Clinical Trials for Children With Cancer

This page is for parents and caregivers of a child with cancer who have been asked about a clinical trial. It covers why trials are so common in childhood cancer, consent, safety checks, costs, and questions to ask.

Clinical trials are a major part of childhood cancer care. NCI says most of the progress in finding curative therapies for childhood cancers has come through clinical trials. At the specialized centers where children are treated, trials are available for most types of childhood cancer, and many patients are offered the chance to take part.

Trials are how doctors learn what works. NCI says most of the progress in identifying curative therapies for childhood cancers has been achieved through clinical trials.

NCI says clinical trials for children are generally designed to compare a potentially better therapy with the therapy that is currently accepted as standard. Ask your team to explain, in plain language, what the trial would test and how it differs from standard treatment.

Before your child joins a trial, the research team explains it to you. NCI says you have the right to ask questions and to learn about all that is involved, including all your treatment options. Children cannot give formal informed consent. Instead they give assent, which means agreeing to take part after an age-appropriate explanation. Parents give permission, and the assent process stresses the child's voice in decisions that affect them.

Joining a trial is voluntary. Some families want every possible option explored. Others prefer the most established path. Both are reasonable. Ask your team to explain, in plain language, exactly what changes for your child if you say yes, and what stays the same if you say no.

NCI says you may withdraw from a trial at any time without penalty. Ask your team what leaving would involve for your child's care.

• What question is this specific trial trying to answer?
• What does the treatment plan look like on the trial, compared with the standard approach off the trial?
• What extra tests or visits does the trial require?
• What are the known side effects, and how will the team manage them?
• Can we change our mind later, and what would that involve?

NCI describes several layers of protection. An institutional review board (IRB) is a committee that evaluates a trial plan before enrollment begins to make sure it is ethical and scientifically sound. A data and safety monitoring board (DSMB) is an independent panel of experts that oversees the trial and can stop it early if serious safety concerns arise or if a treatment is clearly working.

NCI says many trial costs are covered by the study sponsor and by the patient's insurance if there is any. Routine care costs, such as doctor visits, hospital stays and standard treatments, are typically billed to health insurance. Research costs, such as study drugs and research-only tests, are often covered by the sponsor. Some trials also help with travel, lodging and meals. Ask your team about costs and financial help before you decide.

Your child's oncologist is the first source for trials your child may qualify for. NCI's Cancer Information Service (1-800-4-CANCER) can also help identify ongoing clinical trials for children with cancer.

• National Cancer Institute — Children with Cancer: A Guide for Parents.
• National Cancer Institute — Find a Clinical Trial.