The purpose of clinical trials: Studies of promising new or experimental treatments in patients are known as clinical trials. A clinical trial is only done when there is some reason to believe that the treatment being studied may be valuable to the patient. Treatments used in clinical trials are often found to have real benefits. Researchers conduct studies of new treatments to answer the following questions:

• Is the treatment helpful?
• How does this new type of treatment work?
• Does it work better than other treatments already available?
• What side effects does the treatment cause?
• Are the side effects greater or less than the standard treatment?
• Do the benefits outweigh the side effects?
• In which patients is the treatment most likely to be helpful?

Types of clinical trials: There are 3 phases of clinical trials in which a treatment is studied before it is eligible for approval by the FDA (Food and Drug Administration).

Phase I clinical trials: The purpose of a phase I study is to find the best way to give a new treatment and how much of it can be given safely. The cancer care team watches patients carefully for any harmful side effects. The treatment has been well tested in lab and animal studies, but the side effects in patients are not completely known. Doctors conducting the clinical trial start by giving very low doses of the drug to the first patients and increasing the dose for later groups of patients until side effects appear. Although doctors are hoping to help patients, the main purpose of a phase I study is to test the safety of the drug.

Phase II clinical trials: These studies are designed to see if the drug works. Patients are given the highest dose that doesn’t cause severe side effects (determined from the phase I study) and closely observed for an effect on the cancer. The cancer care team also looks for side effects.

Phase III clinical trials: Phase III studies involve large numbers of patient – often several hundred. One group (the control group) receives the standard (most accepted) treatment. The other group receives the new treatment. All patients in phase III studies are closely watched. The study will be stopped if the side effects of the new treatment are too severe or if one group has had much better results than the others.

If you are in a clinical trial, you will have a team of experts taking care of you and monitoring your progress very carefully. The study is especially designed to pay close attention to you.

However, there are some risks. No one involved in the study knows in advance whether the treatment will work or exactly what side effects will occur. That is what the study is designed to find out. While most side effects disappear in time, some can be permanent or even life threatening. Keep in mind, though, that even standard treatments have side effects. Depending on many factors, you may decide to enroll in a clinical trial.

Deciding to enter a clinical trial: Enrollment in any clinical trial is completely up to you. Your doctors and nurses will explain the study to you in detail and will give you a form to read and sign indicating your desire to take part. This process is known as giving your informed consent. Even after signing the form and after the clinical trial begins, you are free to leave the study at any time, for any reason. Taking part in the study does not prevent you from getting other medical care you may need.

To find out more about clinical trials, ask your cancer care team. Among the questions you should ask are:

• Is there a clinical trial for which I would be eligible?
• What is the purpose of the study?
• What kinds of tests and treatments does the study involve?
• What does this treatment do? Has it been used before?
• Will I know which treatment I receive?
• What is likely to happen in my case with, or without, this new treatment?
• What are my other choices and their advantages and disadvantages?
• How could the study affect my daily life?
• What side effects can I expect from the study? Can the side effects be controlled?
• Will I have to be hospitalized? If so, how often and for how long?
• Will the study cost me anything? Will any of the treatment be free?
• If I am harmed as a result of the research, what treatment would I be entitled to?
• What type of long-term follow-up care is part of the study?
• Has the treatment been used to treat other types of cancers?

The American Cancer Society offers a clinical trials matching service for patients, their family, and friends.  Based on the information you provide about your cancer type, stage, and previous treatments, this service can compile a list of clinical trials that match your medical needs. In finding a center most convenient for you, the service can also take into account where you live and whether you are willing to travel.