Developing a Bayesian adaptive design for a phase I clinical trial: a case study for a novel HIV treatment

Statistics in Medicine

Volumn 36, Issue 5, 2017, Pages 754-771

  Mason, Alexina J ^a   Gonzalez Maffe, Juan ^b   Quinn, Killian ^c   Doyle, Nicki ^c   Legg, Ken ^c   Norsworthy, Peter ^c   Trevelion, Roy ^d   Winston, Alan ^c   Ashby, Deborah ^b  

^a LONDON SCHOOL OF HYGIENE AND TROPICAL MEDICINE   (United Kingdom)

^b IMPERIAL COLLEGE LONDON   (United Kingdom)

^c IMPERIAL COLLEGE LONDON   (United Kingdom)

^d c o HIV i Base   (Afghanistan)

Author keywords

Bayesian adaptive designs; continual reassessment method; dose finding studies; fusion inhibitor; HIV clinical trials; phase I

Indexed keywords

HUMAN IMMUNODEFICIENCY VIRUS FUSION INHIBITOR; PEPTIDE DERIVATIVE; GLYCOPROTEIN GP 41; PEPTIDE C34; PEPTIDE FRAGMENT;

ARTICLE; BAYESIAN ADAPTIVE DESIGN; CASE STUDY; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; INTERMETHOD COMPARISON; PHASE 1 CLINICAL TRIAL (TOPIC); PROCESS DEVELOPMENT; RULE BASED DESIGN; SIMULATION; STUDY DESIGN; BAYES THEOREM; BIOASSAY; PROCEDURES;

BAYES THEOREM; CLINICAL TRIALS, PHASE I AS TOPIC; ENDPOINT DETERMINATION; HIV ENVELOPE PROTEIN GP41; HIV FUSION INHIBITORS; HIV INFECTIONS; HUMANS; PEPTIDE FRAGMENTS;

EID: 85006010367     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.7169     Document Type: Article

References (26)

1. Storer BE. Design and analysis of phase I clinical trials. Biometrics 1989; 45:925–937.
2. Le Tourneau C, Lee JJ, Siu LL. Dose escalation methods in phase I cancer clinical trials. Journal of the National Cancer Institute 2009; 101:708–720.
3. Berry SM, Carlin BP, Lee JJ, Muller P. Bayesian Adaptive Methods for Clinical Trials. Chapman & Hall, 2011.
4. O'Quigley J, Pepe M, Fisher L. Continual reassessment method: a practical design for phase 1 clinical trials in cancer. Biometrics 1990; 46:33–48.
5. Korn EL, Midthune D, Chenn TT, Rubinstein LV, Christian MC, Simon RM. A comparison of two phase I trial designs. Statistics in Medicine 1994; 13:1799–1806.
6. Faries D. Practical modifications of the continual reassessment method for phase I cancer clinical trials. Journal of Biopharmaceutical Statistics 1994; 4:147–164.
7. Babb J, Rogatko A, Zacks S. Cancer phase I clinical trials: efficient dose escalation with overdose control. Statistics in Medicine 1998; 17:1103–1120.
8. Elkind MSV, Sacco RL, MacArthur RB, Peerschke E, Neils G, Andrews H, Stillman J, Corporan T, Leifer D, Liu R, Cheung K. High-dose Lovastatin for acute ischemic stroke: results of the phase I dose escalation neuroprotection with statin therapy for acute recovery trial (NeuSTART). Cerebrovascular Diseases 2009; 28:266–275.
9. Kairalla JA, Coffey CS, Thomann MA, Muller KE. Adaptive trial designs: a review of barriers and opportunities. Trials 2012; 13:145.
10. Loke YC, Tan SB, Cai Y, Machin D. A Bayesian dose finding design for dual endpoint phase I trials. Statistics in Medicine 2006; 25:3–22.
11. Grieve AP, Krams M. ASTIN: A Bayesian adaptive dose-response trial in acute stroke. Clinical Trials 2005; 2:340–351.
12. O'Quigley J, Hughes MD, Fenton T. Dose-finding designs for HIV studies. Biometrics 2001; 57:1018–1029.
13. http://www.unaids.org/en/dataanalysis/datatools/aidsinfo/.,J.U.N.P.o.H.A.U.A.
14. Porter K, Johnson AM, Phillips AN, Darbyshire JH. The practical significance of potential biases in estimates of the AIDS incubation periods distribution in the UK Register of HIV Seroconverters. AIDS 1999; 13:1943–1951.
15. Bhaskaran K, Hamouda O, Sannes M, Boufassa F, Johnson AM, Lambert PC, Porter K. Changes in the risk of death after HIV seroconversion compared with mortality in the general population. Journal of the American Medical Association 2008; 300:51–59.
16. May M, Gompels M, Delpech V, Porter K, Post F, Johnson M, Dunn D, Palfreeman A, Gilson R, Gazzard B, Hill T, Walsh J, Fisher M, Orkin C, Ainsworth J, Bansi L, Phillips A, Leen C, Nelson M, Anderson J, Sabin C. Impact of late diagnosis and treatment on life expectancy in people with HIV-1: UK collaborative HIV Cohort (UK CHIC) study. BMJ 2011; 343:d6016.
17. Hasse B, Ledergerber B, Furrer H, Battegay M, Hirschel B, Cavassini M, Bertisch B, Bernasconi E, Weber R. Morbidity and aging in HIV-infected persons: the Swiss HIV cohort study. Clinical Infectious Diseases 2011; 53:1130–1139.
18. Schoen JC, Erlandson KM, Anderson PL. Clinical pharmacokinetics of antiretroviral drugs in older persons. Expert Opinion on Drug Metabolism & Toxicology 2013; 9:573–588.
19. Fung HB, Guo Y. Enfuvirtide: a fusion inhibitor for the treatment of HIV infection. Clinical Therapeutics 2004; 26:352–378.
20. Ingallinella P, Bianchi E, Ladwa NA, Wang YJ, Hrin R, Veneziano M, Bonelli F, Ketas TJ, Moore JP, Miller MD, Pessi A. Addition of a cholesterol group to an HIV-1 peptide fusion inhibitor dramatically increases its antiviral potency. Proceedings of the National Academy of Sciences of the United States of America 2009; 106:5801–5806.
21. Resche-Rigon M, Zohar S, Chevret S. Adaptive designs for dose-finding in non-cancer phase II trials: influence of early unexpected outcomes. Clinical trials 2008; 5:595–606.
22. Garrett-Mayer E. The continual reassessment method for dose-finding studies: a tutorial. Clinical trials 2006; 3:57–71.
23. R Core Team. R: a language and environment for statistical computing. R Foundation for Statistical Computing: Vienna, Austria, 2013. http://www.R-project.org.
24. Lunn DJ, Thomas A, Best NG, Spiegelhalter DJ, WinBUGS - A. Bayesian modelling framework: concepts, structure, and extensibility. Statistics and Computing 2000; 10:325–337.
25. Lunn DJ, Spiegelhalter DJ, Thomas A, Best NG. The BUGS project: evolution, critique and future directions. Statistics in Medicine 2009; 28:3049–3067.
26. Ji Y, Feng L, Liu P, Shpall EJ, Kebriaei P, Champlin R, Berry D, Cooper LJN. Bayesian continual reassessment method for dose-finding trials infusing T cells with limited sample size. Journal of Biopharmaceutical Statistics 2012; 22:1206–1219.