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Unformatted text preview: Handout 3: Basics of Clinical Trial Design Pre-clinical research The pre-clinical phase covers all treatment development that is not carried out in human subjects. There are two main settings: in vitro (tissue culture, infectious organisms in culture) and in vivo (animal models such as mice, rats, non-human primates). Goals of pre-clinical research: To identify compounds that show activity against infectious agents or tumor cells. To identify compounds that bind to specific cell markers. To build devices that have specific properties with tissue. To determine whether interventions are sufficiently promising in animals to warrant human testing. To determine reasonable dosages for first human studies. To refine techniques (e.g. surgery, imaging) needed for treating or monitoring in humans. Examples of statistical challenges in pre-clinical research: Models for outcomes in cell culture (bioassay). Phase I Trials A Phase I trial is designed to find the best dosage of a drug for use in human subjects. Dosage include amount of drug, route and frequency of administration, and how amount is calculated (per body weight, per surface area, different for male and female, etc.) Usually the first studies of treatment in human subjects. Sometimes you want the highest tolerated dosage (cancer chemotherapy) but other times you want the lowest dose that is active (vaccine that induces immune response.) Sample sizes are typically quite small. Patients in treatment (as opposed to prevention) studies are often very sick, desperate. New approaches try to give more patients an effective dose. Not generally randomized. Usually start with a low dose and escalate until you hit dose-limiting toxicities. For vaccine, might start high and reduce until you no longer get protection....
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This note was uploaded on 12/30/2010 for the course BST 252 taught by Professor Tsodikov during the Winter '06 term at UC Davis.
- Winter '06