Many of the progress in diagnosis, therapy, and
clinical care provided to patients affected by end-stage liver,
kidney, and cardiac diseases leading to various forms of treatment,
including transplantation, was achieved thanks to clinical research
studies called "clinical trials", "clinical studies"
or "experimental protocols". Clinical trials are designed
to help health care providers to develop new diagnostic, therapeutic,
or prevention approaches for specific pathologies.
Treatments for patients affected by end-stage diseases have
been developed thanks to highly advanced diagnostic tests and
new treatments. Clinical trials studying the impact of different
diagnostic/treatment methods such as imaging, surgery, radiotherapy,
chemotherapy, cellular and genetic therapy have allowed clinicians
to identify more advanced techniques to treat these diseases.
Clinicians often ask patients to participate in
a clinical trial, or patients may receive information on clinical
trials in progress at specialized centers. The choice to participate
in a clinical trial represents an important and personal decision.
Patients and their families should review all the information
to make an informed decision. It is often useful to consult your
family doctor with your family and friends when deciding whether
or not to participate in a trial. Having identified the available
open trials, the next step is to contact the research staff for
information on specific trials.
This section provides general information on clinical trials,
some FAQs, and a list of current trials at ISMETT/UPMC Italy.
What is a clinical trial?
A clinical trial is an experimental study set to answer specific
questions about new therapies or procedures, or new ways to
use known treatments. Clinical trials are used to determine
whether new drugs or treatments are both safe and effective.
Carefully conducted trials are fastest and safest way to find
effective treatments. The idea of starting a clinical trial
usually comes from researchers and is carried out in compliance
with a series of strict rules (the Protocol). After testing
new therapies or procedures in the laboratory, and if results
are promising, researchers begin planning Phase 1 clinical trials.
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What is a protocol?
All clinical trials are based on a set of rules called
a protocol. 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. While on a clinical
trial, participants are regularly seen by the research staff
to monitor their health and to determine the safety and effectiveness
of the treatment.
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Why are clinical trials important?
Clinical trials are an important contribution to knowledge
and to progress in the fight against many diseases. If a new
treatment tested in a trial proves to be effective, it may become
the new standard treatment helping many seriously ill patients.
Many of today's most effective therapies are the result of long
experimental clinical studies that often open new paths for
future research. The patients participating in a clinical trial
are the first to benefit from the results.
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What happens in a clinical trial?
During a clinical research study, patients are treated
and a medical team assesses the treatment's efficacy. Just as
there are potential risks for participants, every study also
envisages all the necessary safety measures to prevent or tackle
them. When patients take part in a clinical trial, the treatment
is administered in a hospital, clinic, or outpatient unit. Physicians,
nurses, social workers, and other clinicians provide assistance
following patients closely. Frequent check-ups and lab tests
are done within the framework of a set plan of treatment. Patients
play an active role in the trial by documenting their health
conditions. Patients are closely followed even after the end
of the treatment envisaged by the trial.
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How is a clinical study performed? What are
the safety measures for patients?
In a clinical trial the most important aspect is the patient
and his/her well-being, THEN come the trial issues. Science
and medicine are at the patient's service, NOT vice versa. To
protect patients and obtain good results, research is carried
out in strict compliance with all ethical and scientific principles.
Every trial must have a protocol (plan of action) providing
a detailed explanation of all the activity, why and how it will
be done, how many people can participate, which medical tests
are necessary, how frequently these must be repeated, and the
plan of care. To guarantee patient safety, all protocols must
be approved by an Ethics Committee.
All protocols describe the characteristics that
patients must have to enroll in the trial. These characteristics
are the eligibility criteria. They vary from trial to trial
and depend on the target of the research. They include age,
sex, type and stage of the disease, and whether or not patients
have received previous treatment. Strict compliance with eligibility
criteria safeguards patients against risks related to the use
of new drugs or treatments in people with specific characteristics.
In addition, once results are obtained, the eligibility criteria
help researchers identify patients who can benefit from a new
treatment, once its effectiveness is proven. For instance, a
new treatment may be effective against one type of cancer and
not for another, or may be more effective in men than in women.
