Clinical trial design and various phases of clinical trials MCQs With Answer

Clinical trial design and various phases of clinical trials are essential topics for B. Pharm students learning drug development, regulatory requirements, and patient safety. This introduction covers study types (randomized controlled trials, crossover, parallel), key concepts (blinding, placebo control, randomization), and the goals of Phase I (safety, pharmacokinetics), Phase II (dose-finding, preliminary efficacy), Phase III (confirmatory efficacy, large sample size), and Phase IV (post-marketing surveillance). Understanding ethics, informed consent, endpoints, sample size calculation, and data monitoring is vital for designing robust trials and interpreting results. Mastery of these principles prepares students for careers in clinical research, regulatory affairs, and pharmacovigilance. Now let’s test your knowledge with 30 MCQs on this topic.

Q1. What is the primary objective of a Phase I clinical trial?

• Assess long-term safety in large populations
• Evaluate safety and pharmacokinetics in healthy volunteers or patients
• Compare efficacy against standard therapy
• Monitor adverse events after marketing

Correct Answer: Evaluate safety and pharmacokinetics in healthy volunteers or patients

Q2. Which phase primarily focuses on dose-finding and preliminary evidence of efficacy?

• Phase I
• Phase II
• Phase III
• Phase IV

Correct Answer: Phase II

Q3. What is the main aim of a Phase III clinical trial?

• Determine maximum tolerated dose in animals
• Conduct large-scale confirmatory trials to demonstrate efficacy and safety
• Explore pharmacodynamics in vitro
• Collect spontaneous adverse event reports post-launch

Correct Answer: Conduct large-scale confirmatory trials to demonstrate efficacy and safety

Q4. Which term describes a trial design in which neither participants nor investigators know the treatment assignments?

• Open-label
• Single-blind
• Double-blind
• Non-randomized

Correct Answer: Double-blind

Q5. Randomization in clinical trials primarily helps to:

• Increase sample size
• Eliminate the need for informed consent
• Balance known and unknown confounders between groups
• Guarantee positive results

Correct Answer: Balance known and unknown confounders between groups

Q6. Which design is useful when each participant receives both treatments in different periods?

• Parallel-group design
• Crossover design
• Cluster randomized trial
• Factorial design

Correct Answer: Crossover design

Q7. What is a surrogate endpoint?

• A patient-reported outcome only
• An indirect measure expected to predict clinical benefit, like blood pressure for stroke risk
• An endpoint used only in Phase IV
• A safety endpoint measuring adverse events

Correct Answer: An indirect measure expected to predict clinical benefit, like blood pressure for stroke risk

Q8. Which committee reviews safety data and can recommend stopping a trial for harm or benefit?

• Institutional Review Board (IRB) only
• Data and Safety Monitoring Board (DSMB)/Data Monitoring Committee (DMC)
• Regulatory authority auditor
• Sponsor marketing team

Correct Answer: Data and Safety Monitoring Board (DSMB)/Data Monitoring Committee (DMC)

Q9. In oncology dose-escalation, which simple design is commonly used to find the maximum tolerated dose?

• 3+3 design
• Randomized withdrawal
• Stepped-wedge
• Factorial design

Correct Answer: 3+3 design

Q10. Intention-to-treat (ITT) analysis means:

• Analyze only those who completed the trial per protocol
• Analyze participants according to the group they were randomized to, regardless of adherence
• Exclude participants lost to follow-up
• Analyze only the safety population

Correct Answer: Analyze participants according to the group they were randomized to, regardless of adherence

Q11. Which regulatory submission is typically required before conducting clinical trials in humans in the USA?

• New Drug Application (NDA)
• Investigational New Drug (IND) application
• Biologic License Application (BLA)
• Post-marketing surveillance report

Correct Answer: Investigational New Drug (IND) application

Q12. What is a primary endpoint?

• A secondary safety measure
• The main outcome the trial is designed to assess
• An exploratory biomarker only
• A descriptive demographic variable

Correct Answer: The main outcome the trial is designed to assess

Q13. Which error occurs when a true treatment effect is declared absent (false negative)?

• Type I error (alpha)
• Type II error (beta)
• Selection bias
• Confounding

Correct Answer: Type II error (beta)

Q14. Blinding helps prevent which type of bias most directly?

