Clinical Trials Commons^™

Simulations Of A New Response-Adaptive Biased Coin Design, Aleksandra Stein

Department of Statistics: Dissertations, Theses, and Student Research

Modern medical experiments accrue and treat patients--hence obtain treatment response data--throughout a trial. Designs which prospectively plan to modify patient allocation by leveraging accumulating data are response-adaptive randomization (RAR) designs. Many such designs attempt to balance the desire to bias assignment proportions towards a treatment which is performing better against the need to maintain randomization in the face of continued equipoise.

This dissertation consists of simulated investigations into frequentist and ethical properties of an new RAR biased coin design. Chapter 2 proposes a new adaptive design for phase III clinical trials, a modification of the 2001 Bandyopadhyay and Biswas biased …

C-Learning: A New Classification Framework To Estimate Optimal Dynamic Treatment Regimes, Baqun Zhang, Min Zhang

The University of Michigan Department of Biostatistics Working Paper Series

Personalizing treatment to accommodate patient heterogeneity and the evolving nature of a disease over time has received considerable attention lately. A dynamic treatment regime is a set of decision rules, each corresponding to a decision point, that determine that next treatment based on each individual’s own available characteristics and treatment history up to that point. We show that identifying the optimal dynamic treatment regime can be recast as a sequential classification problem and is equivalent to sequentially minimizing a weighted expected misclassification error. This general classification perspective targets the exact goal of optimally individualizing treatments and is new and fundamentally …

Assessing Usual Care In Clinical Trials, Judith A. Erlen, Lisa K. Tamres, Nancy Reynolds, Carol E. Golin, Marc I. Rosen, Robert H. Remien, Julie W. Banderas, Neil Schneiderman, Glenn Wagner, David Bangsberg, Honghu Liu

OHSU-PSU School of Public Health Faculty Publications and Presentations

Researchers designing clinical trials often specify usual care received by participants as the control condition expecting that all participants receive usual care regardless of group assignment. The assumption is that the groups in the study are affected similarly. We describe the assessment of usual care within the 16 studies in MACH 14, a multi-site collaboration on adherence to antiretroviral therapy. Only five of the studies in MACH 14 assessed usual care. Assessment protocols varied as did the timing and frequency of assessments. All usual care assessments addressed patient education focused on HIV, HIV medications, and medication adherence. Our findings support …

Gene Expression Changes Reflect Clinical Response In A Placebo-Controlled Randomized Trial Of Abatacept In Patients With Diffuse Cutaneous Systemic Sclerosis, Eliza F. Chakravarty, Viktor Martyanov, David Fiorentino, Tammara A. Wood, David J. Haddon, Justin A. Jarrell, Paul Utz, Mark Genovese, Michael Whitfield, Lorinda Chung

Dartmouth Scholarship

Systemic sclerosis is an autoimmune disease characterized by inflammation and fibrosis of the skin and internal organs. We sought to assess the clinical and molecular effects associated with response to intravenous abatacept in patients with diffuse cutaneous systemic.