What is a clinical trial?

A clinical trial is a carefully designed investigation to measure the effectiveness of a medical treatment in a particular group of patients. The medical treatment under investigation could be a pharmaceutical product (drug), medical device, or biotechnology therapy. All products tested in clinical trials are approved by the Food and Drug Administration (FDA).

A clinical trial is conducted under the close supervision of a physician investigator. It is a carefully controlled, step-by-step process that assures patients receive careful medical attention.

Why might a patient be interested in a clinical trial?

Patients might volunteer for a clinical trial to receive a promising new therapy before it is generally available to the public. For patients with advanced disease, clinical trials may offer some of the most aggressive options for treatment when conventional therapies have failed. Others may want to help scientific advancement. Another benefit is that medical treatments in a clinical trial are routinely made available at no charge to the patient, family or insurance company.

What are possible drawbacks to participating in a clinical trial?

Clinical trials are not guaranteed to provide better or even comparable care. There may be unforeseen side effects that may be worse than those experienced with standard care. Even if a new treatment or drug is found to be effective in some patients, it may not benefit you because standard treatments proven effective for many people do not help everyone. Not all health insurance and managed care providers cover all patient care costs in a trial. What is covered will vary by plan and by study. To find out in advance what costs are likely to be paid in your case, talk to a doctor, nurse or social worker from the study.

Are there different types of clinical trials?
The majority of clinical trials are "therapeutic" — offering new treatments to those who have advanced diseases such as multiple sclerosis or heart disease. Some are “adjuvant,” such as those testing hormones to prevent recurrence of breast cancer. “Prevention” trials test agents that may help reduce the likelihood of developing diseases for those at high risk. In "translational" trials researchers examine a patient's own genes to determine what genetic makeup responds best to particular treatments.

What are the phases of clinical trials?

Clinical trials are divided into four "phases." Which trial is offered to an individual patient depends upon the stage of their disease, prior treatment and the specific eligibility criteria of the trial.

• Phase I trials are the first step in testing a new treatment in humans after some effectiveness has been shown in animal models. In these studies, researchers look for the best way to give a new treatment (e.g., by mouth, IV drip or injection and how many times a day). They also try to find out if and how the treatment can be given safely (e.g., optimal dose); and they watch for any harmful side effects. Phase I trials are usually offered only to patients with advanced disease who would not be helped by other known treatments. Some patients are helped by these treatments, although in this early stage physicians do not know if the treatment will be effective.

• Phase II trials take the treatment one step further, assessing the activity of a particular therapy in a disease, often building upon leads from the Phase I trial.

• Phase III trials compare the results of people taking the new treatment with results of people taking standard treatment (e.g., which group has better survival rates or fewer side effects). In most cases, studies move into Phase III testing only after a treatment shows promise in Phases I and II. Phase III trials may include hundreds of people around the country.

• Phase IV trials refine the treatment to become part of standard care.

How are clinical trials monitored?

As with any new medication or surgical procedure, treatments are first studied thoroughly in the laboratory. If basic research establishes that a new method or chemical seems likely to succeed safely and effectively in patients, it can be submitted for approval for a clinical trial. Not only must the submission meet regulatory guidelines established by organizations such as the National Cancer Institute (NCI) and the Food and Drug Administration (FDA), but it must also be reviewed first for scientific merit by the Hospital Clinical Investigations Committees. It is then submitted to the USC Health Sciences Institutional Review Board (IRB), a panel of 36 scientists, researchers and others in the community who additionally approve the proposal for safety and ethical issues. Aside from reviewing the science, safety and efficacy, the IRB gives serious consideration to the proposed trial's "informed consent" — the document that physicians will give to patients explaining the trial and its potential risks.

Once approved, clinical trials are monitored through the hospitals’ Clinical Investigations Support Offices, where research nurses follow patients closely and data managers track their progress.

What questions should I ask if I am considering entering a trial?

If you are thinking about taking part in a clinical trial, here are some important questions to ask:

• What is the specific purpose of the study?
• What does the study involve? What kinds of tests and treatments? (Find out what is done and how it is done.)
• What is likely to happen in my case with, or without, this new research treatment?
• What are my other options and their advantages and disadvantages? (Are there standard treatments for my case and how does the study compare with them?)
• How could the study affect my daily life?
• What side effects may I expect from the study? (There can also be side effects from standard treatments and from the disease itself.)
• How long will the study last? (Will it require an extra time commitment on my part?)
• Will I have to be hospitalized? If so, how often and for how long?
• Will I have any costs? Will I be charged for any of the treatments?
• If I experience side effects as a result of the research, what treatment would I receive?
• What type of long-term follow up care is part of the study?

Are clinical trials safe?

Patients considering participation in a clinical trial should discuss it with their physician and/or medical caregivers. They should also seek to understand the credentials and experience of the individuals and the facility involved in conducting the study. The patient's rights and safety are protected in two important ways. First, any physician awarded a research grant by a private company or the National Institutes of Health must obtain approval from an Institutional Review Board before conducting the study. The review board, usually composed of physicians and lay people, is charged with examining the study's protocol to ensure that a patient's rights are protected and that the study does not present undue or unnecessary risk to the patient. Second, anyone participating in a clinical trial in the United States is required to sign an "informed consent" form. This form details the nature of the study, the risks involved, and any potential side effects a patient may experience during the study.

What is an Institutional Review Board?

An Institutional Review Board (often abbreviated as "IRB") is a committee or panel comprised of medical and non-medical members who study and properly consider all clinical protocols involving human subjects. The IRB assesses:

1. The risks to the subjects
2. The anticipated benefits to the subjects and others
3. The importance of the knowledge that may reasonably be expected to result
4. The informed consent process to be employed

What is it like to be a patient in a trial?

Doctors, nurses, social workers and other health professionals from many different specialties may help care for you. They are working together for your good. There is consideration for your privacy and well-being.

If you join a research study, you will be watched closely, and data on your case will be carefully recorded. You may receive more examinations and tests than are usually given. Although they can be inconvenient, these tests can assure extra observation along the way. During the course of a study, if it is clear that a treatment is not in your best interest, you will be removed from the study and you can discuss other options with your physician.

Can you stop your participation in a trial at any time?
Yes. Just as you can refuse to join a study, you may leave a study at any time. Your rights as an individual do not change because you are a patient in a clinical trial. You may choose to take part or not, and you can always change your mind later, even after you enter a trial. You may also refuse to take part in any aspect of the research. If you have questions at any time about any part of the study, be sure to ask your doctors. If you are not satisfied with the answers, you may consider leaving the study. If you decide to leave, it will not be held against you. You can freely discuss other possible treatments and care with your doctors and nurses.