FAQ: Participating in Pediatric Clinical Trials

If your child has been diagnosed with cancer or a blood disorder, it’s important to learn the full range of treatment options – including standard treatments and new options available only through clinical trials.

What are clinical trials?

Clinical trials are patient studies that test new treatments and therapies. The goal of pediatric cancer and blood disorder trials is to find better ways to treat cancer and blood disorders. A trial may test drugs, a new type of radiation therapy, new ways to do surgery, new combinations of treatments, or other new approaches to cancer and blood disorder care.

Is a clinical trial the right option for my child?

For many cancer and blood disorder patients, clinical trials provide new options. Often, clinical trials give children with rare or hard-to-treat conditions access to innovative treatments that may help where traditional options fail.

The first step is to find out if clinical trials are available for your child’s condition — and if so, what types of trials. Then talk with your child’s doctors about the full set of treatment options and what you can expect from a clinical trial. If your child participates on a clinical trial, he /she may stop the treatment on the trial at any time and receive standard therapy instead.

Confused about your options? Contact us.

As an international leader in pediatric cancer and blood disorder clinical research and care, we currently are involved in more than 100 pediatric cancer and blood disorder clinical trials — many of which aren’t available anywhere else.

To learn more, search our current clinical trials by condition — or contact us and we'll help you navigate your options. Email clinicaltrials@danafarberbostonchildrens.org and let us know the best way to get back in touch with you, where you live, your child’s diagnosis, and any treatment your child already has received.

Types of clinical trials

• Phase I trials look at how well a new drug is tolerated. These studies are used to check the highest doses that can be safely given. Generally, the study is only meant to find the best dosage and toxicity, not to show whether the treatment is effective.
• Phase II trials check to see if the treatment is working. The study usually enrolls a small number of patients who all have the same condition.
• Phase III trials compare two treatments, typically an experimental treatment and a standard treatment, to see if the new treatment works better. In Phase III trials, patients are often randomly assigned to a certain treatment group.

Who can participate in a clinical trial?

To qualify for a particular study, participants must meet a carefully defined set of criteria. These usually relate to age and gender, condition type and stage, and the types of treatments they already have received.

What information do participants receive about clinical trials?

Before agreeing to participate, patients learn about possible risks and benefits of the therapy being studied. As the trial progresses, participants are given new information that may affect their willingness to stay in the trial. Participants may withdraw from the trial at any time.

How are patients protected?

Before any clinical trial begins, it must be approved by its host institution's Institutional Review Board (IRB), which includes researchers and physicians. The IRB considers whether proposed studies are safe and well planned and whether they ultimately will advance patient care. It also reviews studies to ensure patients are adequately informed about the risks of participating in clinical research. In all studies, the health of each patient is closely monitored during the course of the trial.

Are clinical trials covered by insurance?

To find out whether or not the cost of a clinical trial is covered by your insurance company, you can talk with members of the study team, one of our financial counselors, or your insurance case manager.