A seamless Phase IIB/III adaptive outcome trial: Design rationale and implementation challenges

Clinical Trials

Volumn 12, Issue 1, 2015, Pages 84-90

  Chen, Y H Joshua ^a   Gesser, Richard ^a,b   Luxembourg, Alain ^a  

^a MERCK RESEARCH LABORATORIES   (United States)

^b SANOFI PASTEUR   (France)

Author keywords

Human; Papillomavirus; Vaccine

Indexed keywords

WART VIRUS VACCINE; CANCER VACCINE;

ADULT; ARTICLE; DRUG DEVELOPMENT; DRUG EFFICACY; FEMALE; FOLLOW UP; HUMAN; IMMUNOGENICITY; INTERPERSONAL COMMUNICATION; MAJOR CLINICAL STUDY; OUTCOME ASSESSMENT; PAPILLOMAVIRUS INFECTION; PRIORITY JOURNAL; UTERINE CERVIX CANCER; WART VIRUS; ADOLESCENT; AGED; CLINICAL TRIAL; CONTROLLED STUDY; DRUG APPROVAL; METHODOLOGY; PHASE 2 CLINICAL TRIAL; PHASE 3 CLINICAL TRIAL; PROCEDURES; RANDOMIZED CONTROLLED TRIAL; UTERINE CERVICAL NEOPLASMS; YOUNG ADULT;

ADOLESCENT; ADULT; AGED; CANCER VACCINES; DRUG APPROVAL; FEMALE; HUMANS; PAPILLOMAVIRUS VACCINES; RESEARCH DESIGN; UTERINE CERVICAL NEOPLASMS; YOUNG ADULT;

EID: 84922361177     PISSN: 17407745     EISSN: 17407753     Source Type: Journal    
DOI: 10.1177/1740774514552110     Document Type: Article

References (24)

1. US Food and Drug Administration. GARDASIL® Highlights of prescribing information, http://www.fda.gov/downloads/biologicsbloodvaccines/vaccines/approvedproducts/ucm111263.pdf (accessed 18 March 2014).
2. De Sanjose S, Quint WG, Alemany L, et al. Human papillomavirus genotype attribution in invasive cervical cancer: a retrospective cross-sectional worldwide study. Lancet Oncol 2010; 11: 1048-1056.
3. Serrano B, Alemany L, Tous S, et al. Potential impact of a nine-valent vaccine in human papillomavirus related cervical disease. Infect Agent Cancer 2012; 7: 38.
4. The FUTURE II Study Group. Quadrivalent vaccine against human papillomavirus to prevent high-grade cervical lesions. N Engl J Med 2007; 356: 1915-1927.
5. Garland SM, Hernandez-Avila M, Wheeler CM, et al. Quadrivalent vaccine against human papillomavirus to prevent anogenital diseases. N Engl J Med 2007; 356: 1928-1943.
6. Joura EA, Leodolter S, Hernandez-Avila M, et al. Efficacy of a quadrivalent prophylactic human papillomavirus (types 6, 11, 16 and 18) L1 virus-like-particle vaccine against high-grade vulval and vaginal lesions: a combined analysis of three clinical trials. Lancet 2007; 369: 1693-1702.
7. Palefsky JM, Giuliano AR, Goldstone S, et al. HPV vaccine against anal HPV infection and anal intraepithelial neoplasia. N Engl J Med 2011; 365: 1576-1585.
8. Temple R. Current definitions of phases of investigation and the role of the FDA in the conduct of clinical trials. Am Heart J 2000; 139: S133-S135.
9. Thall PF, Simon R and Ellenberg S. A two-stage design for choosing among several experimental treatments and a control in clinical trials. Biometrics 1989; 45: 537-547.
10. Liu Q and Pledger GW. Phase 2 and 3 combination designs to accelerate drug development. J Am Stat Assoc 2005; 100: 493-502.
11. Chen YHJ and Anderson KM. A "MiniPool" approach for combined Phase IIB/III trial in patients with lifethreatening disease: an application of the closed testing principle. Stat Biopharm Res 2010; 2: 62-71.
12. Bretz F, Schmidli H, König F, et al. Confirmatory seamless phase II/III clinical trials with hypotheses selection at interim: general concepts. Biom J 2006; 48: 623-634.
13. Posch M, König F, Branson M, et al. Testing and estimation in flexible group sequential designs with adaptive treatment selection. Stat Med 2005; 24: 3697-3714.
14. Jennison C and Turnbull BW. Adaptive seamless designs: selection and prospective testing of hypotheses. J Biopharm Stat 2007; 17: 1135-1161.
15. Wang SJ, Hung HMJ and O'Neill RT. Impacts of type I error rate with inappropriate use of learn for confirm in adaptive designs. Biom J 2010; 52: 798-810.
16. US Food and Drug Administration. U.S. Food and Drug Administration draft guidance for industry on adaptive design clinical trials for drugs and biologics. 25 February 2010, www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/ Guidances/ UCM201790.pdf (accessed 17 June 2014).
17. Joura E. Efficacy and immunogenicity of a novel 9-valent HPV L1 virus-like particle vaccine in 16- to 26-year-old women. In: EUROGIN 2013, 3-6 November, http://www.eurogin.com/2013/images/pdf/EUROGIN-2013-Abstracts-Part-1.pdf (abstract at page 102) (accessed 16June 2014).
18. Chan ISF and Bohidar NR. Exact power and sample size for vaccine efficacy studies. Commun Stat Theory Methods 1998; 27: 1305-1322.
19. Clopper C and Pearson ES. The use of confidence or fiducial limits illustrated in the case of the binomial. Biometrika 1934; 26: 404-413.
20. Stanley M, Pinto LA and Trimble C. Human papillomavirus-immune responses. Vaccine 2012; 30: F83-F87.
21. Villa LL, Ault KA, Giuliano AR, et al. Immunologic responses following administration of a vaccine targeting human papillomavirus Types 6, 11, 16, and 18. Vaccine 2006; 24: 5571-5583.
22. Agresti A. Categorical data analysis. 2nd ed.Hoboken, NJ: John Wiley & Sons, Inc., 2002.
23. Chan ISF, Li P, Sun JX, et al. Multiplicity adjustment in seamless Phase II/III adaptive trials with a biomarker for dose selection. In: Presented at ICSA-ISBS joint conference, Bethesda, MD, 9-12 June 2013.
24. Roberts C, Green T, Hess E, et al. Development of a human papillomavirus competitive luminex immunoassay for nine HPV types. Hum Vaccin Immunother. Epub ahead of print 23 June 2014. DOI: 10.4161/hv.29205.