General Questions

What is a clinical trial?

A clinical trial studies people in a systematic way in order to answer a question. The purpose of a clinical trial is to find a better way to detect, prevent, control, or cure a disease or disorder. Some trials test the effects of treatments on disease, some aim to prevent disease, others study the effects of the disease itself (quality of life, costs, comfort levels). All of these types of trials may be found at the Center for Cardiac & Vascular Research at Washington Adventist Hospital.

The search for better treatment begins with basic research, in laboratories or in animals. This type of research points out methods likely to provide positive outcomes and, as much as possible, shows how to use them safely and effectively. This early research cannot predict exactly how a new treatment will work in people. Thus the clinical trial is done, in which investigators try to duplicate in people the promising results from basic research.

Clinical trials are primarily designed to test the effects of a new or existing treatment on an illness or disorder. The treatment can be a drug, a biological agent, a medical device, or a behavioral change, but it needs to have shown some promise in treating or preventing the health problem before it can be tested in a clinical trial.

Clinical trials are carefully and ethically designed to protect patients from unnecessary side effects and to allow truthful and precise collection and analysis of information to find out more about a disease. In general, clinical trials offer hope for the successful treatment of a disease or illness for future patients.

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Why are clinical trials important?

Advances in medicine and science result from new ideas or approaches developed through research. However, any new treatment can carry risks as well as benefits. Clinical trials help identify how safe and effective a treatment is for its intended use. In the U.S., new drugs or devices must be shown to be safe and effective in clinical trials of a certain number of patients before the Food and Drug Administration (FDA) can approve them for use. Until then, they are considered investigational.

For existing treatments, clinical trials can find out which might be the best for certain types of patients, which might be the most cost-effective, or which might be easier to give. Trials also can help confirm or refute common medical beliefs. For example, aspirin's protective effects against heart attack have been proven time and again. On the other hand, although "everybody knows" that a diet high in fiber can prevent colon and rectal cancer, a recent study has shown that this conventional wisdom may be wrong.

In addition, participation in a clinical trial helps manufacturers make informed decisions about whether to pursue getting a particular drug approved by the Food & Drug Administration (FDA). Finally, data from one patient (who completes the entire study) can be an important part of a drug development program. In the best case scenarios, these data can help get an exciting new drug approved by the FDA, and ultimately, continue the industry's efforts in developing medications that are safer, more effective, and work faster than any before them.

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How are clinical trials conducted?

Each trial is conducted under a protocol, a guide that spells out what is to be done, how it is to be done, and why. Protocols incorporate ways to answer research questions and to safeguard the medical and mental health of patients. Some trials test just one treatment in a group of patients. More typically, a trial will include two or more groups who are as similar as possible, each of which receives a different treatment option: the treatment of interest, a standard treatment for the disease (an active control), an inactive substance (a placebo control), or no treatment at all. All of the groups are then monitored in exactly the same way.

Several things (besides the treatment) can influence (or bias) the results of the study. For example, if the groups being studied are very dissimilar to begin with, any differences between them at the end of the study may simply be an extension of these baseline imbalances. If the groups are as similar as possible to each other before treatment, it is more likely that the treatment alone was responsible for any later differences (good or bad) between groups. Another way to reduce the chance of a physician or patient biasing the study results is randomization. If a patient agrees to be randomized, he or she is assigned by chance to either a treatment or a control group. The researchers do not know which is better; both could be of equal benefit. If the patients are unaware of the treatment they are given, the study is said to be single-blinded. If the researchers also are unaware of which treatment they are giving, then the study is double-blinded.

If a study treatment is obviously not helping the patient, the doctor can decide to remove the patient from the study. The patient also can decide to withdraw and receive any other available standard care. There are regular reviews of the data from a trial, and the information is shared. If there is clear evidence that one of the treatments is harmful or ineffective, or if one treatment is substantially better than another, the study is stopped earlier than planned. All patients in the study are then treated according to the new information, if they and their doctors so choose. The results of the study often are published in medical journals such as the New England Journal of Medicine or elsewhere.

Throughout a trial, the patient's personal doctor is kept informed about the patient's progress. Patients are encouraged to maintain contact with their referring doctors.

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How are a participant's rights and safety protected?

The FDA is the governing agency that develops the policies and guidelines for all medical research, regardless of manufacturer, study phase, or drug type. There are also independent Institutional Review Boards (IRB) that review and approve all study-related documents, such as protocols, Informed Consent forms, physician credentials and eligibility, and patient recruitment materials, such as print advertisements and public service announcements.

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Why might I want to participate in a clinical trial?

There are many reasons that people participate in clinical trials:

• To contribute to research that could help others in the future
• To receive effective treatment for a disease
• To achieve personal goals (stop smoking, for example)
• To seek relief or to feel better
• To obtain medical care
• To have access to treatments that may not be available otherwise

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How can I find out about clinical trials?

