Gillespie SH, Crook AM, McHugh TD, Mendel CM, Meredith SK, Murray SR, et al. Four-month moxifloxacin-based regimens for drug-sensitive tuberculosis. N Engl J Med. 2014;371(17):1577–87.
Article
PubMed
PubMed Central
Google Scholar
World Health Organization. Global Tuberculosis Report 2015. Geneva: WHO; 2015.
Google Scholar
Boeree MJ, Diacon AH, Dawson R, Narunsky K, du Bois J, Venter A, et al. A dose-ranging trial to optimize the dose of rifampin in the treatment of tuberculosis. Am J Respir Crit Care Med. 2015;191(9):1058–65.
Article
CAS
PubMed
Google Scholar
Dooley KE, Obuku EA, Durakovic N, Belitsky V, Mitnick C, Nuermberger EL. World Health Organization group 5 drugs for the treatment of drug-resistant tuberculosis: unclear efficacy or untapped potential? J Infect Dis. 2012;207(9):1352–8.
Article
PubMed
PubMed Central
Google Scholar
Dooley KE, Mitnick CD, Ann DeGroote M, Obuku E, Belitsky V, Hamilton CD, et al. Old drugs, new purpose: retooling existing drugs for optimized treatment of resistant tuberculosis. Clin Infect Dis. 2012;55(4):572–81.
Article
PubMed
PubMed Central
Google Scholar
Dawson R, Narunsky K, Carman D, Gupte N, Whitelaw A, Efron A, et al. Two-stage activity-safety study of daily rifapentine during intensive phase treatment of pulmonary tuberculosis. Int J Tuberc Lung Dis. 2015;19(7):780–6.
Article
CAS
PubMed
Google Scholar
Phillips PPJ, Gillespie SH, Boeree M, Heinrich N, Aarnoutse R, McHugh T, et al. Innovative trial designs are practical solutions for improving the treatment of tuberculosis. J Infect Dis. 2012;205 Suppl 2:S250–7.
Article
PubMed
Google Scholar
Jindani A, Harrison TS, Nunn AJ, Phillips PP, Churchyard GJ, Charalambous S, et al. High-dose rifapentine with moxifloxacin for pulmonary tuberculosis. N Engl J Med. 2014;371(17):1599–608.
Article
PubMed
PubMed Central
Google Scholar
Merle CS, Fielding K, Sow OB, Gninafon M, Lo MB, Mthiyane T, et al. A four-month gatifloxacin-containing regimen for treating tuberculosis. N Engl J Med. 2014;371(17):1588–98.
Article
PubMed
Google Scholar
Medical Research Council. Streptomycin treatment of pulmonary tuberculosis. Br Med J. 1948;2(4582):769–82.
Article
Google Scholar
Cochrane A. 1931-1971: A critical review, with particular reference to the medical profession. In: Teeling-Smith G, Wells N, editors. Medicines for the Year 2000. London: Office of Health Economics; 1979. p. 1–11.
Google Scholar
Fox W, Ellard GA, Mitchison DA. Studies on the treatment of tuberculosis undertaken by the British Medical Research Council tuberculosis units, 1946-1986, with relevant subsequent publications. Int J Tuberc Lung Dis. 1999;3(10 Suppl 2):S231–279.
CAS
PubMed
Google Scholar
US FDA Priftin (Rifapentine) Drug Approval Package. [http://www.accessdata.fda.gov/drugsatfda_docs/nda/2000/21024S5_Priftin.cfm]. Accessed 21 Mar 2016.
Cox E, Laessig K. FDA approval of bedaquiline--the benefit-risk balance for drug-resistant tuberculosis. N Engl J Med. 2014;371(8):689–91.
Article
CAS
PubMed
Google Scholar
Ahuja SD, Ashkin D, Avendano M, Banerjee R, Bauer M, Bayona JN, 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(8):e1001300.
Article
CAS
PubMed
PubMed Central
Google Scholar
Pocock SJ. Clinical trials: a practical approach. Chichester: Wiley; 1983.
Google Scholar
Suntharalingam G, Perry MR, Ward S, Brett SJ, Castello-Cortes A, Brunner MD, et al. Cytokine storm in a phase 1 trial of the anti-CD28 monoclonal antibody TGN1412. N Engl J Med. 2006;355(10):1018–28.
