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 I studies are not done in children until the maximum tolerated dose (highest dose that can be safely given) and the side effects of the drug have first been established in adults.

Phase II clinical trials: These studies are designed to see if the drug works. Patients are given the highest dose that doesnÂ’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 patients—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 your child is in a clinical trial, a team of experts will be taking care of and monitoring your child very carefully. The study is especially designed to pay close attention to your child.

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 your child in a clinical trial.

Deciding to enter a clinical trial: Enrollment in any clinical trial is completely up to you and your child. 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 will not prevent your child from getting other medical care your child may need.

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

• Is there a clinical trial that my child would be eligible for?
• 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 my child receives?
• What is likely to happen in my childÂ’s case with, or without, this new treatment?
• What are our other choices and their advantages and disadvantages?
• How could the study affect my childÂ’s daily life?
• What side effects can we expect from the study? Can the side effects be controlled?
• Will my child have to be hospitalized? If so, how often and for how long?
• Will the study cost anything? Will any of the treatment be free?
• If my child is harmed as a result of the research, what treatment would we 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. You can reach this service at 1-800-303-5691 or on our Web site at http://clinicaltrials.cancer.org. Based on the information you provide about your childÂ’s cancer type, stage, and previous treatments, this service can compile a list of clinical trials that match your childÂ’s 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.

You can also get a list of current clinical trials by calling the National Cancer Institute's Cancer Information Service toll free at 1-800-4-CANCER or by visiting the NCI clinical trials Web site at http://www.cancer.gov/clinicaltrials. Another site, the ChildrenÂ’s Oncology Group is the major clinical trial group for children in the United States. It can be found online at www.childrensoncologygroup.org.

Revised 04-25-05