CTA Biostatistics & Study Design Flashcards

Which type of clinical trial design randomly assigns participants to treatment or control groups to minimize bias?

Answer: Randomized controlled trial (RCT)

A randomized controlled trial (RCT) uses random assignment to treatment or control groups, which is the gold standard for minimizing selection bias in clinical research.

In a double-blind clinical trial, who is unaware of the treatment assignment?

Answer: Both the participant and the investigator

In a double-blind study, both the participant and the investigator are unaware of treatment assignments to prevent performance and detection bias.

What is a Phase I clinical trial primarily designed to evaluate?

Answer: Safety and dosing in a small group of healthy volunteers

Phase I trials are the first human studies focused on assessing safety, tolerability, and pharmacokinetics in a small group, typically healthy volunteers.

What does the term 'placebo-controlled' mean in a clinical trial?

Answer: A control group receives an inactive substance identical in appearance to the treatment

In a placebo-controlled trial, the control group receives an inert substance that looks identical to the active treatment, allowing true comparison of treatment effects.

Which study design follows a group of participants over time to assess outcomes?

Answer: Cohort study

A cohort study follows a defined group of people (a cohort) over time to observe outcomes, making it effective for studying incidence and causality.

What is the primary purpose of a Phase III clinical trial?

Answer: Confirming efficacy and monitoring adverse reactions in a large population

Phase III trials enroll large patient populations to confirm efficacy, monitor adverse reactions, and compare the new treatment to existing standard therapies prior to regulatory approval.