Article
CAS
PubMed
Google Scholar
Piantadosi S. Clinical trials: a methodologic perspective. 2nd ed. Hoboken: Wiley-Interscience; 2005.
Book
Google Scholar
Diacon AH, Dawson R, Hanekom M, Narunsky K, Maritz SJ, Venter A, et al. Early bactericidal activity and pharmacokinetics of PA-824 in smear-positive tuberculosis patients. Antimicrob Agents Chemother. 2010;54(8):3402–7.
Article
CAS
PubMed
PubMed Central
Google Scholar
Diacon AH, Dawson R, du Bois J, Narunsky K, Venter A, Donald PR, et al. Phase II dose-ranging trial of the early bactericidal activity of PA-824. Antimicrob Agents Chemother. 2012;56(6):3027–31.
Article
CAS
PubMed
PubMed Central
Google Scholar
Diacon AH, Dawson R, von Groote-Bidlingmaier F, Symons G, Venter A, Donald PR, et al. 14-day bactericidal activity of PA-824, bedaquiline, pyrazinamide, and moxifloxacin combinations: a randomised trial. Lancet. 2012;380(9846):986–93.
Article
CAS
PubMed
Google Scholar
Diacon AH, Dawson R, von Groote-Bidlingmaier F, Symons G, Venter A, Donald PR, et al. Bactericidal activity of pyrazinamide and clofazimine alone and in combinations with pretomanid and bedaquiline. Am J Respir Crit Care Med. 2015;191(8):943–53.
Article
CAS
PubMed
Google Scholar
Dawson R, Diacon AH, Everitt D, van Niekerk C, Donald PR, Burger DA, et al. Efficiency and safety of the combination of moxifloxacin, pretomanid (PA-824), and pyrazinamide during the first 8 weeks of antituberculosis treatment: a phase 2b, open-label, partly randomised trial in patients with drug-susceptible or drug-resistant pulmonary tuberculosis. Lancet. 2015;385(9979):1738–47.
Article
CAS
PubMed
Google Scholar
Dorman SE, Goldberg S, Stout JE, Muzanyi G, Johnson JL, Weiner M, et al. Substitution of rifapentine for rifampin during intensive phase treatment of pulmonary tuberculosis: study 29 of the tuberculosis trials consortium. J Infect Dis. 2012;206(7):1030–40.
Article
CAS
PubMed
Google Scholar
Mitchison DA. The action of antituberculosis drugs in short-course chemotherapy. Tubercle. 1985;66(3):219–25.
Article
CAS
PubMed
Google Scholar
Prideaux B, Via LE, Zimmerman MD, Eum S, Sarathy J, O'Brien P, Chen C, Kaya F, Weiner DM, Chen PY, et al. The association between sterilizing activity and drug distribution into tuberculosis lesions. Nat Med. 2015;21(10):1223–7.
Article
CAS
PubMed
Google Scholar
Wallis RS, Nacy C. Early bactericidal activity of new drug regimens for tuberculosis. Lancet. 2013;381(9861):111–2.
Article
PubMed
Google Scholar
Rustomjee R, Diacon AH, Allen J, Venter A, Reddy C, Patientia RF, et al. Early bactericidal activity and pharmacokinetics of the diarylquinoline TMC207 in treatment of pulmonary tuberculosis. Antimicrob Agents Chemother. 2008;52(8):2831–5.
Article
CAS
PubMed
PubMed Central
Google Scholar
Diacon AH, Dawson R, Von Groote-Bidlingmaier F, Symons G, Venter A, Donald PR, et al. Randomized dose-ranging study of the 14-day early bactericidal activity of bedaquiline (TMC207) in patients with sputum microscopy smear-positive pulmonary tuberculosis. Antimicrob Agents Chemother. 2013;57(5):2199–203.
Article
CAS
PubMed
PubMed Central
Google Scholar
Diacon AH, Pym A, Grobusch MP, de los Rios JM, Gotuzzo E, Vasilyeva I, et al. Multidrug-resistant tuberculosis and culture conversion with bedaquiline. N Engl J Med. 2014;371(8):723–32.
