Designing clinical trials that accept new arms: An example in metastatic breast cancer

Journal of Clinical Oncology

Volumn 35, Issue 27, 2017, Pages 3160-3168

  Ventz, Steffen ^b   Alexander, Brian M ^a,c   Parmigiani, Giovanni ^a,d   Gelber, Richard D ^a,c,d,e   Trippa, Lorenzo ^a,d  

^a DANA FARBER CANCER INSTITUTE   (United States)

^b UNIVERSITY OF RHODE ISLAND   (United States)

^c HARVARD MEDICAL SCHOOL   (United States)

^d HARVARD SCHOOL OF PUBLIC HEALTH   (United States)

^e New Frontier Science Takeda Pharmaceuticals   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ABEMACICLIB; ALPELISIB; CYCLIN DEPENDENT KINASE 4; CYCLIN DEPENDENT KINASE 6; EVEROLIMUS; LETROZOLE; PALBOCICLIB; RIBOCICLIB; TEMSIROLIMUS; ANTINEOPLASTIC AGENT;

ADVANCED CANCER; ARTICLE; CANCER COMBINATION CHEMOTHERAPY; CANCER HORMONE THERAPY; CANCER PATIENT; CANCER SURVIVAL; COMPARATIVE STUDY; CONTROLLED STUDY; DATA ANALYSIS; DRUG EFFICACY; DRUG INDUSTRY; ERROR; EXPERIMENTAL THERAPY; FEMALE; HEALTH CARE QUALITY; HUMAN; HUMAN EPIDERMAL GROWTH FACTOR RECEPTOR 2 POSITIVE BREAST CANCER; MAJOR CLINICAL STUDY; MEDIAN SURVIVAL TIME; METASTATIC BREAST CANCER; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; POSTMENOPAUSE; PRIORITY JOURNAL; PROGRESSION FREE SURVIVAL; SAMPLE SIZE; SIMULATION; STUDY DESIGN; TIME TO TREATMENT; TREATMENT OUTCOME; BREAST NEOPLASMS; METHODOLOGY; RANDOMIZED CONTROLLED TRIAL (TOPIC);

ANTINEOPLASTIC AGENTS; BREAST NEOPLASMS; FEMALE; HUMANS; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN; SAMPLE SIZE;

EID: 85029691485     PISSN: 0732183X     EISSN: 15277755     Source Type: Journal    
DOI: 10.1200/JCO.2016.70.1169     Document Type: Article

References (30)

