The statistics of phase 0 trials

Statistics in Medicine

Volumn 29, Issue 10, 2010, Pages 1072-1076

  Rubinstein, Larry V ^a   Steinberg, Seth M ^a   Kummar, Shivaani ^a   Kinders, Robert ^b   Parchment, Ralph E ^b   Murgo, Anthony J ^c   Tomaszewski, Joseph E ^a   Doroshow, James H ^a  

^a NATIONAL CANCER INSTITUTE   (United States)

^b SAIC FREDERICK INC   (United States)

^c CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

Author keywords

First in man study; PD driven phase 0 trial; Pharmacodynamic assay; Phase 1 clinical trial; Phase 2 clinical trial

Indexed keywords

2 (2 METHYL 2 PYRROLIDINYL) 1H BENZIMIDAZOLE 4 CARBOXAMIDE;

ARTICLE; CLINICAL TRIAL; DOSE RESPONSE; DRUG ACTIVITY; DRUG DEVELOPMENT; DRUG DOSE REGIMEN; DRUG EFFECT; DRUG SCREENING; ONCOLOGY; PHARMACODYNAMICS; PHASE 0 CLINICAL TRIAL; STATISTICS;

ANTINEOPLASTIC AGENTS; CLINICAL TRIALS AS TOPIC; DATA INTERPRETATION, STATISTICAL; DRUGS, INVESTIGATIONAL; HUMANS; NEOPLASMS; PHARMACOKINETICS;

EID: 77951605058     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.3840     Document Type: Article

References (10)

1. Kummar S, Kinders R, Rubinstein L, Parchment RE, Murgo AJ, Collins J, Pickeral O, Low J, Steinberg SM, Gutierrez M, Yang S, Helman L, Wiltrout R, Tomaszewski JE, Doroshow JH. Compressing drug development timelines in oncology using 'phase 0' trials. Nature Reviews/Cancer 2007; 7:131-139.
2. Murgo AJ, Kummar S, Rubinstein L, Gutierrez M, Collins J, Kinders R, Parchment RE, Ji J, Steinberg SM, Yang SX, Hollingshead M, Chen A, Helman L, Wiltrout R, Tomaszewski JE, Doroshow JH. Designing phase 0 cancer clinical trials. Clinical Cancer Research 2008; 14:3675-3682.
3. Kinders R, Parchment RE, Ji J, Kummar S, Murgo AJ, Gutierrez M, Collins J, Rubinstein L, Pickeral O, Steinberg SM, Yang S, Hollingshead M, Chen A, Helman L, Wiltrout R, Simpson M, Tomaszewski JE, Doroshow JH. Phase 0 clinical trials in cancer drug development: from FDA guidance to clinical practice. Molecular Interventions 2007; 7:325-334.
4. Simon R. Optimal two-stage designs for phase II clinical trials. Controlled Clinical Trials 1989; 10:1-10. (Pubitemid 19099520)
5. Kummar S, Gutierrez M, Kinders R, Rubinstein L, Parchment RE, Philips L, Ji J, Chen A, Horneffer Y, Juwara L, Yancey M, Murgo AJ, Collins JM, Tomaszewski JE, Doroshow JH. Phase 0 pharmacodynamic, pharmacokinetic study of ABT-888, an inhibitor of poly (ADP-ribose) polymerase (PARP), in patients with advanced malignancies. Annals of Oncology 2008; 19(Suppl. 3):20.
6. Kummar S, Kinders R, Gutierrez M, Rubinstein L, Parchment RE, Phillips LR, Ji J, Monks A, Pickeral O, DiPaolo T, Low J, Chen A, Murgo AJ, Collins J, Simpson M, Steinberg S, Yang SX, Horneffer Y, Juwara L, Yancey MA, Hollingshead M, Helman L, Wiltrout R, Tomaszewski JE, Doroshow JH, the NCI Phase 0 Working Group. Phase 0 clinical trial of the poly (ADP-ribose) polymerase (PARP) inhibitor ABT-888 in patients with advanced malignancies. Journal of Clinical Oncology 2009; 27:2705-2711.
7. Karrison TG, Maitland ML, Stadler WM, Ratain MJ. 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. Journal of the National Cancer Institute 2007; 99:1455-1461.
8. Rubinstein L, Dancey J, Korn E, Smith M, Wright J. Early average change in tumor size in a phase 2 trial: efficient endpoint or false promise. Journal of the National Cancer Institute 2007; 99:1422-1423.
9. LoRusso PM. Phase 0 clinical trials: an answer to drug development stagnation? Journal of Clinical Oncology 2009; 27:2586-2588.
10. Editors. Phase 0 trials: a platform for drug development? The Lancet 2009; 374:176.