CMB2000/L05 Clinical Trials I Flashcards
Define a clinical trial.
A research study in which one or more human subjects are prospectively assigned to one or more interventions to evaluate the effects of those interventions on health-related biomedical or behavioural outcomes
What is the difference between clinical studies and trials?
Studies use human participants which can be interventional and observational
Trials involve investigational medicinal products
What was the world’s first clinical trial in 562 BCE?
Comparison of 2 dietary regimes over 10 days
Meat & wine vs legumes & water
Vegetable eaters more nourished at end of experiment and continued diet
Describe the first trial of 2 medical treatments in 1537.
Cauterization of wounds with boiling oil
New oil used on wounds giving reduced inflammation and swelling
Describe one of the earliest controlled clinical trials in 1747.
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
Describe the first UK double blind clinical trial.
1944
MRC large scale trial comparing Patulin with in inert control solution (citrate buffer)
Patulin caused toxic effects (mycotoxin produced by Penecillin)
What was the first ‘true’ randomised control trial?
1948
MRC streptomycin trial for pulmonary tuberculosis
Usual care + streptomycin and usual care
Random allocation (stratified by sex)
Give 2 kinds of experimental studies.
Randomised control trials (RCTs)
Non-randomised control trials (nRCTs)
Give 3 kinds of observational studies.
Cohort studies
Case-control studies
Cross-sectional studies
Ecological studies
What is the key idea of randomised control trials?
Test effectiveness of new treatment while eliminating as many biases as possible
Define a cohort study.
An observational study in which the defined group of people (cohort with similar characteristics) is followed over time
How is a case-control study carried out?
Comparing a group who have a disease or condition with controls who are otherwise as similar as possible
What is the goal of case-control studies?
To find out possible causes of a disease/condition
What is a cross-sectional study?
A snapshot observation of a set of people at 1 time
Why are cross-sectional studies used?
To assess exposure (cause) and a disease (effect)
To compare the rates of diseases and symptoms of an exposed group with an unexposed group
Why are ecological studies used?
To understand relationships between outcome and exposure at a population level
Population - group with shared characteristic
Give the 5 phases of a clinical trial.
Laboratory studies
Phases I-IV
Describe the role of laboratory studies. (4)
Basic & preclinical research
Drug discovery
In vitro and in vivo testing
Model desired effect and predict adverse effects
Describe phase I of clinical trials. (3)
Testing in small group (20-80 people)
Learn about safety and side effects
Several months
52% success
Describe phase II of a clinical trial. (3)
Testing in larger group (100-300)
Emphasis of effectiveness in people with disease/condition
1-4 years
30% success
Describe phase III of a clinical trial. (3)
Large groups of people (1000-3000)
Safety, effectiveness and dosages
Polypharmacy
58% success
When is an NDA (New Drug Application) submitted?
After phase III of a clinical trial
Submitted to e.g., FDA
Describe phase IV of a clinical trial. (2)
Drug available to public and monitored in large, diverse populations
More side effects or treatment benefits become apparent
How was the COVID-19 vaccine manufactured so quickly?
AdVac viral vector (version of adenovirus 26) used in Ebola vaccine used to deliver virus
