Pre-Test: Foundations of Clinical Protocol

Pre-Test: Foundations of Clinical Protocol Development and Research Study Design in SCD

Instructions: This is a general knowledge check on clinical protocol development and research study design for Sickle Cell Disease (SCD) research. Please answer each question to the best of your current knowledge; if you are unsure, choose the best available answer. There is no penalty for an incorrect guess. Time: 5mins

In a parallel-group randomized controlled trial, what happens to participants after assignment?

Every participant receives all study treatments in sequence

Each group receives one assigned treatment for the full study and groups are compared

Participants choose their preferred intervention

Treatment assignments change at every visit without a predefined plan

Crossover designs are usually preferred for acute, rapidly evolving conditions because every participant receives multiple treatments in sequence.

True

Flase

Which design category is most appropriate when the goal is to describe natural history or estimate prevalence?

Observational design

Interventional design

Factorial RCT only

Platform trial only

All else being equal, what happens to the required sample size when the clinically meaningful effect to be detected becomes smaller?

The required sample size becomes smaller

The required sample size becomes larger

Sample size is unaffected

Statistical power becomes unnecessary

Which study design tests two or more interventions — and their combination — simultaneously within a single trial?

Crossover

Cohort

Factorial

Case-control

Which sequence correctly represents the recruitment funnel?

Eligible → Catchment population → Consented → Screened → Retained

Catchment population → Screened → Eligible → Consented → Enrolled and retained

Screened → Consented → Catchment population → Eligible → Retained

Consented → Screened → Eligible → Catchment population → Enrolled

Which ethical principle requires that individuals be treated as autonomous agents and that children receive additional protection?

Justice

Beneficence

Respect for persons

Scientific equipoise

A study design in which participants receive multiple treatments in sequence, separated by a washout period, is best described as which type of design?

Parallel-group

Crossover

Factorial

Cohort

Which of the following factors, if increased, would generally require a LARGER sample size in a clinical trial?

A larger, clinically meaningful effect size

Higher outcome variability between patients

A lower desired statistical power

A higher acceptable alpha (significance) level

When setting eligibility (inclusion/exclusion) criteria for a trial, which of the following represents a real risk?

Criteria that are too narrow, which can slow recruitment

Criteria that are too broad, which can threaten internal validity

Both — either extreme can undermine the trial

Eligibility criteria have little real effect on recruitment or validity

Pre-Test: Foundations of Clinical Protocol

Sickle Cell Disease African Scientific Symposium 2026

Pre-Test: Foundations of Clinical Protocol Development and Research Study Design in SCD