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HIV Clinical Trials Basics
Participating in a clinical trial gives you an opportunity
to contribute to knowledge that becomes the standard
of care for the treatment and prevention of HIV nationally
and globally. With the collaboration of your physician
we will explore the current issues in the ever changing
world of HIV care. HIVinfoSource is dedicated to helping
you find an appropriate clinical trial and to understanding
what it means to volunteer for a study.
What is a clinical
trial?
Why participate
in a clinical trial?
Where do the ideas for
trials come from?
Who sponsors
clinical trials?
What is a protocol?
What is a control
or control group?
What
are the different types of clinical trials?
What are the phases of
clinical trials?
What is an "expanded
access" protocol?
Who can participate
in a clinical trial?
What happens if
I join a clinical trial?
What is informed
consent?
What kind
of preparation should a potential participant make for
the meeting with the research coordinator or doctor?
What should
I consider before deciding to join a trial?
Do I continue to
work with a primary health care provider while in a
trial?
What are side effects
and adverse reactions?
What are the
benefits and risks of participating in a clinical trial?
How is my safety
protected?
Can I leave a clinical
trial after it has begun?
A clinical trial is a research study designed to answer specific questions about vaccines, new therapies or new ways of using known treatments. Clinical trials (also called “medical research” and “research studies”) are used to determine whether new drugs or treatments are both safe and effective. Carefully conducted clinical trials are the fastest and safest way to find effective treatments.
Why participate in a clinical trial?
Volunteers in clinical trials can play a more active role in their own health care, as well as gain access to new research treatments before they are widely available. More importantly, when you participate in a clinical trial you help others by contributing to medical research and knowledge.
Through AIDS clinical trials, much has been learned about how men and women are affected by HIV, both physiologically and socially. We have also learned a great deal about how the disease manifests itself in women and men. For example, we have learned HIV infection is spread more efficiently in women than in men.
Great efforts are underway to find drugs that will benefit men and women in preventing and treating HIV. We also know that HIV manifests itself differently in children than adults. Therefore, when we are studying a drug, it is important to understand how it affects different populations. That’s why it’s important that people consider donating their time to volunteering in a clinical trial.
Where do the ideas for clinical trials come from?
Ideas for clinical trials usually come from researchers and sometimes from our patients. After researchers test new therapies or procedures in the laboratory and in animal studies, the treatments with the most promising laboratory results are moved into clinical trials. During a trial, more and more information is gained about a new treatment, its risks and how well it may or may not work.
Clinical trials are sponsored or funded by a variety of organizations such as the National Institutes of Health (NIH) (which is part of the United States government), medical institutions, foundations, and pharmaceutical companies.
NYU School of Medicine is one of the original participants of the Adult AIDS Clinical Trials Group (Adult ACTG), the largest HIV clinical trials organization in the world. NYU also participates in the Pediatric AIDS Clinical Trails Group via the NIH. These groups play a major role in setting standards of care for HIV infection and opportunistic diseases related to HIV/AIDS throughout the world. The ACTG has been pivotal in providing the data necessary for the approval of therapeutic agents, as well as the treatment and prevention strategies, for many opportunistic infections and malignancies. The ACTG is composed of, and directed by, leading clinical scientists in HIV/AIDS therapeutic research.
A protocol is a study plan on which all clinical trials are based. The plan is carefully designed to safeguard the health of the volunteers as well as answer specific research questions. 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 in a clinical trial, study participants are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.
What is a control or control group?
A control is the standard by which experimental observations are evaluated. In many clinical trials, one group of patients will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.
What are the different types of clinical trials?
Treatment
trials test new treatments, new combinations of
drugs, or new approaches for improving the effectiveness
of current medication combinations.
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, vaccines, or behavior changes.
Screening
trials test the best way to detect certain diseases
or health conditions.
Quality
of Life trials explore ways to improve comfort and
the quality of life for individuals with a chronic illness.
Example of quality of life trials include: medication
schedules that are easier to live with [fewer pills,
less often], fewer side effects.
What are the phases of clinical trials?
Clinical trials are conducted in phases. The trials
at each phase have a different purpose and help scientists
answer different questions:
In Phase I
trials, researchers test a new drug or treatment
in a small group of people (20-80) for the first time
to evaluate its safety, determine a safe dosage range,
and identify side effects.
In Phase II
trials, the study drug or treatment is given to
a larger group of people (50-150) to see if it is effective
and to further evaluate its safety.
In Phase III
trials, the study drug or treatment is given to
large groups of people (400-2,000) 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 and to be available by physician
prescription.
In Phase IV
trials, after the medication is available by prescription,
studies delineate additional information including the
drug's risks, benefits, and optimal use.
What is an Expanded Access protocol?
Occasionally after a drug has proved to be safe and effective, but before it is available by prescription, it can be obtained under an expanded access program sponsored by the manufacturer.
Participation in Clinical Trials
Choosing to participate in a clinical trial is an important personal decision. The following frequently asked questions provide introductory information about clinical trials. In addition, it is important to discuss participating in a trial with your primary care physician. It is also often helpful to talk to family members or friends about deciding to join a trial. After identifying some trial options, the next step is to contact the study research staff and ask questions about specific studies.
Who can participate in a clinical trial?
All clinical trials have guidelines about who can participate. Using inclusion / exclusion criteria is an important principle of medical research that helps to produce reliable results. The factors that allow someone to participate in a clinical trial are called "inclusion criteria" and those that disallow someone from participating are called "exclusion criteria". These criteria are based on such factors as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions. Before joining a clinical trial, you must qualify for the study. Some research studies seek volunteers with illnesses or conditions to be studied in the clinical trial, while others need healthy participants. It is important to note that inclusion and exclusion criteria are not used to reject people personally. 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.
