CLINICAL TRIALS

The Way We Make Progress Against

Disease
 INTRODUCTION
 Clinical trials are conducted to allow safety and efficacy data to be
collected for new drugs or devices.

 Clinical trials include research studies involving people along with

finding better ways to prevent, diagnose, or treat disease.

 Clinical trials translate results of basic scientific research into better

ways to prevent, diagnose, or treat disease.

 Because the clinical trial is designed to test hypothesis and rigorously

monitor and assess what happens, clinical trials can be seen as the
application of scientific method to understanding human or animal
biology.
 TYPES OF CLINICAL TRIALS
 There are two general types of clinical trials:

 Therapeutic clinical trials are used to evaluate new

treatments for childhood cancer as well as other diseases.
These studies are classified as three different types or
phases. Each phase has different goals.

 Non-therapeutic trials are those that involve other

aspects of childhood cancer such as:
 Epidemiology (incidence and causes)
 Cancer Control (how best to deal with side effects)
 Late Effects (long-term complications of treatment
 CLASSIFICATION OF TRIALS
Clinical trials may be classified into following:
 Prevention trials: look for better ways to prevent disease in people.
These approaches may include medicines, vitamins, vaccines, minerals, or
lifestyle changes.

 Screening trials: test the best way to detect certain diseases or health
conditions.

 Diagnostic trials: conducted to find better tests or procedures for

diagnosing a particular disease or condition.

 Treatment trials: test experimental treatments, new combinations of

drugs, or new approaches to surgery or radiation therapy.

 Quality of life trials: explore ways to improve comfort and the quality
of life for individuals with a chronic illness.
 CLINICAL TRIALS PHASES
Phase I aims:
 Test a new treatment in humans that has been tested in
the laboratory and in animal studies.

 Learn how the human body responds to a new treatment

and what the side effects are.

 Find the best way to give a new treatment.

 Find the highest dose of a new treatment with acceptable

side effects.
 Phase II aims:

 Learn if a new treatment works in patients with

specific types of cancer.

 Learn more about how a new treatment affects

the human body.
 Phase III aims:

 Learn if a new treatment that has been shown effective

in treating a specific type of cancer in a Phase II trial
is better in some way (more cures, longer control of
disease, fewer or less serious side effects, or fewer
days in the hospital) than the best current treatment
for the disease.

 These trials are usually offered to patients who have

just been diagnosed with cancer.
 TREATMENT TRIALS
 It refers to trials which test new treatments, new combinations
of drugs, or new approaches to surgery or radiation therapy.

 Placebos are used only when no standard treatment exists.

 A placebo is an inactive pill, liquid, or powder that has no

treatment value.

 In clinical trials, experimental treatments are often compared

with placebos to assess the experimental treatment's
effectiveness.
 PREVENTION TRIALS
 Evaluate the effectiveness of ways to reduce the risk of a
particular disease.

 Enroll healthy people at high risk for developing that disease.

 Action studies (doing something): These focus on finding out whether

actions people take, such as exercising more or quitting smoking, can
prevent cancer.

 Agent studies (taking something): These studies (also called

chemoprevention studies) focus on examining whether taking certain
medicines, vitamins, minerals or food supplements (or a combination of
them) can prevent cancer.
 CLINICAL TRIAL PROTOCOL
 A protocol is a study plan on which all clinical trials are based.

 A protocol describes what types of people may participate in the

trial; the schedule of tests, procedures, medications, and dosages;
and the length of the study

 A recipe or blueprint
 Strict scientific guidelines:
--Purpose of study
--How many people will participate
--Who is eligible to participate
--How the study will be carried out
--What information will be gathered about participants
--Endpoints
 BENEFITS OF PARTICIPATING

 If you have a disease that cannot be treated with an

existing drug or regimen, participation might provide you
with a successful treatment before it becomes available
to others. 

 If you are debilitated in some way, you may have the

opportunity to test a regimen that would improve your
quality of life.

 The drugs and protocols offered during clinical trials

are often provided at no cost to participants.
RISKS OF PARTICIPATING

 There may be side effects or outcomes that are

unpleasant. They may last only for a short time, or
they may affect you for the rest of your life.

 The time and attention required of participants may be

long. It may require hours of testing, miles of travel,
hospital stays or complicated dosing. 

 New doesn't always mean better.

 Why Do So Few People Participate in
Clinical Trials?

Sometimes patients:

 Don’t know about clinical trials.

 Don’t have access to clinical trials.
 May be afraid or suspicious of research.
 Can’t afford to participate.
 May not want to go against health care and
provider’s wishes.
 REFERENCES
 About.com: Patient Empowerment

 ClinicalTrials.gov: Glossary

 ICH guideline for Good Clinical Practice:

Consolidated Guidance.
THANKS!