• Selection bias
• Observer and performance bias
• Sampling error
• Publication bias

Correct Answer: Observer and performance bias

Q15. Phase IV clinical trials are primarily conducted to:

• Assess initial human safety
• Confirm efficacy before approval
• Monitor long-term safety and rare adverse events after marketing
• Test drugs in animals

Correct Answer: Monitor long-term safety and rare adverse events after marketing

Q16. Allocation concealment prevents:

• Participants from dropping out
• Investigators from foreseeing upcoming assignments and biasing enrollment
• Adverse events from being reported
• Regulatory approval

Correct Answer: Investigators from foreseeing upcoming assignments and biasing enrollment

Q17. Which design tests two interventions simultaneously in different combinations?

• Adaptive design
• Factorial design
• Crossover design
• Single-arm study

Correct Answer: Factorial design

Q18. A serious adverse event (SAE) is characterized by which outcome?

• Mild headache only
• Results in death, life-threatening event, hospitalization, or disability
• Minor, expected side effect
• Any event reported in Phase IV only

Correct Answer: Results in death, life-threatening event, hospitalization, or disability

Q19. Which analysis set includes only participants who fully adhered to the protocol?

• Intention-to-treat (ITT)
• Per-protocol (PP)
• Safety population
• Full analysis set

Correct Answer: Per-protocol (PP)

Q20. Which concept ensures participants understand risks, benefits, and alternatives before enrollment?

• Randomization
• Informed consent
• Blinding
• Allocation concealment

Correct Answer: Informed consent

Q21. Which trial phase often includes pharmacodynamic (PD) assessments along with pharmacokinetics (PK)?

• Preclinical studies
• Phase I and Phase II
• Phase IV only
• Bioequivalence studies only

Correct Answer: Phase I and Phase II

Q22. What is adaptive trial design?

• A trial fixed in design with no interim looks
• A design allowing pre-planned modifications based on interim data
• A non-randomized observational study
• Only used for vaccines

Correct Answer: A design allowing pre-planned modifications based on interim data

Q23. Equivalence trials are designed to demonstrate that a new treatment is:

• Clearly superior to control
• Not unacceptably worse and not unacceptably better than control within predefined margins
• Only safe in animals
• Effective only in post-marketing settings

Correct Answer: Not unacceptably worse and not unacceptably better than control within predefined margins

Q24. Which guideline provides international standards for good clinical practice?

• ICH-GCP (International Council for Harmonisation – Good Clinical Practice)
• FDA GLP only
• CFR Title 21 exclusively for EU
• WHO GMP for manufacturing only

Correct Answer: ICH-GCP (International Council for Harmonisation – Good Clinical Practice)

Q25. What does a Data Safety Monitoring Board (DSMB) typically review?

• Marketing strategies
• Interim safety and efficacy data to protect participants
• Only preclinical toxicology reports
• Informed consent forms after study completion

Correct Answer: Interim safety and efficacy data to protect participants

Q26. In sample size calculation, increasing power reduces the risk of which error?

• Type I error (false positive)
• Type II error (false negative)
• Sampling bias
• Observer bias

Correct Answer: Type II error (false negative)

Q27. Which trial design randomizes groups (e.g., clinics) rather than individuals?

• Cluster randomized trial
• Cross-sectional study
• Case-control study
• Adaptive individual randomization

Correct Answer: Cluster randomized trial

Q28. A SUSAR is:

• A suspected unexpected serious adverse reaction that is both serious and unexpected
• A routine monitoring visit
• A statistical analysis plan
• A manufacturing defect report

Correct Answer: A suspected unexpected serious adverse reaction that is both serious and unexpected

Q29. Which endpoint is most appropriate for a confirmatory Phase III trial in cardiovascular outcomes?

• Change in a surrogate biomarker only
• Major adverse cardiovascular events (MACE) such as MI, stroke, and death
• Number of lab tests performed
• Preclinical efficacy in animals

Correct Answer: Major adverse cardiovascular events (MACE) such as MI, stroke, and death

Q30. Pharmacovigilance after approval mainly focuses on:

• Initial dose-ranging in healthy volunteers
• Detecting, assessing, and preventing adverse effects and drug-related problems in the real world
• Nonclinical toxicology studies
• Development of new chemical entities in the lab

Correct Answer: Detecting, assessing, and preventing adverse effects and drug-related problems in the real world

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