We encourage anyone interested in obtaining more information about clinical trials being conducted at the Center for Cardiac & Vascular Research at Washington Adventist Hospital to call 301-891-5612 or your doctor about any studies he or she may be conducting or ones that they may know of in your area.

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Why are trials divided into phases?

Trials in each phase are designed to reveal certain information. Patients may be eligible for studies in different phases depending on their general condition and type of disease. More patients take part in the later-phase studies than in earlier ones.

In a Phase I study, a new treatment is given to relatively few people (20 to 100 healthy volunteers, typically). The researchers must find the best way to give a new treatment and how much of it can be given safely. They watch carefully for any harmful side effects. The treatment has been tested in laboratory and animal studies, but no one knows how people will react. Phase I studies may involve significant risks for this reason.

Phase II studies determine the treatment's effects on the disease or disorder. This phase of testing usually involves a few hundred patients, mostly those who have the disease or disorder of interest. These are usually randomized, controlled studies, so that the treatment's safety and effectiveness (efficacy) can be measured more accurately. This stage of drug development can last for up to 2 years.

Each new phase of study builds on information from previous phases. If a treatment is shown to be effective in treating the disease in Phase II, it moves on to Phase III testing. Here it is compared with a placebo or with standard treatment(s), to see which is more effective, or (in some cases) to see whether it is at least as good as an existing treatment (equivalent). These studies can go on for many years and can involve thousands of people with the disease or disorder. After Phase III trials, the drug or device company can request approval from the FDA to sell the drug in the U.S. as a treatment for the particular disease.

In Phase IV studies, the treatment has been approved for a given use by the FDA, but certain aspects of the treatment are examined in particular situations. For example, a new drug may be tested in combination with other drugs, or in other patient groups (children. for example). Phase IV trials allow drug and device companies to identify side effects that may not appear until very large numbers of patients have received the treatment. Phase IV studies also are done to support other uses for the drug or device, for example, treatment of another disease.

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What happens after a study is complete?

After a study is complete, the FDA decides whether to continue or halt the drug's development. If it remains in development, the manufacturer may build in a "compassionate use" extension for the study. This means that eligible patients may receive the study drug for a predefined amount of time without having to undergo any typical study procedures, such as blood tests or clinical ratings. This usually occurs in later studies, such as Phase III and Phase IV (post-marketing surveillance).

In some cases, a study doctor may dispense approved medication samples and provide additional consultation free of charge. These are just a few of the ways he or she might show appreciation for a patient's involvement in a study.

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What Are My Rights as a Participant in a Clinical Trial?

Once you decide to participate in a clinical trial, you will need to sign an Informed Consent form. This form provides a detailed explanation of the purpose of the trial and the types of treatment to be studied. It also discusses the probability of your receiving a given treatment, the known risks and benefits of each treatment, and any costs related to the study that you would be expected to pay. The consent form also lets you know whom to contact more information, and fully explains your rights in a clinical trial, including:

• the right to confidentiality
• the right to medical treatment for any trial-related injury
• the right to withdraw from the study without penalty or loss of other medical care

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Is It Safe for Me to Participate in a Clinical Trial?

Before any clinical trial can begin to enroll patients, its protocol and Informed Consent form must be approved by the FDA. Then, at individual hospitals or research centers, approval also must be granted by an Independent Review Board. This is a committee of doctors and lay people who make sure that the potential risks do not outweigh the potential benefits of the study.

Before you agree to participate in a study, you should:

• Have a clear understanding of the study. The research staff must explain the goals, potential benefits, and potential risks of the study to your satisfaction.
• Check with your personal doctor, who knows your medical history and can help you decide whether you should participate in a clinical trial. Your doctor also can be a source of information about the risks and benefits of the proposed treatment(s).
• Have a good understanding about your current state of health. You will be told about the known side effects and other health risks of the treatment(s), but not all of the risks can be known when you decide to participate in a trial.

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What are some questions I may want to ask my study doctor?

• How long does the average trial last?
• What if I'm on other medication(s) from my regular doctor?
• What are the risks involved?
• Are there any tests requiring painful shots or blood samples? What should I do if I want a friend/parent to join a study?
• What kinds of medical problems would prevent participation in the clinical trial?
• Do I have to talk to my health insurance company before enrolling in a study?
• What if I miss a dose?
• Can I drop out at any time?
• Are there placebos involved?

If you decide that a clinical trial may be right for you, make a list of questions and discuss them with the study doctor at your initial visit.

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The Center for Cardiac & Vascular Research Washington Adventist Hospital 7600 Carroll Avenue, 6th Floor Takoma Park, MD 20912 (301) 891-6226 / 1-866-894-CCVR