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Bright Futures

Articles and Updates from Phoenix Children's

September 30, 2020
Top Questions to Ask When Considering a Childhood Cancer Clinical Trial

“Your child has cancer.” It’s a nightmare no parent wants to contemplate, yet more than 15,000 kids in the U.S. are diagnosed with cancer every year. And while there is good news – survival rates have climbed significantly in the last 50 years – pediatric cancer remains a leading cause of death for youth under 14, second only to accidents.

As Childhood Cancer Awareness Month comes to a close, Phoenix Children’s is shining a spotlight on ongoing research efforts and clinical trials that are growing our knowledge and providing new hope for children fighting cancer.

For many families facing pediatric, clinical trials offer much-needed hope, but for others, they invoke fear. If you’re considering a clinical trial for your child, this Q&A provides answers to six important questions:

What’s the purpose of a clinical trial?

A clinical trial is a medical research study that is guided by a specific plan of action. Patients volunteer to participate to test the efficacy and safety of new drugs and medical devices. The goal is to define the best course of action for a new drug/device and ensure safety for patients with the disease. The courses of chemotherapy (such as Daunorubicin and Vincristine) and radiation that are commonly used to treat cancer today were once researched and revised during clinical trials.

Clinical trials serve an important purpose in the medical field. They provide an opportunity to test many types of therapies, such as new drugs or medical devices, and allow doctors to answer important questions about medical care as they develop new and improved ways to treat patients. They also enable patients to gain access to new research treatments before they are widely available, while helping future generations by contributing to medical research and advancing patient care.

Are clinical trials safe for my child?

Though there are risks associated with clinical trials, patient health and safety is closely monitored. Before beginning any clinical trial, the risks and side effects of each research treatment are clearly communicated. Patient families are encouraged to review this information and ask questions to ensure they understand.

Before the trial can even begin, it must be approved by an Institutional Review Board (IRB). The IRB is an independent committee of physicians, statisticians, community advocates and others that ensure a clinical trial is ethical and the rights of study participants are protected. They are responsible for monitoring the trial to make sure the risks are as low as possible and are worth any potential benefits.

What is the cost to participate in a clinical trial?

There are generally no added costs associated with participation in a clinical trial. Standard of care treatments (including exams, laboratory work, X-ray, etc.) are covered by the patient’s insurance. Other therapies or studies related to the trial are covered by the trial’s sponsor, such as a pharmaceutical company. Families are encouraged to confirm what’s covered and what’s not before enrolling in a clinical trial.

Is my child eligible to participate?

Although clinical trial participants are volunteers, there are strict criteria dictating who can(not) take part in the study. Criteria may include everything from child’s age to the stage of a disease and previous treatment history. These guideposts are used to ensure safety of patients and efficacy of the trial. Before deciding whether or not to join, patients should learn as much as possible about the clinical trial and feel comfortable asking the members of the healthcare team questions about it. Questions could include: What kinds of tests and experimental therapy(ies) are involved? How might this clinical trial affect my daily life? What type of long-term follow-up care is required for this study?

What’s a “Phase I” clinical trial? What do the phases mean?

Clinical trials happen in four phases, which allow researchers to answer specific questions during each phase. For most trials, participants are only involved in one phase.

  • Phase I trials are the first step in testing a new therapy in children. They typically focus on how a new treatment should be administered. These trials usually enroll a very small number of participants.
  • Phase II trials focus on evaluating how well the new drug works and continue to test the safety of the drug.
  • Phase III trials compare the results of people receiving the new treatment with those of people undergoing the standard treatment. This phase enrolls large numbers of participants and may even take place at doctors' offices, clinics and research institutions nationwide.
  • Phase IV trials evaluate the treatment after it has been approved and is being marketed. This final phase examines the side effects, risks and benefits of a treatment over a long period of time and may include thousands of participants.

Why would I consider enrolling my child in a clinical trial?

Clinical trials offer children and families access to the latest, most cutting-edge treatments available while also helping researchers refine precise drug dosages and efficacy. Phoenix Children’s embraces robust translational pediatric research, including cellular therapy and other emerging treatments. Our Center for Cancer & Blood Disorders is the largest center of its kind in Arizona. At any given time, we have more than 150 active studies – with hundreds of patients taking part – that are guiding the future of medicine.

If you have more questions about clinical trials, check out our FAQ on clinical trials or consult with your child’s physician.

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