FREQUENTLY ASKED QUESTIONS

What is a clinical trial?

A clinical trial is a study of a research study utilizing volunteer subjects to help in answering specific health questions. The purpose of the clinical trial is to answer questions about the safety and efficacy of trial medications and/or new techniques of using known therapies that are safe and effective under controlled environments. A clinical trial is used to help the Food and Drug Administration (FDA) determine whether or not the trial treatment or medication should be approved for use by the general public.

Why do people participate in clinical trials?

A common reason for participation is the lack of a generally accepted therapy for a patient's ailment, or in the alternative, the partial ineffectiveness of available therapies. Participating in a clinical trial may offer the patient a new alternative to “standard” treatment before it would become available to the general public. Many people volunteer for participation because they have a particular health problem, such as osteoporosis, diabetes or high blood pressure, and want to do all that they can to prevent such medical problems and complications for their children. Others want to contribute to research efforts in hopes to help others in the future.

Participation in a clinical trial also brings the patient into a close doctor-patient relationship and provides patients with ongoing and continuous medical treatment for the duration of the study, free of charge. Participation in many of the trials can also provide a monetary benefit to the patient for their time and effort involved with the study.

Why participate in a clinical trial?

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 other by contributing to medical research.

Who can participate in a clinical trial?

All clinical trials have different guidelines set out as to who will be able to participate. Using selective criteria to determine eligible study participants will help to produce reliable results. The factors that allow someone to participate in a clinical trial are called “inclusion criteria.” The factors that would exclude someone from participation in a clinical trial are called “exclusion criteria.” These criteria are based on a number of factors including age, gender, the type and stage of disease, previous treatment history and the presence or absence of other medical conditions. Before joining a clinical trial, a potential 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 re used to identify appropriate participants for that particular trial and ensure each patient's safety. The criteria will also help researchers to answer the questions they plan to study.

What happens during a clinical trial?

The clinical trial process depends on the kind of trial being conducted. The clinical trial team includes a doctor(s) and a clinical research coordinator(s). The team works together to check the health of the participant at the beginning of the trial, give specific instructions for participation in the trial, monitor the participant carefully throughout the trial's duration and stay in touch with the participant after the trial is completed.

For all types of trials, the participant works with a research team. Clinical trial participation is most successful when the study guidelines (protocol) are carefully followed and there is frequent contact with the research staff.

What is informed consent?

Informed consent is the process of learning the key facts about a clinical trial before deciding whether or not to participate. It is a continuing process through the study to provide all up-to-date information to study participants. To help an individual decided whether or not to participate, the doctors and nurses involved in the trial will explain the details of the study. The research team then provides an informed consent document that includes details about the study such as its purpose, duration, required procedures, visit summaries and key contacts. Risks and potential benefits are also explained in the informed consent. After reviewing the informed consent, the potential participant may then decide whether he or she will sign the document and agree to participate in the trial. Informed consent is not a contract and the participant may withdraw from the trial at any time.

What are the potential benefits and risks of participating in a clinical trial?

• Play an active role in their own health care;
• Gain access to new research treatments before they are widely available;
• Help others by contributing to medical research;
• Obtain expert medical care throughout the duration of the trial .

• There may be unpleasant side effects to treatment; The treatment may not be effective for every participant;
• The trial guidelines (protocol) may require more time and attention than a non-trial treatment option, including trips to the trial site, more tests, or complex dosage requirements.

What are side effects and adverse reactions?

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

How is the safety of the participant protected?

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 carefully controlled guidelines (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.

What should people consider before participating in a trial?

People should know as much as possible about the clinical trial and feel comfortable asking the members of the research team questions about it and the care expected while participating in the trial. The following questions might be helpful for the participant to discuss with the research team. Some of the answers to these questions may be 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 experimental treatment being tested may be effective? Has it been tested before?
• What kinds of tests and experimental 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 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 experimental treatment is working? Will results of the trials be provided to me?
• Who will be in charge of my care?

Does a participant continue to work with a primary health care provider while in a trial?

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 trial guidelines (protocol).

Can a participant leave a clinical trial after it has begun?

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, as well as his or her reason(s) for leaving the trial.

Where do the ideas for trials come from?

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.

Who sponsors clinical trials?

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, research clinics or community clinics.

What is a protocol?

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.

What is a placebo?

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.

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 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 an experimental 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 experimental 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.

In Phase III trials, the experimental 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 experimental drug or treatment to be used safely.

In Phase IV trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.

Lillestol Research conducts both Phase III and IV clinical trials.

© An Introduction to Clinical Trials. 18 September 2006. National Library of Medicine .