Challenges of phase III study design for trials of new drug regimens for the treatment of TB

Future Medicinal Chemistry

Volumn 2, Issue 8, 2010, Pages 1273-1282

  Phillips, Patrick P J ^a   Nunn, Andrew J ^a  

^a MEDICAL RESEARCH COUNCIL   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

AMINOSALICYLIC ACID; ANTI HUMAN IMMUNODEFICIENCY VIRUS AGENT; ETHAMBUTOL; ETHIONAMIDE; ISONIAZID; NONNUCLEOSIDE REVERSE TRANSCRIPTASE INHIBITOR; PROTIONAMIDE; PYRAZINAMIDE; RIFAMPICIN; RIFAPENTINE; STREPTOMYCIN; THIOACETAZONE;

ANTIBIOTIC SENSITIVITY; CLINICAL TRIAL; DRUG EFFICACY; DRUG TREATMENT FAILURE; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; MORTALITY; PRIORITY JOURNAL; RECURRENT DISEASE; REINFECTION; REVIEW; TREATMENT DURATION; TREATMENT OUTCOME; TREATMENT RESPONSE; TUBERCULOSIS;

ANTITUBERCULAR AGENTS; CLINICAL TRIALS, PHASE III AS TOPIC; HUMANS; TUBERCULOSIS;

EID: 77956043009     PISSN: 17568919     EISSN: None     Source Type: Journal    
DOI: 10.4155/fmc.10.221     Document Type: Review

References (38)

