Clinical Trial

Li Zhou
School of Public Health and Management

Chongqing Medical University

2020
 Different study designs
Epidemiological
studies

Analytical studies Descriptive studies

Observational Experimental
studies studies

Ex. Case –control Ex. Clinical trails

Cohort RCT 2
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 Experimental epidemiology
 Experimental epidemiology is an
experiment in which the intervention is
applied to individuals (patients or healthy
people) and its effects are evaluated.
 Experimental studies include clinical trials,
field trials and community trials.

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 What are clinical trials?
 Clinical trials are medical research studies
involving people.
 They aim to test whether different treatments are
safe and how well they work.
 They aim to find the best ways to:
 Prevent disease and reduce the number of people who become ill;
 Treat illness to improve survival or increase the number of
people cured
 Improve the quality of life for people living with illness
 Diagnose diseases and health problems.
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 Historical Minute
First “Clinical Trials”

 Clinical Trials have a long history –

Dr. Lind’s, a ship surgeon, trial of oranges
& limes for scurvy [1747]

Prevention of Scurvy – Orange & Lemon 5

Core Components of Clinical Trials

 Involve human subjects

 Move forward in time
 Most have a comparison CONTROL group
 Must have method to measure intervention
 Focus on unknowns: effect of medication

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Core Components of Clinical Trials

 Must review existing scientific data &

build on that knowledge
 Test a certain hypothesis
 Study protocol must be built on sound &
ethical science
 Control for any potential biases
 Most study medications, procedures,
and/or other interventions
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Essential features of clinical trials

 Randomized
 Controlled

 Blinding

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 Randomization

 With Randomization, the determination of

assignment to treatment group is based on
probability alone and is not influenced by the
preference of the physician or patient.
 Maximizes the probability that two groups will
be similar in background characteristics .

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 Randomization

 The purpose of randomization is to achieve

“equality” of baseline characteristics of
treatment groups, so that the comparison of
treatment is considered fair.
 How to assess the equality of the treatment
groups?
 Comparing the demographic and prognosis
factors between two groups. 10
 Distribution of baseline characteristics of patients
enrolled in the diabetes therapy trial
Standard intensive
Characteristic therapy therapy
N=378 N=346
Age(years) 26 8 277
Adolescents (%) 19 16
Male sex (%) 54 49
White race (%) 96 96
Duration of IDDM(years) 2.61.4 2.6 1.4
Insulin dose 0.62 0.26 0.620.25
Glycosylated hemoglobin(%) 8.81.7 8.8 1.6
Mean blood glucose 22980 23486
Body weight(% of ideal) 10314 1031311
 Controlled trials
Controlled trials are designed to compare
different treatments.
 Trail group (intervention group): new

treatment
 Control group: standard treatment

In situations where there is no standard treatment the

control group may not be given any treatment at all or
may be given a placebo.

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 A placebo treatment is designed to appear
very similar to the treatment being tested.
 Placebo Effect: A beneficial response to
a substance, device, or procedure that
cannot be accounted for on a basis of
pharmacological or other direct physical
action.
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 Blind trials
 To account for the placebo effect and to
reduce the introduction of bias duo to
the conceptions of patients and
clinicians, studies may be conducted in
a blinding fashion
 “blinding“ means that the treatment
assignments is not known to certain
persons.
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 Single Blind Study: A clinical trial where the
participant does not know the identity of the
treatment received

 Double Blind Study: A clinical trial in which

neither the patient nor the treating
investigators know the identity of the
treatment being administered.
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Summary of various types of blinding to
assignment of treatment in clinical trials

Knowledge of
Treatment Assignment
Blinding patient Investigator

None Yes Yes

Single NO Yes
Double NO NO 16
 Phases of Clinical Trials

 Most trials that involve new drugs go

through a series of steps:
• #1: Experiments in the laboratory
• #2: Once deemed safe, go through
1-4 phases

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 Phases of Clinical Trials

 Phase I: Small group [20-80] for 1st time to

evaluate safety, determine safe dosage range

 Phase II: larger group [100-300] to confirm

effectiveness, further evaluate safety and
side effects

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Phases of Clinical Trials (cont.)
 Phase III: larger group [1,000-3,000] to
compare the effects of new drugs with the
standard treatment, find out how well the new
drug work and how long the effects last

 Phase IV: carried out after a new drug has been

shown to work and has been given a license, to
find out the long-term risks and benefits,
possible rare side effects.
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 Summary
 1. What are clinical trials?
 2. The essential features of clinical trials.
 3. What is Placebo Effect?
 4. Two types of Blind Study.

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The end!!!

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