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What are clinical trial phases?
Clinical trials of experimental
drugs or therapies proceed through four phases:
In Phase-1 clinical trials, researchers test
a new drug or treatment for the first time in a small number
of people (20-80) to evaluate its safety, determine a safe dosage
range, and identify side effects.
In Phase-2 clinical trials, the drug or treatment
is administered to a larger group of people (100-300) to further
assess its safety and effectiveness.
In Phase-3 clinical trials, the drug or treatment
is administered to large groups of people (1,000-3,000) to further
determine effectiveness, monitor side effects, compare it to
commonly used treatments, and collect information that will
allow to use the drug or treatment safely.
The Phase-4 clinical trials are performed after
the drug or treatment has been authorized for medical prescription
and has been marketed. These studies continue testing the drug
or treatment to collect information on the effect in various
populations and any side effects associated to long-term use.
Patients are randomly assigned to the group receiving the
new treatment (treatment group) or to the standard group (control
group) to ensure the trial's impartiality.
In some studies (blind trials) researchers do not
tell patients which group they have been assigned to. This is
a way to eliminate any risk of errors in interpreting the information
and ensure its objective assessment. At times, knowing the drug
being taken may affect the reaction to it. For instance, patients
who are aware they are receiving a new treatment may expect
specific positive effects and report promising signs because
they want to believe they are feeling better. In this case,
trial objectivity would be impaired and the results described
would be better than they actually are.
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What is a blinded or masked trial?
A blinded or masked trial is one in which participants
do not know whether they are in the experimental or control
group. Patients in the experimental group receive the drugs
or treatments being tested, whereas those in the control group
receive a standard treatment or no treatment.
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What is a double-blind or double-masked trial?
A double-blind or double-masked trial is one
in which neither the participants nor the research team know
which participants are receiving the experimental treatment
and which ones are receiving either a standard treatment or
a placebo. The purpose of these trials is to avoid that the
expectations of patients and physicians alike on the experimental
drug or therapy may affect the outcome.
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How are patients participating in a trial
safeguarded?
Strict guidelines protect people who choose to participate
in clinical trials. All clinical trials carried out at ISMETT
must be approved and monitored by the Institutional Review Board
(IRB) and by the Ethics Committee (EC) to make sure the risks
are as low as possible and are worth any potential benefits.
An IRB is a scientific committee that ensures
the scientific content of a clinical trial, its scientific importance,
the cogency of the hypothesis, appropriateness of the experiment
plan, statistical analysis, adequacy of participants and feasibility
with regard to the completion of the trial within a reasonable
time-frame.
An EC is an independent committee composed of
physicians, pharmacists, nurses, experts in bioethics, legal
affairs, patients’ rights, and other experts that guarantees
the ethic content of the trial and the safeguard of the rights
of all participants. By law, all institutions performing or
supporting biomedical research on humans must have an EC giving
its initial approval to a study and to review it on a periodic
basis.
Patients must be fully aware they have rights
both before they give consent to participate in a clinical research
study and during treatment:
Participating to a trial is a choice. You can
discuss it with your physician and decide together the best
treatment.
Throughout the trial physicians and nurses
closely follow the patient's response to the treatment.
If researchers realize the treatment may have
negative effects, the patient is immediately excluded from the
trial.
Patients can discuss with physicians whether
it is possible to receive a different treatment.
Patients are entitled to leave the trial
at any time.
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What is informed consent?
Informed consent is the process
of learning the key facts about a clinical trial before you
decide whether or not to participate. These facts include:
The goal of the research
The goals of the researchers
What will be done during the trial and for how long
What risks are involved in the trial
What benefits can be expected from the trial
What other treatments are available
The fact the patient has the right to leave
the trial at any time
If a patient is considering joining a clinical trial, the
research staff will provide the informed consent that includes
the details of the study. If a patient is not a native Italian
speaker, he/she may ask for a translator or that the consent
be written in another language. Because joining a clinical trial
is an important decision, patients should ask any question on
informed consent before making a decision. We recommend patients
discuss the consent with their families and friends. Talking
about the various options with others can help make the decision
easier.