1. World Health Organization. Global Tuberculosis Control - Epidemiology, Strategy, Financing. World Health Organization Report, Geneva, Switzerland (2009).
2. 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. 3(10), S231-S279 (1999).
3. East and Central African/British Medical Research Council. Controlled clinical trial of 4 short-course regimens of chemotherapy (three 6-month and one 8-month) for pulmonary tuberculosis. Tubercle. 64(3), 153-166 (1983).
4. Tuberculosis Service/British Medical Research Council Singapore. Clinical trial of six-month and four-month regimens of chemotherapy in the treatment of pulmonary tuberculosis: the results up to 30 months. Tubercle. 62(2), 95-102 (1981).
5. Combs DL, O'Brien RJ, Geiter LJ. USPHS tuberculosis short-course chemotherapy trial 21: effectiveness, toxicity and acceptability. The report of final results. Ann. Intern. Med. 112(6), 397-406 (1990).
6. British Thoracic Society. A controlled trial of 6 months' chemotherapy in pulmonary tuberculosis. Final report: results during the 36 months after the end of chemotherapy and beyond. Br. J. Dis. Chest. 78(4), 330-336 (1984).
7. Algerian working group/British Medical Research Council. Controlled clinical trial comparing a 6-month and a 12-month regimen in the treatment of pulmonary tuberculosis in the Algerian Sahara. Algerian working group/British Medical Research Council cooperative study. Am. Rev. Respir. Dis. 129(6), 921-928 (1984).
8. Fox W. Whither short-course chemotherapy? Br. J. Dis. Chest 75(4), 331-357 (1981).
9. Johnson JL, Hadad DJ, Dietze R et al. Shortening treatment in adults with noncavitary tuberculosis and two-month culture conversion. Am. J. Respir. Crit. Care Med., 18, 558-563 (2009).
10. Ma Z, Lienhardt C, McIlleron H, Nunn AJ, Wang X. Global tuberculosis drug development pipeline: the need and the reality. The Lancet 375(9731), 2100-2109 (2010).
11. Alderson P, Chalmers I. Survey of claims of no effect in abstracts of Cochrane reviews. BMJ 326(7387), 475 (2003).
12. Greene WL, Concato J and Feinstein AR. Claims of equivalence in medical research: are they supported by the evidence? Ann. Intern. Med. 132(9), 715-722, (2000).
13. Brown D, Volkers P, Day S. An introductory note to CHMP guidelines: choice of the noninferiority margin and data monitoring committees. Stat. Med. 25(10), 1623-1627 (2006).
14. Vynnycky E, Fine PE. The natural history of tuberculosis: the implications of age-dependent risks of disease and the role of reinfection. Epidemiol. Infect. 119(2), 183-201 (1997).
15. East African/British Medical Research Council. Controlled clinical trial of five short-course (4-month) chemotherapy regimens in pulmonary tuberculosis. Second report of the 4th study. Am. Rev. Respir. Dis. 123(2), 165-170 (1981).
16. Medical Research Council. Streptomycin treatment of pulmonary tuberculosis. Br. Med. J. 2(4582), 769-782 (1948).
17. Fox W, Sutherland I, Daniels M. A five-year assessment of patients in a controlled trial of streptomycin in pulmonary tuberculosis: report to the tuberculosis chemotherapy trials committee of the medical research council. Q. J. Med. 23(91), 347-366 (1954).
18. East African/British Medical Research Council. Controlled clinical trial of four short-course (6-month) regimens of chemotherapy for treatment of pulmonary tuberculosis. Second report. J. Lancet, 1(7816), 1331-1338 (1973).
19. Aber VR, Allen BW, Mitchison DA, Ayuma P, Edwards EA, Keyes AB. Quality-control in tuberculosis bacteriology. 1. Laboratory studies on isolated positive cultures and the efficiency of direct smear examination. Tubercle. 61(3), 123-133 (1980).
20. Mitchison DA. Treatment of tuberculosis. The Mitchell lecture 1979. J. R. Coll. Physicians Lond. 14(2), 91-99 (1980).
21. Buechner HA. When is pulmonary tuberculosis cured? J. Lancet 319(8287), 1462-1462 (1982).
22. Nunn AJ, Phillips PPJ, Mitchison DA. Timing of relapse in short-course chemotherapy trials for tuberculosis [short communication]. Int. J. Tuberc. Lung Dis. 14, 241-242(2) (2010).
23. Hanna BA, Ebrahimzadeh A, Elliott LB et al. Multicenter evaluation of the BACTEC MGIT 960 system for recovery of mycobacteria. J. Clin. Microbiol. 37(3), 748-752 (1999).
24. Phillips PPJ, Fielding K. Surrogate markers for poor outcome treatment for tuberculosis: results from extensive multi-trial analysis. Int. J. Tuberc. Lung. Dis. 12, S146 (2008).
25. Temple RJ. A regulatory authority's opinion about surrogate endpoints. In: Clinical Measurement in Drug Evaluation. Nimmo GT, Tucker WS (Eds). Wiley, NY, USA, 3-22 (1995).
26. The Evaluation of Surrogate Endpoints. Burzykowski T, Molenberghs G, Buyse ME (Eds). Springer, NY, USA (2005).
27. Kaul S, Diamond GA. Making sense of noninferiority: a clinical and statistical perspective on its application to cardiovascular clinical trials. Prog. Cardiovasc. Dis. 49(4), 284-299 (2007).
28. Wiens BL, Zhao W. The role of intention to treat in analysis of noninferiority studies. Clin. Trials, 4(3), 286-291 (2007).
29. Jindani A, Nunn AJ, Enarson DA. Two 8-month regimens of chemotherapy for treatment of newly diagnosed pulmonary tuberculosis: international multicenter randomized trial. Lancet, 364(9441), 1244-1251 (2004).
30. Treatment of Tuberculosis: Guidelines - 4th Edition. World Health Organization, Geneva WHO/HTM/TB/2009.420 (2009).
31. Burman WJ, Cotton MF, Gibb DM, Walker AS, Vernon AA, Donald PR. Ensuring the Involvement of children in the evaluation of new tuberculosis treatment regimens. PLoS Med. 5(8), e176(2008).
32. Narayanan S, Swaminathan S, Supply P et al. Impact of HIV infection on the recurrence of tuberculosis in South India. J. Infect. Dis. 201(5), 691-703 (2010).
33. Crampin AC, Mwaungulu JN, Mwaungulu FD et al. Recurrent TB: relapse or reinfection? The effect of HIV in a general population cohort in Malawi. AIDS, 24(3), 417-426 (2010).
34. Chaisson RE, Churchyard GJ. Recurrent tuberculosis: relapse, reinfection, and HIV. J. Infect. Dis. 201, 653-655(2010)
35. Guidance for industry noninferiority clinical trials: draft guidance www.fda.gov/downloads/Drugs/ GuidanceComplianceRegulatoryInformation/ Guidances/UCM202140.pdf
36. FDA guidance on conducting noninferiority clinical trials. Points to consider on switching between superiority and noninferiority www.ema.europa.eu/pdfs/human/ ewp/048299en.pdf
37. EMEA guidance on conducting noninferiority clinical trials. Drug approval package for Priftin (Rifapentine) tablets www.accessdata.fda.gov/drugsatfda- docs/ nda/2000/21024S5-Priftin.cfm
38. Centers for Disease Control and Prevention www.cdc.gov/tb/publications/ guidelines/tb- hiv-drugs/rifampin-therapy.htm