1. Hay M, Thomas DW, Craighead JL, et al: Clinical development success rates for investiga-tional drugs. Nat Biotechnol 32:40-51, 2014
2. Freidlin B, Korn EL, Gray R, et al: Multi-arm clinical trials of new agents: Some design considerations. Clin Cancer Res 14:4368-4371, 2008
3. Wason JM, Stecher L, Mander AP: Correcting for multiple-testing in multi-arm trials: Is it necessary and is it done? Trials 15:364, 2014
4. Rugo HS, Olopade OI, DeMichele A, et al: Adaptive randomization of veliparib–carboplatin treatment in breast cancer. N Engl J Med 375:23-34, 2016
5. Park JW, Liu MC, Yee D, et al: Adaptive randomization of neratinib in early breast cancer. N Engl J Med 375:11-22, 2016
6. Moore KN, Mannel RS: Is the NCI MATCH trial a match for gynecologic oncology? Gynecol Oncol 140:161-166, 2016
7. Esserman LJ, Woodcock J: Accelerating identification and regulatory approval of investi-gational cancer drugs. JAMA 306:2608-2609, 2011
8. Harrington D, Parmigiani G: I-SPY 2–A glimpse of the future of phase 2 drug development? N Engl J Med 375:7-9, 2016
9. Mullard A: Multicompany trials adapt to disciplines beyond cancer. Nat Med 20:3, 2014
10. Barker AD, Sigman CC, Kelloff GJ, et al: I-SPY 2: An adaptive breast cancer trial design in the setting of neoadjuvant chemotherapy. Clin Pharmacol Ther 86:97-100, 2009
11. Sydes MR, Parmar MK, James ND, et al: Issues in applying multi-arm multi-stage methodology to a clinical trial in prostate cancer: The MRC STAMPEDE trial. Trials 10:39, 2009
12. Burnett AK, Russell NH, Hills RK, et al: Optimization of chemotherapy for younger patients with acute myeloid leukemia: Results of the medical research council AML15 trial. J Clin Oncol 31: 3360-3368, 2013
13. Hills RK, Burnett AK: Applicability of a “Pick a Winner” trial design to acute myeloid leukemia. Blood 118:2389-2394, 2011
14. Elm JJ, Palesch YY, Koch GG, et al: Flexible analytical methods for adding a treatment arm mid-study to an ongoing clinical trial. J Biopharm Stat 22: 758-772, 2012
15. Lieberman JA: Effectiveness of antipsychotic drugs in patients with chronic schizophrenia: Efficacy, safety and cost outcomes of CATIE and other trials. J Clin Psychiatry 68:e04, 2007
16. Saville BR, Berry SM: Efficiencies of platform clinical trials: A vision of the future. Clin Trials 13: 358-366, 2016
17. Hobbs BP, Chen N, Lee JJ: Controlled multiarm platform design using predictive probability. Stat Methods Med Res0962280215620696, 2016. In press
18. Berry SM, Connor JT, Lewis RJ: The platform trial: An efficient strategy for evaluating multiple treatments. JAMA 313:1619-1620, 2015
19. Jennison C, Turnbull BW: Statistical approaches to interim monitoring of medical trials: A review and commentary. Stat Sci 5:299-317, 1990
20. Lan KG, DeMets DL: Discrete sequential boundaries for clinical trials. Biometrika 70:659-663, 1983
21. Trippa L, Lee EQ, Wen PY, et al: Bayesian adaptive randomized trial design for patients with recurrent glioblastoma. J Clin Oncol 30:3258-3263, 2012
22. Wason JM, Trippa L: A comparison of Bayesian adaptive randomization and multi-stage designs for multi-arm clinical trials. Stat Med 33: 2206-2221, 2014
23. Ventz S, Trippa L: Bayesian designs and the control of frequentist characteristics: A practical solution. Biometrics 71:218-226, 2015
24. Ventz S, Barry WT, Parmigiani G, et al: Bayesian response-adaptive designs for basket trials. Biometrics 10.1111/biom.12668 [epub ahead of print on February 17, 2017]
25. Xu Y, Trippa L, Müller P, et al: Subgroup-based adaptive (suba) designs for multi-arm biomarker trials. Stat Biosci 8:159-180, 2016
26. Cardoso F, Costa A, Norton L, et al: ESO-ESMO 2nd international consensus guidelines for advanced breast cancer (ABC2). Breast 23:489-502, 2014
27. Mouridsen H, Gershanovich M, Sun Y, et al: Phase III study of letrozole versus tamoxifen as first-line therapy of advanced breast cancer in postmenopausal women: Analysis of survival and update of efficacy from the International Letrozole Breast Cancer Group. J Clin Oncol 21:2101-2109, 2003
28. Finn RS, Crown JP, Lang I, et al: The cyclin-dependent kinase 4/6 inhibitor palbociclib in combination with letrozole versus letrozole alone as first-line treatment of oestrogen receptor-positive, HER2-negative, advanced breast cancer (PALOMA-1/TRIO-18): A randomised phase 2 study. Lancet Oncol 16: 25-35, 2015
29. Collett D: Modeling Survival Data in Medical Research. London, United Kingdom, Chapman and Hall, 1994
30. Law MG, Emery S: Selective exclusion of treatment arms in multi-arm randomized clinical trials. Stat Med 22:19-30, 2003