This volume is a comprehensive textbook for investigators entering the rapidly growing field of translational and experimental clinical research. The book offers detailed guidelines for designing and conducting a study and analyzing and reporting results and discusses key ethical and regulatory issues. Chapters address specific types of studies such as clinical experiments in small numbers of patients, pharmacokinetics and pharmacodynamics, and gene therapy and pharmacogenomic studies. A major section describes modern techniques of translational clinical research, including gene expression, identifying mutations and polymorphisms, cloning, transcriptional profiling, proteomics, cell and tissue imaging, tissue banking, evaluating substrate metabolism, and in vivo imaging.

Table of Contents

SECTION I: Planning and Designing Clinical Research 1 * Defining the Research Question 3 * Searching the Literature 15 * Variables and Measurements 31 * Study Eligibility and Participant Selection 45 * Recruiting Research Participants 54 * Reducing Bias 67 * Observational Study Designs 73 * Experimental Study Designs 84 * Estimating Sample Size 97 * Database Development 113 * Case Report Form Development 122 * Data Collection and Quality Control 136 * Monitoring Experimental Interventions 152 SECTION II: Special Topics 161 * Responsible Conduct of Research 163 * Regulatory Oversight 176 * Funding Clinical Research 189 * Research Budgeting 198 *"Small" Clinical Experiments 203 * Clinical Studies in Pediatric Minority Populations 209 * Pharmacokinetics and Pharmacodynamics 212 * Gene Therapy and Pharmacogenomic Studies 223 * Preparing for Gene Therapy Studies 237 SECTION III: Analyzing and Reporting Results 247 * Overview: Hypothesis Testing and Summary Statistics 249 * Group Comparisons 258 * Regression 271 * Multivariate Analysis 284 * Biostatistical Consulting 297 * Statistical Computing 303 * Presenting Data in Manuscripts 308 * Visual Presentation of Data 320 SECTION IV: Modern Techniques of Translational Clinical Research 331 * Fundamentals of Gene Expression 333 * Identifying Mutations and Polymorphisms 343 * Cloning Methods 356 * Transcriptional Profiling 369 * Proteomics 385 * Cell and Tissue Imaging Techniques 396 * Non-Imaging Cell and Tissue Techniques and Tissue Banking 413 * Evaluating Substrate Metabolism 435 * Medical Images as Scientific Data 450 Index 465