Clinical trial phases and protocol design for phytomolecules MCQs With Answer

Introduction: This quiz collection focuses on clinical trial phases and protocol design specifically for phytomolecules, tailored for M.Pharm students. It covers key concepts required to translate botanical compounds from bench to bedside, including preclinical safety, IND considerations, phase-wise objectives, dose-escalation strategies, pharmacokinetics of complex plant-derived substances, and regulatory issues unique to phytomedicines such as standardization and botanical drug INDs. The questions emphasize protocol elements: endpoints, sample size, randomization, blinding, inclusion/exclusion criteria, DSMB roles, and adaptive designs. Use these MCQs to test and deepen your understanding of designing robust, ethical, and regulatory-compliant clinical trials for phytomolecules.

Q1. Which phase of clinical trials primarily assesses safety, tolerability, pharmacokinetics, and pharmacodynamics of a phytomolecule in a small group of healthy volunteers or patients?

• Phase 0
• Phase I
• Phase II
• Phase III

Correct Answer: Phase I

Q2. For a botanically derived compound with complex constituents, which preclinical study is most critical to predict human systemic exposure and support first-in-human dosing?

• Repeated-dose toxicity only
• Pharmacokinetic and ADME studies in relevant animal species
• Genotoxicity testing only
• Carcinogenicity studies

Correct Answer: Pharmacokinetic and ADME studies in relevant animal species

Q3. Which regulatory submission is required before initiating clinical trials of a new phytomolecule in humans in most jurisdictions?

• New Drug Application (NDA)
• Investigational New Drug (IND) application or equivalent
• Marketing Authorization Application (MAA)
• Certificate of Analysis (CoA)

Correct Answer: Investigational New Drug (IND) application or equivalent

Q4. Which trial design is most suitable for early human microdosing studies to gather human PK data with sub-therapeutic exposure of a phytomolecule?

• Crossover therapeutic trial
• First-in-human, single-dose Phase I dose-escalation
• Phase 0 microdosing study (exploratory IND)
• Randomized Phase III efficacy trial

Correct Answer: Phase 0 microdosing study (exploratory IND)

Q5. In designing a clinical protocol for a plant-derived drug, which element addresses variability in composition between batches and assures reproducible clinical effects?

• Placebo selection
• Standardization and chemical fingerprinting of the botanical extract
• Choice of primary endpoint
• Randomization schedule

Correct Answer: Standardization and chemical fingerprinting of the botanical extract

Q6. Which dose-escalation method is commonly used in Phase I oncology trials but may be adapted for phytomolecule safety studies to identify Maximum Tolerated Dose (MTD)?

• Randomized block design
• 3+3 design
• Cross-sectional design
• Case-control design

Correct Answer: 3+3 design

Q7. Which primary endpoint is most appropriate for a Phase II efficacy trial of a phytomolecule intended to reduce inflammatory biomarkers?

• Long-term overall survival
• Change in validated biomarker level from baseline
• Cost-effectiveness ratio
• Market uptake after approval

Correct Answer: Change in validated biomarker level from baseline

Q8. When designing inclusion/exclusion criteria for a clinical trial of a phytomolecule, which rationale is most important?

• Maximize number of eligible participants regardless of safety
• Balance generalizability with participant safety and ability to detect the drug effect
• Exclude all comorbid conditions to ensure homogeneity
• Include only pediatric subjects

Correct Answer: Balance generalizability with participant safety and ability to detect the drug effect

Q9. Which statistical consideration determines the number of subjects required to detect a clinically meaningful effect of a phytomolecule in a randomized trial?

• Blinding method
• Sample size calculation based on effect size, alpha, and power
• Manufacturing batch size
• Number of study sites only

Correct Answer: Sample size calculation based on effect size, alpha, and power

Q10. For a botanical extract with known potential herb–drug interactions, what important pharmacokinetic assessment should be included in early clinical trials?