The person choosing to participate in a clinical
trial may at any time request a copy of the informed consent
for reference purposes. The informed consent is more than just
a form to sign. It is a process that covers the entire trial
and the patient's participation. Patients must feel free to
ask the research team any questions before, during, or after
the study.
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Who can participate in a clinical trial?
All clinical trials have guidelines about who can be included
in the program. Guidelines are based on such factors as age,
type of disease, medical history, and current medical conditions.
Before joining a clinical trial, one must qualify for the study.
Some research studies seek volunteers with specific illnesses
or conditions to be studied during the trial, while others need
healthy volunteers. Healthy volunteers participate in Phase
1 trials, some vaccine studies, and trials on research on prevention
care for pediatric or adult patients.
The factors that allow participation in a clinical
trial are called inclusion criteria, whereas the factors that
keep people from participating are called exclusion criteria.
It is important to note that inclusion and exclusion criteria
are not used to reject people for personal reasons. Instead,
the criteria are used to identify appropriate participants and
keep them safe. The criteria help ensure that researchers will
be able to answer the questions they plan to study.
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What is a placebo?
A placebo is an inactive tablet, liquid, or powder that
has no therapeutic effect. In clinical trials, experimental
treatments are often compared to placebos to assess the treatment's
effectiveness. In some studies, the participants in the control
group will receive a placebo instead of an active drug or treatment.
When there is no standard treatment for a disease the
new treatment is often compared to a placebo. Patients are informed
of the possibility they may receive a placebo before deciding
whether or not to enroll in the study.
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What is a control or a control group?
A control is the standard by which experimental observations
are evaluated. In many clinical trials, the treatment group
is administered an experimental drug or treatment, and the control
group is given a standard treatment for the illness or placebo.
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What are side effects or adverse reactions?
Side effects are any undesired actions or effects of a
drug or treatment. Experimental treatments are assessed for
both immediate and long-term side effects.
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What are the benefits and risks associated
with clinical trials?
Benefits of clinical trials:
Play an active role in your treatment
Gain access to new treatments that are not available to the public
Obtain expert medical care at leading health care facilities during the trial
Help others by contributing to medical research
Risks associated with clinical trials:
Possible side effects or adverse reactions to drugs or treatments
The treatment may not be effective
The protocol may require a lot of your
time to reach the study site, treatments, hospital stays or
complex dosage requirements
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What should I know before I enroll in a clinical
trial?
You should know as much as possible about the trial. It
is important to feel at ease asking questions and the research
staff should be able to answer in a clear and comprehensible
manner. A list of typical questions is provided below.
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How should I prepare for a meeting with a
coordinator or physician in charge of the research?
Prepare questions in advance
Ask a friend or relative to accompany you for moral support and to listen to the answers.
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What questions should I ask?
Here are some useful questions on performing a clinical trial:
Why is the trial being carried out?
What is the trial's purpose?
Who reviewed and approved the trial?
Why does the research team believe the therapy, drug, or medical device will be effective?
Here are some questions on trial participation:
Where will the trial be carried out?
What type of drugs, therapies, procedures, and/or tests will I receive or undergo during the trial?
Will these be painful? If so, for how long?
What is the difference between the tests envisaged by the trial and those I would do outside the trial?
How long will the trial last?
How often will I have to go to the study site?
Who will provide health care at the end of the trial?
Will I be able to take my usual medications during the trial?
What drugs, procedures, or treatments should I avoid during the trial?
What are my duties during the trial?
Will the trial require a hospital stay?
Will the trial research team cooperate with my family doctor during my participation in the trial?
Can others come to learn of my participation in the trial?
Can I speak to other trial participants?
Will I be informed of the trial's outcome?
What other treatment options do I have?
Questions on risks and benefits:
What's the difference between trial risks and benefits, compared to other approved therapies?
What are the possible immediate and long-term side effects?
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Can I leave a clinical trial after it has
begun?
Yes, you can leave a clinical trial at any time. If you
plan to stop participating in a clinical trial, let the researchers
know why you are leaving the study.
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