Adding experimental arms to platform clinical trials: Randomization procedures and interim analyses

Biostatistics

Volumn 19, Issue 2, 2018, Pages 199-215

  Ventz, Steffen ^a   Cellamare, Matteo ^b,c   Parmigiani, Giovanni ^b   Trippa, Lorenzo ^b  

^a University of Rhode Island   (United States)

^b HARVARD SCHOOL OF PUBLIC HEALTH   (United States)

^c SAPIENZA UNIVERSITY OF ROME   (Italy)

Author keywords

Bootstrap; Multi arm clinical trials; Outcome adaptive randomization; Platform trials

Indexed keywords

TUBERCULOSTATIC AGENT;

CLINICAL TRIAL (TOPIC); COMPUTER SIMULATION; HUMAN; METHODOLOGY; RANDOMIZATION; RANDOMIZED CONTROLLED TRIAL (TOPIC); STANDARDS; STATISTICAL ANALYSIS; STATISTICAL MODEL; TUBERCULOSIS;

ANTITUBERCULAR AGENTS; CLINICAL TRIALS AS TOPIC; COMPUTER SIMULATION; DATA INTERPRETATION, STATISTICAL; HUMANS; MODELS, STATISTICAL; RANDOM ALLOCATION; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN; TUBERCULOSIS;

EID: 85044764442     PISSN: 14654644     EISSN: 14684357     Source Type: Journal    
DOI: 10.1093/biostatistics/kxx030     Document Type: Article

References (23)

1. BARKER, A. D., SIGMAN, C. C., KELLOFF, G. J., HYLTON, N. M., BERRY, D.A. AND ESSERMAN, L. J. (2009). I-spy 2: An adaptive breast cancer trial design in the setting of neoadjuvant chemotherapy. Clinical Pharmacology & Therapeutics 86, 97-100.
2. BERRY, S. M., CARLIN, B. P., LEE, J. J. AND MULLER, P. (2010). Bayesian Adaptive Methods for Clinical Trials. Boca Raton, FL: CRC Press.
3. BERRY, S. M., CONNOR, J.T. AND LEWIS, R. J. (2015). The platform trial: an efficient strategy for evaluating multiple treatments. JAMA 313, 1619-1620.
4. BURNETT, A. K., RUSSELL, N. H., HILLS, R. K., HUNTER, A. E., KJELDSEN, L., YIN, J., GIBSON, B. E., WHEATLEY, K. AND MILLIGAN, D. (2013). Optimization of chemotherapy for younger patients with acute myeloid leukemia: results of the medical research council AML15 trial. Journal of Clinical Oncology, 31, 3360-3368.
5. CELLAMARE, M., VENTZ, S., BAUDIN, E., MITNICK, C. D. AND TRIPPA, L. (2017). A Bayesian response-adaptive trial in tuberculosis: the endTB trial. Clinical Trials 14, 17-28.
6. COHEN, D. R., TODD, S., GREGORY, W.M. AND BROWN, J. M. (2015). Adding a treatment arm to an ongoing clinical trial: a review of methodology and practice. Trials 16, 179.
7. EISELE, J. R. (1994). The doubly adaptive biased coin design for sequential clinical trials. Journal of Statistical Planning and Inference 38, 249-261.
8. ELM, J. J., PALESCH, Y. Y., KOCH, G. G., HINSON, V., RAVINA, B. AND ZHAO, W. (2012). Flexible analytical methods for adding a treatment arm mid-study to an ongoing clinical trial. Journal of Biopharmaceutical Statistics 22, 758-772.
9. US FDA. (2013). Guidance for industry: codevelopment of two or more new investigational drugs for use in combination. FDA. https://www.fda.gov/downloads/drugs/guidances/ucm236669.pdf.
10. FREIDLIN, B., KORN, E. L., GRAY, R. AND MARTIN, A. (2008). Multi-arm clinical trials of new agents: some design considerations. Clinical Cancer Research 14, 4368-4371.
11. HILLS, R. K. AND BURNETT, A. K. (2011). Applicability of a Pick aWinner trial design to acute myeloid leukemia. Blood 118, 2389-2394.
12. HOBBS, B. P., CHEN, N. AND LEE, J. J. (2016). Controlled multi-arm platform design using predictive probability. Statistical Methods in Medical Research, pii: 0962280215620696. [Epub ahead of print].
13. HU, F. AND ZHANG, L.-X. (2004). Asymptotic properties of doubly adaptive biased coin designs for multitreatment clinical trials. Annals of Statistics, 32, 268-301.
14. LAN, K. K. G. AND DEMETS, D. L. (1983). Discrete sequential boundaries for clinical trials. Biometrika 70, 659-663.
15. LEE, J. J., GU, X. AND LIU, S. (2010). Bayesian adaptive randomization designs for targeted agent development. Clinical Trials 7, 584-596.
16. LIEBERMAN, J. A., STROUP, T. S., MCEVOY, J. P., SWARTZ, M. S., ROSENHECK, R. A., PERKINS, D. O., KEEFE, R. S., DAVIS, S. M., DAVIS, C. E., LEBOWITZ, B. D. and others. (2005). Effectiveness of antipsychotic drugs in patients with chronic schizophrenia. NEJM 353, 1209-1223.
17. ROSENBERGER, W.F.ANDHU, F. (1999). Bootstrap methods for adaptive designs. Statistics in Medicine 18, 1757-1767.
18. THALL, P. F.ANDWATHEN, J.K. (2007). Practical Bayesian adaptive randomisation in clinical trials. European Journal of Cancer 43, 859-866.
19. TRIPPA, L., LEE, E. Q., WEN, P. Y., BATCHELOR, T. T., CLOUGHESY, T., PARMIGIANI, G. AND ALEXANDER, B. M. (2012). Bayesian adaptive randomized trial design for patients with recurrent glioblastoma. JCO 30, 3258-3263.
20. VENTZ, S., BARRY, W. T., PARMIGIANI, G. AND TRIPPA, L. (2017). Bayesian response-adaptive designs for basket trials. Biometrics, doi: 10.1111/biom.12668. [Epub ahead of print].
21. WASON, J. AND TRIPPA, L. (2014). A comparison of Bayesian adaptive randomization and multi-stage designs for multi-arm clinical trials. Statistics in Medicine 33, 2206-2221.
22. WASON, J. M., STECHER, L. AND MANDER, A. P. (2014). Correcting for multiple-testing in multi-arm trials: is it necessary and is it done? Trials 15, 364.
23. YUAN, Y., GUO, B., MUNSELL, M., LU, K. AND JAZAERI, A. (2016). MIDAS: a practical Bayesian design for platform trials with molecularly targeted agents. Statistics in Medicine 35, 3892-3906.