Article
PubMed
Google Scholar
Tang S, Yao L, Hao X, Liu Y, Zeng L, Liu G, et al. Clofazimine for the treatment of multidrug-resistant tuberculosis: prospective, multicenter, randomized controlled study in China. Clin Infect Dis. 2015;60(9):1361–7.
PubMed
Google Scholar
Phillips PP, Mendel CM, Burger DA, Crook A, Nunn AJ, Dawson R, et al. Limited role of culture conversion for decision-making in individual patient care and for advancing novel regimens to confirmatory clinical trials. BMC Med. 2016;14(1):19.
Article
PubMed
PubMed Central
Google Scholar
Phillips PPJ, Fielding K, Nunn AJ. An evaluation of culture results during treatment for tuberculosis as surrogate endpoints for treatment failure and relapse. PLoS One. 2013;8(5):e63840.
Article
CAS
PubMed
PubMed Central
Google Scholar
Wallis RS, Wang C, Doherty TM, Onyebujoh P, Vahedi M, Laang H, et al. Biomarkers for tuberculosis disease activity, cure, and relapse. Lancet Infect Dis. 2010;10(2):68–9.
Article
PubMed
Google Scholar
Phillips PP, Davies GR, Mitchison DA. Biomarkers for tuberculosis disease activity, cure, and relapse. [Correspondence]. Lancet Infect Dis. 2010;10(2):69–70. author reply 70-61.
Article
PubMed
Google Scholar
Hay M, Thomas DW, Craighead JL, Economides C, Rosenthal J. Clinical development success rates for investigational drugs. Nat Biotechnol. 2014;32(1):40–51.
Article
CAS
PubMed
Google Scholar
Treatment Action Group, Frick M. 2015 report on tuberculosis research funding trends, 2005–2014: a decade of data. 2015. http://www.treatmentactiongroup.org/tbrd2015. Accessed 21 Mar 2016.
Boeree M, Hoelscher M; The PanACEA consortium. High-dose rifampin, SQ109 and moxifloxacin for treating tb: The PanACEA MAMS-TB Trial. In: 2015 Conference on retroviruses and opportunistic infection (CROI), 23–26 February 2015. Seattle, Washington: CROI Foundation; 2015.
Nunn AJ, Phillips PPJ, Mitchison DA. Timing of relapse in short-course chemotherapy trials for tuberculosis [Short communication]. Int J Tuberc Lung Dis. 2010;14:241–242(242).
CAS
PubMed
Google Scholar
Dorman SE, Savic RM, Goldberg S, Stout JE, Schluger N, Muzanyi G, et al. Daily rifapentine for treatment of pulmonary tuberculosis. A randomized, dose-ranging trial. Am J Respir Crit Care Med. 2015;191(3):333–43.
Article
CAS
PubMed
Google Scholar
Machin D. Sample size tables for clinical studies. 3rd ed. Wiley-Blackwell: Chichester; 2008.
Book
Google Scholar
Berry SM. Bayesian adaptive methods for clinical trials. Boca Raton: CRC Press; 2011.
Google Scholar
Wallis RS, Peppard T, Hermann D. Month 2 culture status and treatment duration as predictors of recurrence in pulmonary tuberculosis: model validation and update. PLoS One. 2015;10(4):e0125403.
Article
PubMed
PubMed Central
Google Scholar
Parker CC, Sydes MR, Mason MD, Clarke NW, Aebersold D, de Bono JS, et al. Prostate radiotherapy for men with metastatic disease: a new comparison in the STAMPEDE trial. Clin Oncol (R Coll Radiol). 2013;25(5):318–20.
Article
CAS
Google Scholar
O'Hagan A, Stevens JW, Campbell MJ. Assurance in clinical trial design. Pharm Stat. 2005;4(3):187–201.
Article
Google Scholar
PUSH, PULL, POOL: Accelerating innovation and access to medicines for tuberculosis. [http://www.msfaccess.org/3Ps-project]. Accessed 21 Mar 2016.
Ventz S, Trippa L. Bayesian designs and the control of frequentist characteristics: a practical solution. Biometrics. 2014;71(1):218–26.
Article
PubMed
Google Scholar