What is a clinical trial?

The clinical trial process depends on the kind of trial being conducted. The clinical trial team includes doctors and nurses as well as social workers and other health care professionals. They check the health of the participant at the beginning of the trial, give specific instructions for participating in the trial, monitor the participant carefully during the trial, and stay in touch after the trial is completed.

Some clinical trials involve more tests and doctor visits than the participant would normally have for an illness or condition. For all types of trials, the participant works with a research team. Clinical trial participation is most successful when the protocol is carefully followed and there is frequent contact with the research staff.

Ideas for clinical trials usually come from researchers. After researchers test new therapies or procedures in the laboratory and in animal studies, the experimental treatments with the most promising laboratory results are moved into clinical trials. During a trial, more and more information is gained about an experimental treatment, its risks and how well it may or may not work.

There are many reasons why people volunteer to take part in a clinical trial:

• Some volunteers have a condition that is being treated effectively but want to help doctors find out more about it in order to develop new treatments.
• Sometimes volunteers have a friend or loved one with an illness or injury and they want to participate in a research study as a way of helping that person and others who may suffer from the same condition.
• It may be that there is no effective treatment for an illness or injury or the existing treatment has harsh side effects. Participating in a clinical trial may offer potential treatment options that might otherwise be unavailable.
• It may be that there is no effective treatment for an illness or injury or the existing treatment has harsh side effects. Participating in a clinical trial may offer potential treatment options that might otherwise be unavailable.
• Many people choose to participate in a study even though it might not be able to help them directly. Knowing that others may be able to benefit from their efforts is a rewarding experience.
• Sometimes people volunteer because they are compensated for their time and effort.

No, you don’t have to have a specific illness or condition to participate in a clinical trial. In fact, there are many trials that need healthy volunteers. Participants who are healthy are often needed to provide information that can be compared with people who have a particular disease.

Every clinical trial includes guidelines about who is eligible to participate. The guidelines are based on factors such as your age, gender, whether or not you have a certain disease and your previous treatment or medical history.

Some of the ways to determine if a clinical trial is right for you include:

• Comparing the reason why you’re interested in participating to the question the trial hopes to answer. You should think about whether the trial has the potential to fulfill your hopes and expectations. You should also weigh the potential risks and benefits of participating in the trial.
• Informing yourself about the questions the trial is trying to answer by researching on your own through reputable websites and the library and by asking questions. You may also want to talk to your family, friends or your personal physician.

YCCI staff members, study doctors, nurses and coordinators are available to answer your questions so that you can make an informed decision.

You should know as much as possible about the trial and feel comfortable asking the research team any questions about what’s involved. The following questions may be helpful:

What is the purpose of the trial?
Who is going to be in the study?
How many volunteers are needed for the study?
How long will the trial last?
Why do researchers believe the treatment being tested may be effective?
What drugs, devices, procedures, tests, examinations and visits are required in order to participate?
Will I have to change my daily activities or diet if I participate?
Have there been other trials similar to this one?
What are the standard treatment options for my condition if I choose not to participate?
Are there other clinical trials for which I might be eligible?
What are the potential risks of this trial?
What are the expected benefits to me if I participate?
Who will pay for the study treatment?
Will I incur any costs?
Will I be reimbursed for expenses or paid for my participation?
Will the results of the trial or tests and procedures be available to me?
Who will be in charge of my care?
Whom should I contact if I have questions about the trial?

As with most medical treatments, there may be some risks to participating in a clinical trial. Possible risks include the following:

• Your participation may require more time and attention than routine clinical care. This may involve visits to the study site, phone calls from the study doctor or staff, frequent treatments or procedures, answering questions, a hospital stay or a more complicated drug regimen.
• The treatment or procedure may not be effective for every individual.
• The treatment being studied may involve potential unpleasant or serious side effects.

The sponsor of the research usually pays for all research-related costs. If a study involves routine tests, treatments or procedures that you would normally undergo as part of the standard course of treatment for your condition, then you or your insurance company may be asked to cover these fees. Before the trial begins, be sure to ask the trial staff which fees may be billable to you or your insurance company.

Clinical trials are sponsored or funded by a variety of organizations or individuals such as physicians, medical institutions, foundations, voluntary groups, and pharmaceutical companies, in addition to federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD), and the Department of Veteran's Affairs (VA). Trials can take place in a variety of locations, such as hospitals, universities, doctors' offices, or community clinics.

Informed consent is the process of 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 information for participants. To help someone decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study. If the participant's native language is not English, translation assistance can be provided. Then the research team provides an informed consent document that includes details about the study, such as its purpose, duration, required procedures, and key contacts. Risks and potential benefits are explained in the informed consent document. The participant then decides whether or not to sign the document. Informed consent is not a contract, and the participant may withdraw from the trial at any time.

A protocol is a study plan on which all clinical trials are based. The plan is carefully designed to safeguard the health of the participants 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, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.

Most human use of investigational new drugs takes place in controlled clinical trials conducted to assess safety and efficacy of new drugs. Data from the trials can serve as the basis for the drug marketing application. Sometimes, patients do not qualify for these carefully-controlled trials because of other health problems, age, or other factors. For patients who may benefit from the drug use but don't qualify for the trials, FDA regulations enable manufacturers of investigational new drugs to provide for "expanded access" use of the drug. For example, a treatment IND (Investigational New Drug application) or treatment protocol is a relatively unrestricted study. The primary intent of a treatment IND/protocol is to provide for access to the new drug for people with a life-threatening or serious disease for which there is no good alternative treatment. A secondary purpose for a treatment IND/protocol is to generate additional information about the drug, especially its safety. Expanded access protocols can be undertaken only if clinical investigators are actively studying the experimental treatment in well-controlled studies, or all studies have been completed. There must be evidence that the drug may be an effective treatment in patients like those to be treated under the protocol. The drug cannot expose patients to unreasonable risks given the severity of the disease to be treated.

Some investigational drugs are available from pharmaceutical manufacturers through expanded access programs listed in ClinicalTrials.gov. Expanded access protocols are generally managed by the manufacturer, with the investigational treatment administered by researchers or doctors in office-based practice. If you or a loved one are interested in treatment with an investigational drug under an expanded access protocol listed in ClinicalTrials.gov, review the protocol eligibility criteria and location information and inquire at the Contact Information number.

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. In some studies, the participants in the control group will receive a placebo instead of an active drug or experimental treatment.

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.