• Food frequency questionnaire only
• In vitro enzyme inhibition without in vivo confirmation
• Clinical drug interaction studies evaluating CYP and transporter modulation
• Subjective tolerability surveys only

Correct Answer: Clinical drug interaction studies evaluating CYP and transporter modulation

Q11. Which ethical oversight body must review and approve the clinical protocol before enrolling participants in most countries?

• Clinical trial sponsor’s marketing department
• Data Safety Monitoring Board (DSMB)
• Institutional Review Board (IRB) or Ethics Committee (EC)
• Manufacturing Quality Unit

Correct Answer: Institutional Review Board (IRB) or Ethics Committee (EC)

Q12. In a randomized, placebo-controlled trial, what is the main purpose of blinding the investigators and participants?

• To simplify data collection
• To reduce bias in outcome assessment and reporting
• To allow flexible dosing during the trial
• To increase enrollment speed

Correct Answer: To reduce bias in outcome assessment and reporting

Q13. Which document provides the detailed plan for objectives, design, methodology, statistical considerations, and organization of a clinical trial for a phytomolecule?

• Investigator’s brochure only
• Clinical study protocol
• Informed consent form only
• Case report form (CRF) template

Correct Answer: Clinical study protocol

Q14. What role does a Data Safety Monitoring Board (DSMB) play in a randomized clinical trial of a phytomolecule?

• Manufactures the investigational botanical product
• Independently reviews interim safety data and advises on trial continuation or modification
• Responsible for marketing strategies post-approval
• Randomizes participants to treatment arms

Correct Answer: Independently reviews interim safety data and advises on trial continuation or modification

Q15. Which adaptive design feature can be useful in early-phase phytomolecule trials to modify allocation or sample size based on interim results?

• Fixed sample parallel design only
• Adaptive randomization and sample size re-estimation
• Unblinded final analysis only
• Complete replacement of control arm without interim review

Correct Answer: Adaptive randomization and sample size re-estimation

Q16. For a phytomolecule intended for chronic use, which Phase III trial characteristic is essential to evaluate long-term safety and efficacy?

• Short single-dose exposure
• Longer duration with adequate follow-up and monitoring of adverse events
• Only pharmacokinetic sampling for 24 hours
• Exclusion of all elderly subjects

Correct Answer: Longer duration with adequate follow-up and monitoring of adverse events

Q17. When selecting endpoints for clinical trials of phytomolecules with multi-component mechanisms, which approach is most appropriate?

• Use only subjective patient-reported outcomes
• Define a hierarchical endpoint strategy combining clinical outcomes and validated biomarkers
• Rely solely on in vitro assay endpoints
• Choose endpoints based on convenience rather than biological relevance

Correct Answer: Define a hierarchical endpoint strategy combining clinical outcomes and validated biomarkers

Q18. What is the primary concern when using a placebo control in trials of traditional herbal preparations?

• Regulatory approval is always faster with placebo
• Creating a matching placebo that mimics taste, smell, and appearance to maintain blinding
• Placebos improve pharmacokinetic profiles
• Placebos eliminate the need for informed consent

Correct Answer: Creating a matching placebo that mimics taste, smell, and appearance to maintain blinding

Q19. In post-marketing (Phase IV) studies of a phytomolecule, what primary activity helps detect rare or long-term adverse events?

• Preclinical animal toxicology
• Pharmacovigilance and spontaneous adverse event reporting systems
• Phase I single ascending dose studies
• Short-term bioequivalence studies

Correct Answer: Pharmacovigilance and spontaneous adverse event reporting systems

Q20. For botanical drug development, which manufacturing and quality requirement must be addressed in the clinical protocol to ensure patient safety and data integrity?

• Flexible, undocumented extraction methods
• Good Manufacturing Practice (GMP) compliant production and validated analytical methods for identity and potency
• No need for batch records if clinical grade is claimed
• Only final product sensory testing

Correct Answer: Good Manufacturing Practice (GMP) compliant production and validated analytical methods for identity and potency

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