Clinical Trials Course (Spring 2012)

(Rice University: STAT 630; UT GSBS: GS01 0813)

Syllabus

Lecture Notes (PowerPoint Files)

(for course use only, not for distribution or any other purposes)

Slides and Material Lectures

[TO BE UPDATED AS THE COURSE GOES ALONG]

Lecture 1: 1/10/12 (Lee 1)

Reference:

Lee, Event Chart, TAS 2000

Lewis, ICH E9, SIM 1999

Lippman, LIT, JNCI 2001

Nottage, Clinical Trial Design, JCO 2002

Greenfield, Historical Highlights of Randomized Controlled Trials, SoCRA SOURCE 2004

Lee, clinical trial designs for anticancer therapies, 2007

Capdeville, Nature Review 2002

Lecture 2: 1/12/12 (Thall 1)

Thall Homework #1

Lecture 3: 1/17/12 (Lee 1)

Reference:

Belmont Report

Lecture 4: 1/19/12 (Lee 2)

Lecture 5: 1/26/12 (Lee 3)

Lecture 6: 1/31/12 (Lee 4)

PhaseIsim

Lecture 7: 2/2/12 (Lee 5)

Lecture 8: 2/7/12 (Hobbs 1)

Lecture 9: 2/9/12 (Lee 5)

ksb1prob.R

Schultz paper

Simon two-stage design paper

Lee and Liu predictive probability paper

PII87.exe

DOSBOX.exe

Lecture 10: 2/14/12 (Lee 6)

Lecture 11: 2/16/12 (Lee 7)

Lecture 12: 2/21/12 (Lee 8)

Lecture 13: 2/23/12 (Hobbs 2)

Dr. Thall’s References

1. Thall PF, Simon R. Practical Bayesian guidelines for phase IIB clinical trials. Biometrics 50: 337-349, 1994.

2. Thall PF, Wooten LH, Tannir N. Monitoring event times in early phase clinical trials: some practical issues. Clinical Trials. 2:467-478, 2005.

3. Thall PF, Simon R, Estey EH. Bayesian sequential monitoring designs for single-arm clinical trials with multiple outcomes. Stat in Medicine 14:357-379, 1995.

4. Thall PF, Sung H-G. Some extensions and applications of a Bayesian strategy for monitoring multiple outcomes in clinical trials. Stat in Medicine, 17:1563-1580, 1998

5. Wathen JK, Thall PF, Cook, JD, Estey EH. Accounting for patient heterogeneity in phase II clinical trials. Stat in Medicine. 27:2802-2815, 2008.

6. Thall PF, Wathen JK, Bekele BN, Champlin RE, Baker LO, Benjamin RS. Hierarchical Bayesian approaches to phase II trials in diseases with multiple subtypes. Stat in Medicine, 22: 763-780, 2003.

7. Thall PF, Russell KT. A strategy for dose finding and safety monitoring based on efficacy and adverse outcomes in phase I/II clinical trials. Biometrics 54:251-264, 1998.

8. Thall PF, Cook JD. Dose-finding based on efficacy-toxicity trade-offs. Biometrics, 60:684-693, 2004.

9. Thall PF, Cook JD, Estey EH. Adaptive dose selection using efficacy-toxicity trade-offs: illustrations and practical considerations. J Biopharmaceutical Stat. 16:623-638, 2006.

10. Thall PF, Nguyen H, Estey EH. Patient-specific dose-finding based on bivariate outcomes and covariates. Biometrics. 64:1126-1136, 2008.

11. Thall PF, Millikan RE, Mueller P, Lee S-J. Dose-finding with two agents in phase I oncology trials. Biometrics, 59:487-496, 2003.

12. Bekele BN, Thall PF. Dose-finding based on multiple toxicities in a soft tissue sarcoma trial. J American Statistical Association, 99:26-35, 2004.

13. Houede N, Thall PF, Nguyen H, Paoletti X, Kramar A. Utility-based optimization of combination therapy using ordinal toxicity and efficacy in phase I/II trials. Biometrics. 66:532-540, 2010.

14. Thall PF, Szabo A, Nguyen HQ, Amlie-Lefond CM, Zaidat OO. Optimizing the concentration and bolus of a drug delivered by continuous infusion. Biometrics. 67:1638-1646, 2011.

15. Braun TM, Yuan Z, Thall PF. Determining a maximum tolerated schedule of a cytotoxic agent. Biometrics, 61:335-343, 2005.

16. Braun TM, Thall PF, Nguyen H, de Lima M. Simultaneously optimizing dose and schedule of a new cytotoxic agent. Clinical Trials, 4:113-124, 2007.

17. de Lima M, Giralt S, Thall PF, Silva LP, Wang X, Jones RB, Komanduri K, Braun TM, Nguyen HQ, Champlin R, Garcia-Manero G. Maintenance therapy with low-dose azacitidine after allogeneic hematopoietic stem cell transplantation for relapsed AML or MDS: a dose and schedule finding study. Cancer. 116:5420-5431, 2010.

18. Thall PF, Inoue LYT, Martin T. Adaptive decision making in a lymphocyte infusion trial. Biometrics, 58:560-568, 2002.

19. Thall PF, Simon R, Ellenberg SS. Two-stage selection and testing designs for comparative clinical trials. Biometrika 75: 303-310, 1988.

20. Schaid DJ, Wieand HS, Therneau TM. Optimal two-stage screening designs for survival comparisons. Biometrika 77: 507-513. 1990.

21. Thall PF, Nguyen HQ, Wang X, Wolff JE. A hybrid geometric phase II/III clinical trial design based on treatment failure time and toxicity. J Statistical Planning and Inference. 142:944-955, 2012.

22. Inoue LYT, Thall PF, Berry, DA. Seamlessly expanding a randomized phase II trial to phase III. Biometrics, 58:823-831, 2002.

23. Thall PF, Millikan R, Sung, H-G. Evaluating multiple treatment courses in clinical trials. Stat in Medicine, 19: 1011-1028, 2000

24. ThallPF, LogothetisC, PagliaroL, WenS, BrownMA, WilliamsD, MillikanR. Adaptive therapy for androgen independent prostate cancer: A randomized selection trial including four regimens. J National Cancer Institute. 99:1613-1622, 2007

25. Wang L, Rotnitzky A, Lin X, Millikan R, Thall PF. Evaluation of viable dynamic treatment regimes in a sequentially randomized trial of advanced prostate cancer. J. American Statistical Association. In press.

26. Thall PF, Wooten LH, Logothetis CJ, Millikan R, Tannir NM. Bayesian and frequentist two-stage treatment strategies based on sequential failure times subject to interval censoring. Stat in Medicine. 26:4687-4702, 2007.

27. Thall PF, Sung H-G, Estey EH. Selecting therapeutic strategies based on efficacy and death in multi-course clinical trials. J American Statistical Association, 97:29-39, 2002.

28. Thall PF, Champlin RE, Andersson BE. Comparison of 100-day mortality rates associated with IV busulfan and cyclophosphamide versus other preparative regimens in allogeneic bone marrow transplant for chronic myelogenous leukemia: Bayesian sensitivity analyses of confounded treatment and center effects. Bone Marrow Transplantation, 33: 1191-1199, 2004.