LWBK1006-16 LWW-Govindan-Review December 12, 2011 18:55
CHAPTER 16 DESIGN AND
ANALYSIS OF CLINICAL TRIALS
FENG GAO•KATHRYN TRINKAUSDIRECTIONS Each of the numbered items below is followed by lettered answers. Select the
ONE lettered answer that is BEST in each case unless instructed otherwise.QUESTIONS
Question 16.1. Which of the following is NOT true concerning a clinical trial?
A. The sample size of a clinical trial is fixed and determined before the
trial begins.
B. Clinical trials are prospectively planned experiments involving
human subjects.
C. A well-chosen control group provides a baseline against which to
measure effects of experimental treatment.
D. Clinical trials test a clearly stated hypothesis using a predefined anal-
ysis plan.Question 16.2. Phase I trials on cytotoxic agents are designed to determine a dose that is
appropriate for use in Phase II trials. Which of the following statements
is TRUE of Phase I trials?
A. Phase I trials involve the first use of a drug, device, or procedure in
humans in a new disease setting or a new combination of therapies.
B. Patients for Phase I trials are usually not chosen on the basis of their like-
lihood of having a favorable response to the experimental treatment.
C. Phase I trials do not require statistical justification because the sample
sizes are usually small.
D. A and B.Question 16.3. Which of the following regarding the conventional 3+3 Phase I designs
is NOT true?
A. Participants are often those with end-stage disease whose treatment
options have been exhausted.
B. Dose levels are usually prespecified and based on a modified Fibonacci
series.
C. Cohorts of three to six patients are treated at each dose level, and the
initial dose is chosen to cause little or no toxicity.
D. The optimal dose recommended for Phase II is chosen using a dose-
toxicity curve generated from a statistical model fitted to the full data.Corresponding Chapter inCancer: Principles & Practice of Oncology,Ninth Edition: 70 (Design and Analysis of
Clinical Trials).183