal of Clinical Oncology

Volumn 26, Issue 8, 2008, Pages 1204-1205

  Dowlati, Afshin ^a   Fu, Pingfu ^b  

^a UNIVERSITY HOSPITALS OF CLEVELAND   (United States)

^b CASE WESTERN RESERVE UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTINEOPLASTIC AGENT; BEVACIZUMAB; EPIDERMAL GROWTH FACTOR RECEPTOR 2; EPIDERMAL GROWTH FACTOR RECEPTOR ANTIBODY; PLACEBO; TRASTUZUMAB; TUMOR MARKER;

ALTERNATIVE HYPOTHESIS; CANCER GROWTH; CLINICAL TRIAL; COMBINATION CHEMOTHERAPY; DIAGNOSTIC ACCURACY; DOSE CALCULATION; DRUG DESIGN; DRUG DOSE COMPARISON; DRUG EFFICACY; DRUG MEGADOSE; EDITORIAL; HUMAN; LUNG NON SMALL CELL CANCER; MONOTHERAPY; NEOPLASM; PHASE 2 CLINICAL TRIAL; POPULATION ENRICHMENT; POPULATION RISK; PRIORITY JOURNAL; RANDOMIZATION; SURVIVAL TIME; TREATMENT RESPONSE; BIOASSAY; FEMALE; METABOLISM; METHODOLOGY; NOTE;

ANTINEOPLASTIC COMBINED CHEMOTHERAPY PROTOCOLS; CLINICAL TRIALS, PHASE II AS TOPIC; ENDPOINT DETERMINATION; FEMALE; HUMANS; NEOPLASMS; RESEARCH DESIGN; TUMOR MARKERS, BIOLOGICAL;

EID: 41649089533     PISSN: 0732183X     EISSN: None     Source Type: Journal    
DOI: 10.1200/JCO.2007.15.2827     Document Type: Editorial

References (17)

1. Simon R: Optimal two-stage designs for phase II clinical trials. Control Clin Trials 10:1-10, 1989
2. Case DL, Morgan TM: Design of phase II cancer trials evaluating survival probabilities. BMC Med Res Methodol 3:6, 2003
3. Karrison TG, Maitland ML, Stadler WM, et al: Design of phase II cancer trials using a continuous endpoint of change in tumor size: Application to a study of sorafenib and erlotinib in non small-cell lung cancer. J Natl Cancer Inst 99:1455-1461, 2007
4. Dent S, Zee B, Dancey J, et al: Application of a new multinomial phase II stopping rule using response and early progression. J Clin Oncol 19:785-791, 2001
5. El-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
6. Kris MG, Natale RB, Herbst RS, et al: Efficacy of gefitinib, an inhibitor of the epidermal growth factor receptor tyrosine kinase, in symptomatic patients with non-small cell lung cancer: A randomized trial. JAMA 290:2149-2158, 2003
7. Goffin J, Baral S, Tu D, et al: Objective responses in patients with malignant melanoma or renal cell cancer in early clinical studies do not predict regulatory approval. Clin Cancer Res 11:5928-5934, 2005
8. Sandler AB, Gray R, Perry MC, et al: Paclitaxel-carboplatin alone or with bevacizumab for non-small-cell lung cancer. N Engl J Med 355:2542-2550, 2006
9. Johnson DH, Fehrenbacher L, Novotny WF, et al: Randomized phase II trial comparing bevacizumab plus carboplatin and paclitaxel with carboplatin and paclitaxel alone in previously untreated locally advanced or metastatic non-small-cell lung cancer. J Clin Oncol 22:2184-2191, 2004
10. Slamon DJ, Leyland-Jones B, Shak S, et al: Use of chemotherapy plus a monoclonal antibody against HER2 for metastatic breast cancer that overexpresses HER2. N Engl J Med 344:783-792, 2001
11. Sequist LV, Bell DW, Lynch TJ, Haber DA: Molecular predictors of response to epidermal growth factor receptor antagonists in non-small-cell lung cancer. J Clin Oncol 25:587-595, 2007
12. Geyer CE, Forster J, Lindquist D, et al: Lapatinib plus capecitabine for HER2-positive advanced breast cancer. N Engl J Med 355:2733-2743, 2006
13. Rosner GL, Stadler W, Ratain M: Randomized discontinuation design: Application to cytostatic antineoplastic agents. J Clin Oncol 20:4478-4484, 2002
14. Ratain MJ, Eisen T, Stadler WM, et al: Phase II placebo-controlled randomized discontinuation trial of sorafenib in patients with metastatic renal cell carcinoma. J Clin Oncol 24:2505-2512, 2006
15. Capra WB: Comparing the power of the discontinuation design to that of the classic randomized design on time-to-event endpoints. Control Clin Trials 25:168-177, 2004
16. Leber PD, Davis CS: Threats to the validity of clinical trials employing enrichment strategies for sample selection. Control Clin Trials 19:178-187, 1998
17. Bagai RK, Dowlati A: Evolution of statistical methodology and design of phase II/III clinical trials in non-small cell lung cancer. J Clin Oncol 24:391s, 2006 (suppl; abstr 7108)