What happens if I join a clinical trial?
The clinical trial process depends on the kind of trial being conducted (See What are the different types of clinical trials?) The clinical trial team includes doctors, nurses and other health care professionals. They check your health at the beginning of the trial, give you specific instructions for participating in the trial, monitor you carefully during the trial, and stay in touch with you after the study is completed.
Some clinical trials involve more tests and doctor visits than would normally have for an illness or condition. For all types of studies you work with a research team. Clinical trial participation is most successful when the protocol is carefully followed and you have frequent contact with the research staff.
Informed consent is the process of your learning the key facts about a clinical trial before deciding whether or not to participate. It is also a continuing process throughout the study to provide you with important information. To help you decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study. If your native language is not English, we will provide a translator. Then our staff will provide you with an informed consent document that includes details about the study, such as its purpose, duration, required procedures, and key contacts. The possible risks and potential benefits of participating in the study are explained in the informed consent document. The staff will review the consent thoroughly with you and make sure all your questions are answered before you decide whether or not to sign the consent. The Informed Consent is not a contract, and you may withdraw from the trial at any time.
What kind of preparation should a potential participant make for the meeting with the research coordinator or doctor?
- Plan ahead and write down possible questions to ask.
- Ask a friend or relative to come along for support and to hear the responses to the questions.
- Bring a tape recorder to record the discussion to replay later.
What should I consider before deciding to join a trial?
You should know as much as possible about the clinical trial before you decide to participate. It is important that you feel comfortable asking the members of the study staff questions about the studyit, including what will be expected of you and what the risks and benefits to you will be.
The following questions might be helpful for you to discuss with the study team. Some of the answers to these questions are found in the informed consent document.
- What is the purpose of the study?
- Who is going to be in the study?
- Why do researchers believe the new treatment being tested may be effective? Has it been tested before?
- What kinds of tests and treatments are involved?
- How do the possible risks, side effects, and benefits in the study compare with my current treatment?
- How might this trial affect my daily life?
- How long will the trial last?
- Will hospitalization be required?
- Who will pay for the treatment?
- Will I be reimbursed for other expenses?
- What type of long-term follow up care is part of this study?
- How will I know that the treatment is working? Will results of the trials be provided to me?
- Who will be in charge of my care?
- Will participation in a study limit future treatment options?
Do I continue to work with a primary health care provider while in a trial?
Yes. You must have a primary care provider when you participate in a research study. Most clinical trials provide one-half to three year treatments related to a designated illness or condition, but do not provide extended or complete primary health care. Your primary care provider will be kept informed of the status of the study and (with your permission) will be sent copies of your laboratory results on a regular basis. In addition, by having your health care provider work with the research team, you can ensure that other medications or treatments will not conflict with the protocol.
What are side effects and adverse reactions?
Side effects occur with many HIV medications currently in use. They also may occur with medications being evaluated in clinical trials. Side effects are any undesired actions or effects of drug or treatment [which may include headache, nausea, rash, loose stools, or other physical problems].
What are the benefits and risks of participating in a clinical trial?
- Benefits
Clinical trials that are well-designed and well-executed are the best treatment approach for eligible participants to:- Play an active role in their own health care.
- Gain access to new research treatments before they are widely available.
- Obtain expert medical care at leading health care facilities during the trial.
- Help others by contributing to medical research.
- Take advantage of frequent monitoring of your health status and early detection of complications
- Help you with taking medicines correctly
- Obtain free lab work and often free trial medicines
- Have personalized, friendly, confidential care
- Risks
There are risks to clinical trials.- There may be unpleasant, serious side effects to treatment when studying very new medications that have not been used before.
- The treatment may not be effective for you.
- The protocol may require more of your time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays or complex dosage requirements.
Your safety is the most important thing to us when you participate in a clinical trial.
The ethical and legal codes that govern medical practice also apply to clinical research. In addition, most clinical research is federally regulated with built in safeguards to protect the participants. The trial follows a carefully controlled protocol designed to detect any laboratory or physical abnormality as early as possible. The safety and progress of the study are reviewed by an external data and safety monitoring board, composed of physicians and statisticians who are not part of the clinical trials group. When the study is completed, researchers report the results of the trial the participants and their care providers. The results are also reported at scientific meetings, in medical journals, and provided to the NIH and Food and Drug Administration. Individual participants' names will remain secret and will not be mentioned in these reports.
Every clinical trial in the U.S., including NYU School of Medicine, must be approved and monitored by an Institutional Review Board (IRB) to make sure the risks are as low as possible and are worth any potential benefits. An IRB is an independent committee of physicians, statisticians, community advocates, and others that ensures that a clinical trial is ethical and that the rights of study participants are protected. All institutions that conduct or support biomedical research involving people must, by federal regulation, have an IRB that initially approves and periodically reviews the research.
Ways that you can protect your rights as a study volunteer:
- Carefully read the informed consent document.
- Ask questions of the research staff.
- Ask to speak with a study volunteer.
- Join the Community Advisory Board.
- Remember that the choice to be a participant in a clinical study is always voluntary.
Can I leave a clinical trial after it has begun?
Yes. You can choose to leave a clinical trial, at any time. When withdrawing from the trial, you should let the research team know about it, along with your reasons for leaving the study.