Patient Info: Frequently Asked Questions

A clinical trial (also referred to as clinical research) is a research study involving human volunteers to help answer specific health questions. Carefully conducted clinical trials are the fastest and safest way to find treatments that work in people and ways to improve health. Interventional trials determine whether experimental treatments or new ways of using known therapies are safe and effective under controlled environments. Observational trials address health issues in large groups of people or populations in natural settings.

Participants in clinical trials can play a more active role in their own health care, gain access to new research treatments before they are widely available, and help others by contributing to medical research.

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. 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, a participant must qualify for the study. Some research studies seek participants 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.

The clinical trial process depends on the kind of trial being conducted (see below.) 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 prior to participating in the trial, monitor the participant carefully during the trial, and stay in touch with the participant 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. (See question #10 for more information on protocals.)

• Diagnostic trials are conducted to find better tests or procedures for diagnosing a particular disease or condition. These trials usually include people who have signs or symptoms of the disease or condition being studied.
• Open Label trials are studies in which both the doctor and participant knows which study treatment is being given to the participant.
• 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.
• Quality of Life trials (or Supportive Care trials) explore ways to improve comfort and the quality of life for individuals with a chronic illness.
• Randomized Trials are studies in which partipants are randomly assigned to a treatment arm as part of a study. Occasionally, a placebo is utilized as part of a randomized trial. Some randomized trials are blinded studies and some are "double-blinded."
• Screening trials test the best way to detect certain diseases or health conditions.
• Treatment trials test new treatments, new combinations of drugs or new approaches to surgery or radiation therapy.

Clinical trials are conducted in phases. The trials at each phase have a different purpose and help scientists answer different questions.

• 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.
• Phase II trials — the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
• Phase III trials — the study drug or treatment is given to large groups of people (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 — post marketing studies delineate additional information including the drug's risks, benefits and optimal use.

Ideas for clinical trials usually come from researchers. 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 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). Sometimes researchers also perform unfunded, independent research as part of a university research center. Trials can take place in a variety of locations, such as hospitals, universities, doctors' offices or community clinics.

An IND is a new drug, antibiotic drug or biological drug that is used in a clinical investigation. In the U.S., the Food & Drug Administration (FDA), must approve a substance as a drug before it can be marketed. The approval process involves several steps including preclinical laboratory and animal studies, clinical trials to study safety and effectiveness, and filing of an Investigational New Drug application.

The human use of INDs take place in controlled clinical trials which are conducted to assess the safety and efficacy of new drugs and the conduct of these studies is governed by the FDA. Once the required clinical trials have been completed, a New Drug Application (NDA) must be submitted by the manufacturer of a drug to the FDA in order to obtain a license to market the drug commercially.

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.

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 treatment's effectiveness. In some studies, the participants in the control group (see below) will receive a placebo instead of an active drug or 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.

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.

BENEFITS — Clinical trials that are well-designed and executed are the best 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.

RISKS — There are risks to clinical trials, such as:

• Possible unpleasant, serious or even life-threatening side effects to treatment.
• Treatment may not be effective for the participant.
• Protocol may require more of their time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays or complex dosage requirements.

Side effects and adverse reactions are any undesired actions or effects of drug or treatment. Negative or adverse effects may include headache, nausea, hair loss, skin irritation or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects.

The ethical and legal codes that govern medical practice also apply to clinical trials. In addition, most clinical research is federally regulated with built in safeguards to protect the participants. The trial follows a carefully controlled protocol, a study plan which details what researchers will do in the study. As a clinical trial progresses, researchers report the results of the trial at scientific meetings, to medical journals and to various government agencies. Individual participants' names will remain secret and will not be mentioned in these reports.

People should know as much as possible about the clinical trial and feel comfortable asking the members of the health care team questions about it. The following questions might be helpful for the participant to discuss with the health care team. Answers can be found in the Informed Consent document to many of the following questions:

• 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?
• What kind of care can I expect to receive while in the trial?
• How might this trial affect my daily life?
• How long will the trial last?
• Will hospitalization be required?
• What is the cost of the trial?
• 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?

• 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.

Yes. Most clinical trials provide short-term treatments related to a designated illness or condition, but do not provide extended or complete primary health care. In addition, by having the health care provider work with the research team, the participant can ensure that other medications or treatments will not conflict with the protocol.

Yes. A participant can leave a clinical trial at any time. When withdrawing from the trial, the participant should let the research team know about it, along with the reasons for leaving the study.