Childhood Cancer: About Clinical Trials

Approved by the Cancer.Net Editorial Board, 08/2023

ON THIS PAGE: You will learn more about clinical trials, which are the main way that new medical approaches are studied to see how well they work. Use the menu to see other pages.

What are clinical trials?

Doctors and scientists are always looking for better ways to care for children with cancer. To make scientific advances, doctors conduct research studies called clinical trials that involve volunteers. Every drug that is now approved by the U.S. Food and Drug Administration (FDA) was tested in a clinical trial.

Clinical trials are used for all types of childhood cancer. Many focus on new treatments to learn if they are safe, effective, and possibly better than the existing treatments or standard of care. These types of studies evaluate new drugs, different combinations of existing treatments, new approaches to radiation therapy or surgery, and new methods of treatment.

Children who participate in clinical trials can be some of the first to get a treatment before it is available to the public. However, there are some risks with clinical trials, including possible side effects and the chance that the new treatment may not work better than the standard of care. Families are encouraged to talk with their health care team about the pros and cons of joining a specific study.

Some clinical trials study new ways to relieve symptoms and side effects during treatment, while others study ways to manage the late effects that may happen a long time after treatment. Talk with your child’s doctor about clinical trials that might be available.

Deciding to join a clinical trial

Parents and children decide to participate in clinical trials for many reasons. For some, a clinical trial is the best treatment option available. Because standard treatments are not perfect, patients are often willing to face the added uncertainty of a clinical trial in the hope of a better result. Others volunteer for clinical trials because they know that these studies are a way to continue the progress in treating childhood cancer. Even if they do not benefit directly from the clinical trial, their participation may benefit future children with cancer.

Insurance coverage and the costs of clinical trials differ by location and research study. Some insurance programs reimburse patients' expenses from participating in a clinical trial. Other insurance programs do not reimburse expenses. It is important to talk with the research team and your insurance company first to learn if and how your child's treatment in a clinical trial will be covered. Learn more about health insurance coverage of clinical trials.

Sometimes people have concerns that their child may receive no treatment in a clinical trial by being given a placebo or a “sugar pill.” You and your child will always be told when a placebo is used in a study. Find out more about placebos in cancer clinical trials.

Patient safety and informed consent

To join a clinical trial, parents and children must participate in a process known as informed consent. Informed consent means that parents give permission for a younger child to participate in a clinical trial and that a teenager gives their assent to participate. Your doctor will explain informed consent and should:

  • Describe the purposes of the clinical trial and what researchers are trying to learn.

  • Describe all of the treatment options so that the person understands how the new treatment differs from the standard treatment.

  • List all of the known risks of the new treatment, which may or may not be different from the risks of standard treatment.

  • Explain what will be required of each person in order to participate in the clinical trial, including the number of doctor visits, tests, and the schedule of treatment.

Clinical trials also have certain rules called “eligibility criteria” that can help structure the research and keep patients safe. You and the research team will carefully review these criteria together. Your child will need to meet all of the eligibility criteria in order to participate in a clinical trial. Learn more about eligibility criteria in clinical trials.

People who participate in a clinical trial may stop participating at any time for any reason, personal or medical. The reasons may include that the new treatment is not working or there are serious side effects. Your child's doctors will continue to provide the best available treatment for your child regardless of whether or not you choose for your child to participate in a clinical trial or decide for your child to stop participating in the trial after initially enrolling.

Clinical trials are also closely monitored by experts who watch for any problems with each study. It is important that parents of children participating in a clinical trial talk with their child’s doctor and researchers about who will be providing their child's treatment and care during the clinical trial, after the clinical trial ends, and/or if they choose to leave the clinical trial before it ends.

Finding a clinical trial

Research through clinical trials is ongoing for all types of cancer. For specific topics being studied for childhood cancer, learn more in the Latest Research section.

Cancer.Net offers more information about cancer clinical trials in other areas of the website, including a complete section on clinical trials.

There are many resources and services to help you search for clinical trials for childhood cancer, including the following services. Please note that these links will take you to separate, independent websites:

  • This U.S. government database lists publicly and privately supported clinical trials.

  • World Health Organization (WHO) International Clinical Trials Registry Platform. The WHO coordinates health matters within the United Nations. This search portal gathers clinical trial information from many countries’ registries.

Read more about the basics of clinical trials matching services.

The next section in this guide is Latest Research. It explains areas of scientific research for childhood cancer. Use the menu to choose a different section to read in this guide.