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Research Articles

Efficient Estimation of the Efficacy and Safety Endpoints for Clinical Trials with Preplanned Dose Titrations

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Pages 214-224 | Received 12 Jul 2021, Accepted 31 Dec 2021, Published online: 24 Feb 2022

References

  • Aroda, V. R., Bain, S. C., Cariou, B., Piletič, M., Rose, L., Axelsen, M., Rowe, E., and DeVries, J. H. (2017), “Efficacy and Safety of Once-Weekly Semaglutide Versus Once-Daily Insulin Glargine as Add-on to Metformin (With or Without Sulfonylureas) in Insulin-Naive Patients with Type 2 Diabetes (SUSTAIN-4): A Randomised, Open-Label, Parallel-Group, Multicentre, Multinational, Phase 3a Trial,” The Lancet Diabetes & Endocrinology, 5, 355–366. DOI: 10.1016/S2213-8587(17)30085-2.
  • Bartlett, J. W. (2018), “Covariate Adjustment and Estimation of Mean Response in Randomised Trials,” Pharmaceutical Statistics, 17, 648–666. DOI: 10.1002/pst.1880.
  • Cox, D. R. (1972), “Regression Models and Life-Tables,” Journal of the Royal Statistical Society, Series B, 34, 187–202. DOI: 10.1111/j.2517-6161.1972.tb00899.x.
  • Freedman, D. A. (2008), “Randomization Does not Justify Logistic Regression,” Statistical Science, 23, 237–249. DOI: 10.1214/08-STS262.
  • Frias, J. P., Nauck, M. A., Van, J., Kutner, M. E., Cui, X., Benson, C., Urva, S., Gimeno, R. E., Milicevic, Z., Robins, D., and Haupt, A. (2018), “Efficacy and Safety of LY3298176, a Novel Dual GIP and GLP-1 Receptor Agonist, in Patients with Type 2 Diabetes: A Randomised, Placebo-Controlled and Active Comparator-Controlled Phase 2 Trial,” The Lancet, 392, 2180–2193. DOI: 10.1016/S0140-6736(18)32260-8.
  • Johnson, R., and Wichern, D. (1998), Applied Multivariate Statistical Analysis (4th ed.), Upper Saddle River, NJ: Prentice Hall.
  • Kapitza, C., Nosek, L., Jensen, L., Hartvig, H., Jensen, C. B., and Flint, A. (2015), “Semaglutide, a Once-Weekly Human GLP-1 Analog, Does not Reduce the Bioavailability of the Combined Oral Contraceptive, Ethinylestradiol/Levonorgestrel,” The Journal of Clinical Pharmacology, 55, 497–504. DOI: 10.1002/jcph.443.
  • Kaplan, E. L., and Meier, P. (1958), “Nonparametric Estimation from Incomplete Observations,” Journal of the American Statistical Association, 53, 457–481. DOI: 10.1080/01621459.1958.10501452.
  • Klein, J. P., and Moeschberger, M. L. (2003), Survival Analysis: Techniques for Censored and Truncated Data (Vol. 1230), New York: Springer.
  • Liang, K.-Y., and Zeger, S. L. (2000), “Longitudinal Data Analysis of Continuous and Discrete Responses for Pre-post Designs,” Sankhyā: The Indian Journal of Statistics, Series B, 62, 134–148.
  • Lu, K. (2010), “On Efficiency of Constrained Longitudinal Data Analysis Versus Longitudinal Analysis of Covariance,” Biometrics, 66, 891–896. DOI: 10.1111/j.1541-0420.2009.01332.x.
  • Mallinckrodt, C., and Lipkovich, I. (2016), Analyzing Longitudinal Clinical Trial Data: A Practical Guide, Boca Raton: CRC Press.
  • Mantel, N. (1966), “Evaluation of Survival Data and Two New Rank Order Statistics Arising in its Consideration,” Cancer Chemotherapy Reports, 50, 163–170.
  • Molenberghs, G., Bijnens, L., and Shaw, D. (1997), “Linear Mixed Models and Missing Data,” in Linear Mixed Models in Practice, eds. G. Verbeke, and G. Molenberghs, pp. 191–274, New York: Springer.
  • Nauck, M. A., Petrie, J. R., Sesti, G., Mannucci, E., Courrèges, J.-P., Lindegaard, M. L., Jensen, C. B., and Atkin, S. L. (2016), “A Phase 2, Randomized, Dose-Finding Study of the Novel Once-Weekly Human GLP-1 Analog, Semaglutide, Compared with Placebo and Open-Label Liraglutide in Patients with Type 2 Diabetes,” Diabetes Care, 39, 231–241.
  • Peto, R., and Peto, J. (1972), “Asymptotically Efficient Rank Invariant Test Procedures,” Journal of the Royal Statistical Society, Series A, 135, 185–207. DOI: 10.2307/2344317.
  • Qu, Y. (2019), “Can a Multiple Ascending Dose Study Serve as an Informative Proof-of-Concept Study?” Statistics in Medicine, 38, 354–362. DOI: 10.1002/sim.7982.
  • Qu, Y., Liu, Z., Fu, H., Sethuraman, S., and Kulkarni, P. M. (2019), “Modeling the Impact of Preplanned Dose Titration on Delayed Response,” Journal of Biopharmaceutical Statistics, 29, 287–305. DOI: 10.1080/10543406.2018.1535499.
  • Qu, Y., and Luo, J. (2015), “Estimation of Group Means When Adjusting for Covariates in Generalized Linear Models,” Pharmaceutical Statistics, 14, 56–62. DOI: 10.1002/pst.1658.
  • Rubin, D. B. (2004), Multiple Imputation for Nonresponse in Surveys (Vol. 81), New York: Wiley.
  • Wang, M., Luo, J., Fu, H., and Qu, Y. (2014), “Piecewise Negative Binomial Regression in Analyzing Hypoglycemic Events with Missing Observations,” Journal of Biometrics & Biostatistics, 5, 195.
  • Weisstein, E. W. (2021), “Multinomial Distribution,” MathWorld–A Wolfram Web Resource.

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