A Bayesian response-adaptive trial in tuberculosis: The endTB trial

Clinical Trials

Volumn 14, Issue 1, 2017, Pages 17-28

  Cellamare, Matteo ^a,b   Ventz, Steffen ^a,c   Baudin, Elisabeth ^d   Mitnick, Carole D ^e,f   Trippa, Lorenzo ^a  

^a HARVARD SCHOOL OF PUBLIC HEALTH   (United States)

^b SAPIENZA UNIVERSITY OF ROME   (Italy)

^c University of Rhode Island   (United States)

^d EPICENTRE   (France)

^e HARVARD MEDICAL SCHOOL   (United States)

^f PARTNERS IN HEALTH   (United States)

Author keywords

adaptive designs; Bayesian analysis; clinical trial; preliminary outcomes; Tuberculosis

Indexed keywords

ARTICLE; BAYESIAN RESPONSE ADAPTIVE RANDOMIZATION; CONTROLLED STUDY; EFFECT SIZE; EXPERIMENTAL DESIGN; HUMAN; HYPOTHESIS; INTERMETHOD COMPARISON; MULTIDRUG RESISTANT TUBERCULOSIS; PHASE 3 CLINICAL TRIAL (TOPIC); RANDOMIZATION; RANDOMIZED CONTROLLED TRIAL (TOPIC); SAMPLE SIZE; SIMULATION; THERAPY EFFECT; TREATMENT OUTCOME; ADAPTIVE CLINICAL TRIAL (TOPIC); ALGORITHM; BAYES THEOREM; COMPUTER SIMULATION; METHODOLOGY;

BEDAQUILINE; NITROIMIDAZOLE DERIVATIVE; OPC-67683; OXAZOLE DERIVATIVE; QUINOLINE DERIVATIVE; TUBERCULOSTATIC AGENT;

ADAPTIVE CLINICAL TRIALS AS TOPIC; ALGORITHMS; ANTITUBERCULAR AGENTS; BAYES THEOREM; COMPUTER SIMULATION; DIARYLQUINOLINES; HUMANS; NITROIMIDAZOLES; OXAZOLES; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN; TREATMENT OUTCOME; TUBERCULOSIS, MULTIDRUG-RESISTANT;

EID: 85012100244     PISSN: 17407745     EISSN: 17407753     Source Type: Journal    
DOI: 10.1177/1740774516665090     Document Type: Article

References (51)

