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Biostatistics · Biostatistics Fundamentals

Biostatistics: Clinical Trial Design and Phases

Randomized controlled trial phases, blinding methods, randomization techniques, and intention-to-treat versus per-protocol analysis, one concept per card.

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What is the primary goal of a Phase I clinical trial?
To assess safety, tolerability, and appropriate dosage in a small group of participants.
Phase I trials typically enroll how many participants?
A small group, often 20 to 100 participants (frequently healthy volunteers).
What is the primary goal of a Phase II clinical trial?
To evaluate preliminary efficacy and further assess safety.
Approximately how many participants are typically enrolled in a Phase II trial?
Roughly 100 to 300 participants.
What is the primary goal of a Phase III clinical trial?
To confirm efficacy and monitor side effects in a large population, often compared to the standard treatment.
Phase III trials typically enroll how many participants?
Several hundred to several thousand participants.
What happens during a Phase IV clinical trial?
Post-marketing surveillance to monitor long-term safety and effectiveness after the drug is approved.
What is a Phase 0 trial?
An exploratory trial using very small, subtherapeutic drug doses to gather early pharmacokinetic data.
What does RCT stand for?
Randomized controlled trial.
What is the main purpose of randomization in a clinical trial?
To distribute known and unknown confounding factors evenly between treatment groups.
What is simple randomization?
Assigning participants to groups purely by chance, like a coin flip, with no restrictions.
What problem can simple randomization cause, especially in small trials?
It can result in unequal group sizes by chance.
What is block randomization?
A method that assigns participants in fixed-size blocks to keep group sizes balanced throughout the trial.
What is stratified randomization used for?
To ensure balance of a known prognostic factor, such as age or disease severity, between treatment groups.
What is cluster randomization?
Randomizing whole groups, such as clinics or communities, rather than individual participants.

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