Transcript e79ce07b3e7804d

‫بسم هللا الرحمن الرحيم‬
‫جامعة أم درمان اإلسالمية‬
‫كلية الطب و العلوم الصحية ‪ -‬قسم طب المجتمع‬
‫مساق البحث العلمي ‪ /‬الدفعة ‪21‬‬
‫‪Basics of‬‬
‫‪Clinical Trials‬‬
• A clinical trial is a research study in human
volunteers to answer specific health
questions.
• Carefully conducted clinical trials are the
safest and fastest scientific way to:
– Test new treatments;
– Test new combinations of drugs;
– Test new approaches to surgical procedure or
radiation, medical device, or of nutrition or
behavioral changes in people;
• In order to find out if is safe and effective for
people to use to improve health.
• Some studies are done in different populations,
such as:
• In children, in whom the treatment was not
previously tested;
• Or to compare already existing treatments to
determine which is better;
• Or to study different ways to use the standard
treatments (=current, approved treatments)
• So, they will be more effective, easier to use,
and/ or to decrease side effects;
• The overall purpose of a clinical trial is to learn,
not to treat patients.
Clinical Trials
• Are conducted according to a plan called a
protocol.
• The protocol describes: what types of patients
may enter the study, schedules of tests and
procedures, drugs, dosages, and length of
study, as well as the outcomes that will be
measured.
• Each person participating in the study must
agree to the rules set out by the protocol.
Kinds of clinical trials
• 1- Prevention trials: look for better ways to
prevent disease in people who have never had
the disease or to prevent a disease from
returning. These approaches may include
medicines, vitamins, vaccines, minerals, or
lifestyle changes.
• 2- Diagnostic trials: (for new techniques) to
find better tests for diagnosing a particular
disease.
3- Screening trials: (for new techniques) to test
the best way to detect certain diseases or
health conditions;
4- Treatments trials: to test new treatments or
new ways to use existing treatments;
5- Quality of Life trials (or Supportive Care
trials): options for improving the quality of life
for individuals with a chronic illness.
Who can contribute in the clinical trials?
• It is important to test drugs and medical products
in:
1- People whom are meant to be helped.
2- Ensure that a variety of people of different ages,
races, ethnic groups, and genders are included in
clinical trials (as people may respond differently).
3- People who have used standard (approved)
treatment options; but either did not work for
them, or unable to tolerate certain side effects. So
clinical trials may provide another option when
standard therapy has failed.
4- People who want to contribute to the advancement
of medical knowledge.
Possible Risks
• There are generally known & unknown risks
associated with clinical trials, such as:
1- There may be unpleasant, serious, or lifethreatening side effects resulting from the
treatment;
2- The treatment may not be effective for some
participants;
3- The protocol may require more of the
participant's time and attention than a standard
treatment. (To visit the study site on a regular
basis, be subjected to additional tests, get more
treatments than are normally necessary, stay in
the hospital or follow complex dosage
requirements, etc. ...)
Phases of Clinical Trials
• After a treatment is tested in the laboratory, it can
go to human testing . There are four phases of
human testing:
Phase I: Researchers test a new treatment in a small
group of healthy volunteers (20-80) for the first
time to test its safety, identify the maximum
tolerated dose, find a safe dosage range and
identify side effects.
Phase II: The treatment is given to a larger group of
patients (100-300) with disease who might benefit
from the drug, to see if it is effective, to evaluate
its safety and to gather information regarding safe
dose range.
Phase III: The treatment is given to large groups of
patients
(1,000-3,000)
to
confirm
its
effectiveness, monitor side effects, compare it to
commonly used treatments and collect
information that will allow the drug or treatment
to be used safely.
Phase IV: Trials are carried out in the general
population after the new drug has been approved
for use, investigators are looking for additional
information, including the treatment's risks,
benefits, and optimal use.
Phase V: Trials may be conducted to determine
better dosing guidelines, new formulations,
effects on different populations or new
indications.
What is informed consent?
• Informed consent: is the process of learning
the key fact about the clinical trial, it gives
complete information about why the trial is
being done, process and how long will it take,
benefits and risks of a study, and is written
clearly, so participants can understand it.
Methodology
1. Ethical approval and informed consent.
2. Inclusion and exclusion criteria: clinical trials have
guidelines about who will participate, using Inclusion and
exclusion criteria.
• Inclusion criteria: are the factors that allow one to
participate in the trial;
• Exclusion criteria: are the factors that disallow one to
participate in the trial;
• These criteria are based on factors such as: age, gender,
type and stage of diseases, previous treatment & other
medical conditions;
• These criteria help to reduce the amount of variation in
the study, without threatening the scientific integrity of
the trial, by removing medical variations that might
complicate analyzing the results.
3- Placebo:
• The control group receives dummy treatment
which is indistinguishable from the treatment
under study.
• A low-dose group: for the situation when a
placebo group can not be used because of
ethical issues.
4- Methods of Blinding
a- In a single-blinded trial, only the patient is
unaware of the treatment;
b- Double-blinded means that neither the patients
nor the investigator is aware of the treatment. (to
prevent assessment and response biases).
However, in several fields, such as surgery, it may
be impossible to run a double-blinded study.
c- In a triple-blinded trial, patient, investigator and
data-monitoring body are unaware of the
treatment group;
• While in open trials, the investigators and patients
know what treatment each patient takes.
5- Evaluation and endpoint measurements.
6- Protocol deviations.
Protocol of a clinical trial includes the following:• Protocol of a clinical trial includes the following:
1- Definition
2- Importance
3- Format:
(a) Title page;
(b) Background and hypothesis;
(c) Study design;
(d) Materials and methodology;
(e) Data collection and handling; (f) Statistical analysis;
(g) References;
(h) Summary.
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