Designing a clinical trial

Question 1

What is a clinical trial?

Answer 1

A type of research that studies new tests and treatments and evaluates their effects on human health outcomes

Question 2

What is the difference between aims and objectives?

Answer 2

Aims: set out what you want to achieve by the end of the study

Objectives: How you will achieve the aim through defined measurable outcomes

Question 3

What is a protocol?

Answer 3

A set of instructions to carry out the experimental procedures to address the primary research question

The protocol provides a scientific basis for the study.
(can use SPIRIT guidelines to create a protocol)

Question 4

Give examples of what is included in the protocol

Answer 4

Background 
Objectives 
Design 
Intervention 
Follow up visits and schedules 
Safety assessments 
Data analysis

Question 5

What is the purpose of comparative trials? (superiority trials)

Answer 5

To demonstrate if treatment X is superior to standard treatment in terms of outcome measures.
The null hypotheses: 2 treatments have the same effect

Question 6

What is the purpose of equivalence trials? (non-inferiority trials)

Answer 6

To demonstrate if X is no worse than standard treatment in terms of outcome measures
The null hypotheses: 2 treatments are different

The difference is set by predefined margins

Question 7

When would non-inferiority trials be used?

Answer 7

Bioequivalence trials (to demonstrate the PK and PD equivalence)
when testing new treatment against a placebo is unethical (e.g. cancer treatments)
To test if new treatment has advantages over standard treatment.

Question 8

Explain a parallel group design

Answer 8

Independent groups are allocated to different treatments

Question 9

Explain a crossover study design

Answer 9

Participants receive the same treatments in different orders/sequences.
This could involve a washout period in between.