1. World Health Organization (WHO). Global tuberculosis report 2015. Geneva: WHO, 2015
2. Brigden G, Nyang'wa B-T, du Cros P, et al. Principles for designing future regimens for multidrug-resistant tuberculosis. Bull World Health Organ 2014; 92: 68-74
3. Breitstein J, Spigelman M,. The role of product development partnerships in advancing the development of drugs for unmet needs. Clin Pharmacol Ther 2013; 93: 468-470
4. Bratton DJ, Phillips PP, Parmar MK,. A multi-arm multi-stage clinical trial design for binary outcomes with application to tuberculosis. BMC Med Res Methodol 2013; 13: 139
5. Horsburgh CR, Shea KM, Phillips P, et al. Randomized clinical trials to identify optimal antibiotic treatment duration. Trials 2013; 14: 88
6. Barker A, Sigman C, Kelloff G, et al. I-SPY 2: An adaptive breast cancer trial design in the setting of neoadjuvant chemotherapy. Clin Pharmacol Ther 2009; 86: 97-100
7. Zhou X, Liu S, Kim ES, et al. Bayesian adaptive design for targeted therapy development in lung cancer-a step toward personalized medicine. Clin Trials 2008; 5: 181-193
8. Connor JT, Elm JJ, Broglio KR,. ESETT and ADAPT-IT investigators. Bayesian adaptive trials offer advantages in comparative effectiveness trials: an example in status epilepticus. J Clin Epidemiol 2013; 66: S130-S137
9. Collins SP, Lindsell CJ, Pang PS, et al. Bayesian adaptive trial design in acute heart failure syndromes: moving beyond the mega trial. Am Heart J 2012; 164: 138-145
10. Berry DA,. Adaptive clinical trials: the promise and the caution. J Clin Oncol 2011; 29: 606-609
11. Trippa L, Wen PY, Parmigiani G, et al. Combining progression-free survival and overall survival as a novel composite endpoint for glioblastoma trials. Neuro Oncol 2015; 17: 1106-1113
12. Berry SM, Carlin BP, Lee JJ, et al. Bayesian adaptive methods for clinical trials. Boca Raton, FL: CRC Press, 2010
13. Svensson EM, Aweeka F, Park JG, et al. Model-based estimates of the effects of efavirenz on bedaquiline pharmacokinetics and suggested dose adjustments for patients coinfected with HIV and tuberculosis. Antimicrob Agents Chemother 2013; 57: 2780-2787
14. Svensson EM, Dooley KE, Karlsson MO,. Impact of lopinavir-ritonavir or nevirapine on bedaquiline exposures and potential implications for patients with tuberculosis-HIV coinfection. Antimicrob Agents Chemother 2014; 58: 6406-6412
15. Van Heeswijk R, Dannemann B, Hoetelmans R,. Bedaquiline: a review of human pharmacokinetics and drug-drug interactions. J Antimicrob Chemother 2014; 69: 2310-2318
16. Dooley KE, Park JG, Swindells S, et al. Safety, tolerability, and pharmacokinetic interactions of the antituberculous agent TMC207 (bedaquiline) with efavirenz in healthy volunteers: AIDS Clinical Trials Group Study A5267. J Acquir Immune Defic Syndr 2012; 59: 455-462
17. Falzon D, Jaramillo E, Schünemann H, et al. WHO guidelines for the programmatic management of drug-resistant tuberculosis: 2011 update. Eur Respir J 2011; 38: 516-528
18. Diacon AH, Pym A, Grobusch MP, et al. Multidrug-resistant tuberculosis and culture conversion with bedaquiline. New Engl J Med 2014; 371: 723-732
19. Johnston JC, Shahidi NC, Sadatsafavi M, et al. Treatment outcomes of multidrug-resistant tuberculosis: a systematic review and meta-analysis. PLoS ONE 2009; 4: e6914
20. Ahuja SD, Ashkin D, Avendano M, et al. Multidrug resistant pulmonary tuberculosis treatment regimens and patient outcomes: an individual patient data meta-analysis of 9,153 patients. PLoS Med 2012; 9: e1001300
21. Orenstein EW, Basu S, Shah NS, et al. Treatment outcomes among patients with multidrug-resistant tuberculosis: systematic review and meta-analysis. Lancet Infect Dis 2009; 9: 153-161
22. Skripconoka V, Danilovits M, Pehme L, et al. Delamanid improves outcomes and reduces mortality in multidrug-resistant tuberculosis. Eur Respir J 2013; 41: 1393-1400
23. Becerra MC, Appleton SC, Franke MF, et al. Recurrence after treatment for pulmonary multidrug-resistant tuberculosis. Clin Infect Dis 2010; 51: 709-711
24. Food & Drug Administration. Draft Guidance: Pulmonary tuberculosis: developing drugs for treatment. Washington DC, USA: FDA, 2013
25. Yin G, Chen N, Lee JJ,. Phase II trial design with Bayesian adaptive randomization and predictive probability. J R Stat Soc Ser C Appl Stat 2012; 61: 219-235
26. Trippa L, Lee EQ, Wen PY, et al. Bayesian adaptive randomized trial design for patients with recurrent glioblastoma. J Clin Oncol 2012; 30: 3258-3263
27. Thall PF, Wathen JK,. Practical Bayesian adaptive randomisation in clinical trials. Eur J Cancer 2007; 43: 859-866
28. Alexander BM, Wen PY, Trippa L, et al. Biomarker-based adaptive trials for patients with glioblastoma-lessons from I-SPY 2. Neuro Oncol 2013; 15: 972-978
29. Wason J, Trippa L,. A comparison of Bayesian adaptive randomization and multi-stage designs for multi-arm clinical trials. Stat Med 2014; 33: 2206-2221
30. Ventz S, Trippa L,. Bayesian designs and the control of frequentist characteristics: A practical solution. Biometrics 2015; 71: 218-226
31. Rosenberger WF, Hu F,. Bootstrap methods for adaptive designs. Stat Med 1999; 18: 1757-1767
32. Bowden J, Trippa L,. Unbiased estimation for response adaptive clinical trials. Stat Methods Med Res. Epub ahead of print 11 2015. DOI: 10.1177/0962280215597716
33. Trippa L, Rosner GL, Müller P,. Bayesian enrichment strategies for randomized discontinuation trials. Biometrics 2012; 68: 203-211
34. Anderson JR, High R,. Alternatives to the standard Fleming, Harrington, and O'Brien futility boundary. Clin Trials 2011; 8: 270-276
35. Food and Drug Administration. Draft guidance for industry: adaptive design clinical trials for drugs and biologics. Washington DC, USA: FDA, 2010. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm201790.pdf
36. Simon R,. Adaptive treatment assignment methods and clinical trials. Biometrics 1977; 33: 743-749
37. Korn EL, Freidlin B,. Outcome-adaptive randomization: Is it useful? J Clin Oncol 2011; 29: 771-776
38. Thall P, Fox P, Wathen J,. Statistical controversies in clinical research: scientific and ethical problems with adaptive randomization in comparative clinical trials. Ann Oncol 2015; 26: 1621-1628
39. Hey SP, Kimmelman J,. Are outcome-adaptive allocation trials ethical? Clin Trials 2015; 12: 102-106
40. Berry DA,. Commentary on Hey and Kimmelman. Clin Trials 2015; 12: 107-109
41. Buyse M,. Commentary on Hey and Kimmelman. Clin Trials 2015; 12: 119-121
42. Joffe S, Ellenberg SS,. Commentary on Hey and Kimmelman. Clin Trials 2015; 12: 116-118
43. Korn EL, Freidlin B,. Commentary on Hey and Kimmelman. Clin Trials 2015; 12: 122-124
44. Lee JJ,. Commentary on Hey and Kimmelman. Clin Trials 2015; 12: 110-112
45. Saxman SB,. Commentary on Hey and Kimmelman. Clin Trials 2015; 12: 113-115
46. Jindani A, Hatherill M, Charalambous S, et al. A multicentre randomized clinical trial to evaluate high-dose rifapentine with a quinolone for treatment of pulmonary TB: The RIFAQUIN trial. Atlanta, GA: Program and abstracts of the 20th Conference on Retroviruses and Opportunistic Infections, 2013
47. Jindani A, Harrison TS, Nunn AJ, et al. High-dose rifapentine with moxifloxacin for pulmonary tuberculosis. New Engl J Med 2014; 371: 1599-1608
48. Rustomjee R, Lienhardt C, Kanyok T, et al. A Phase II study of the sterilising activities of ofloxacin, gatifloxacin and moxifloxacin in pulmonary tuberculosis. Int J Tuberc Lung Dis 2008; 12: 128-138
49. Altman DG, Royston JP,. The hidden effect of time. Stat Med 1988; 7: 629-637
50. Gler MT, Skripconoka V, Sanchez-Garavito E, et al. Delamanid for multidrug-resistant pulmonary tuberculosis. New Engl J Med 2012; 366: 2151-2160
51. World Health Organization. Global tuberculosis report 2014. Geneva: WHO, 2014