CMB2000/L05 Clinical Trials I

Question 1

Define a clinical trial.

Answer 1

-A research study
-one or more human subjects
-prospectively assigned
-one or more interventions
-evaluate the effects of
-interventions
-health-related
-biomedical
-or behavioural outcomes

Question 2

What is the difference between clinical studies and trials?

Answer 2

Studies use human participants which can be interventional and observational
Trials involve investigational medicinal products

Question 3

What was the world’s first clinical trial in 562 BCE?

Answer 3

Comparison of 2 dietary regimes over 10 days
Meat & wine vs legumes & water
Vegetable eaters more nourished at end of experiment and continued diet

Question 4

Describe the first trial of 2 medical treatments in 1537.

Answer 4

Cauterization of wounds with boiling oil
New oil used on wounds giving reduced inflammation and swelling

Question 5

Describe one of the earliest controlled clinical trials in 1747.

Answer 5

Treatment of scurvy on HMS Salisbury
12 patients allocated either quart of cider, 25 drops of H2SO4, nutmeg, seawater or citrus fruits
Lemon/lime juice now compulsory in British navy

Question 6

Describe the first UK double blind clinical trial.

Answer 6

1944
MRC large scale trial comparing Patulin with in inert control solution (citrate buffer)

Question 7

What was the first ‘true’ randomised control trial?

Answer 7

1948
MRC streptomycin trial for pulmonary tuberculosis
Usual care + streptomycin and usual care
Random allocation (stratified by sex)

Question 8

Give 2 kinds of experimental studies.

Answer 8

Randomised control trials (RCTs)
Non-randomised control trials (nRCTs)

Question 9

Give 3 kinds of observational studies.

Answer 9

Cohort studies
Case-control studies
Cross-sectional studies
Ecological studies

Question 10

What is the key idea of randomised control trials?

Answer 10

Test effectiveness of new treatment while eliminating as many biases as possible

Question 11

Define a cohort study.

Answer 11

An observational study in which the defined group of people (cohort with similar characteristics) is followed over time

Question 12

How is a case-control study carried out?

Answer 12

Comparing a group who have a disease or condition with controls who are otherwise as similar as possible

Question 13

What is the goal of case-control studies?

Answer 13

To find out possible causes of a disease/condition

Question 14

What is a cross-sectional study?

Answer 14

A snapshot observation of a set of people at 1 time

Question 15

Why are cross-sectional studies used?

Answer 15

To assess exposure (cause) and a disease (effect)
To compare the rates of diseases and symptoms of an exposed group with an unexposed group

Question 16

Why are ecological studies used?

Answer 16

To understand relationships between outcome and exposure at a population level
Population - group with shared characteristic

Question 17

Give the 5 phases of a clinical trial.

Answer 17

Laboratory studies
Phases I-IV

Question 18

Describe the role of laboratory studies.

Answer 18

Basic & preclinical research
Drug discovery
In vitro and in vivo testing
Model desired effect and predict adverse effects

Question 19

Describe phase I of clinical trials. (3)

Answer 19

Testing in small group (20-80 people)
Learn about safety and side effects
Several months
52% success

Question 20

Describe phase II of a clinical trial. (3)

Answer 20

Testing in larger group (100-300)
Emphasis of effectiveness in people with disease/condition
1-4 years
30% success

Question 21

Describe phase III of a clinical trial. (3)

Answer 21

Large groups of people (1000-3000)
Safety, effectiveness and dosages
Polypharmacy
58% success

Question 22

When is an NDA (New Drug Application) submitted?

Answer 22

After phase III of a clinical trial
Submitted to e.g., FDA

Question 23

Describe phase IV of a clinical trial. (2)

Answer 23

Drug available to public and monitored in large, diverse populations
More side effects or treatment benefits become apparent

Question 24

How was the COVID-19 vaccine manufactured so quickly?

Answer 24

AdVac viral vector (version of adenovirus 26) used in Ebola vaccine used to deliver virus