Research Matters: Why Should My Child Participate in Clinical Research?

September 9th, 2016 by Amy Basken

This month we are doing a throwback to the posts regarding research. Today, CCHD welcomes back NIH medical officer and pediatric cardiologist Dr. Kristin Burns. Dr. Burns answers some frequently asked questions about clinical research and provides information about ongoing clinical research studies. This article was originally posted in 2016.

Have you ever noticed flyers posted in the waiting room of your child’s healthcare provider’s office advertising clinical research studies? Has your child’s healthcare provider ever invited you to learn more about a research study or asked if you want your child to participate in clinical research? If so, have you wondered, “What is clinical research and why should my child participate?”

What is clinical research?

Clinical research is a series of tests or observations that help scientists learn about how safe or effective medications, devices, and treatments are in humans or how diseases progress over time.
A clinical trial is a specific type of clinical research study that compares treatments against each other. Participants are often assigned randomly (like a coin flip) to one treatment or another and their outcomes are compared.
Clinical research is different than the medical care your child receives from their healthcare provider. Research tries to understand whether a treatment may help a group of people with a certain condition in the future. Medical care focuses on the individual needs of a single person at the present time.

Why is it important for children to be in clinical research studies?

Many medicines used in children have not been tested in children to see if they are safe or if they work well. Because children are not just small adults and are still growing and developing, their bodies may work differently than adults, their health conditions may be different from adults, and medicines that work for adults may not work well or may be unsafe for children.
Therefore, it is important to do research studies involving children to test treatments and learn about pediatric diseases.

How does it benefit my child to be in a clinical research study?

By being in a research study, it is possible that your child might get access to newer drugs or treatments. Whether your child is assigned to get the experimental treatment, an existing treatment or a placebo (a sugar pill), your child is likely to have closer monitoring during a study, and you may learn more about your child’s condition by being in a research study.
It is possible that your child’s condition may improve by taking an experimental treatment. But it is also possible that an experimental treatment might not work better than existing treatments.
Your child’s participation may help other children with the same condition in the future. It may lead to the development of new treatments that work better, or it may prevent children from receiving a treatment that was proven in a research study to be unsafe or to not work well.

Is it safe for my child to participate in clinical research?

In addition to the doctors and nurses who will be monitoring the children in the research study, independent review boards, ethics committees, and safety monitoring boards have reviewed and approved the design of each study and will be monitoring its progress for safety.

It is your choice whether you want your child to participate in clinical research. Whether or not you decide to participate, your child’s medical care will not be affected.

What clinical research studies are going on now for children with congenital heart disease?

The Pediatric Heart Network, funded by the National Heart, Lung, and Blood Institute at the National Institutes of Health (NIH), performs clinical research studies for individuals with congenital heart disease.
The Pediatric Heart Network is currently enrolling participants in the FUEL Trial (Fontan Exercise Longitudinal Assessment). Teens who have had a Fontan operation and who are 12 to 18 years of age will be randomly assigned to 6 months of treatment with either a medication called Udenafil or placebo pills (that don’t contain any medication). The study will test whether treatment with Udenafil improves the ability to exercise. Previous studies have shown that, in people who have had a Fontan operation, decreasing ability to exercise over time is associated with worsening heart failure and increasing hospitalizations. This study hopes to identify a possible preventative treatment that could improve Fontan function over time and delay the development of heart failure. More information about the FUEL Trial can be found here.
Other clinical research studies may also be going on in your area or for your child’s condition. Ask your healthcare provider about other research studies that are available to your child.

Where can I learn more about clinical research?

The Children and Clinical Studies website is a helpful place to learn more about participating in clinical research, and to hear from families who have decided to participate and those who chose not to participate.
The Children and Clinical Studies website also includes a fantasy video game adventure, The Paper Kingdom, that helps children learn more about clinical research.
The Pediatric Heart Network website includes information for parents and teens and describes some of its studies in congenital heart disease, including the FUEL trial.
ClinicalTrials.gov is a searchable database that includes information on thousands of clinical research studies around the world.

Burns Formal Photo Kristin M. Burns, M.D. is a medical officer in the Heart Development and Structural Diseases Branch in the Division of Cardiovascular Sciences at the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH) and a pediatric cardiologist at the Children’s National Medical Center. Dr. Burns received her B.A. in Biochemistry and German from Wellesley College and her M.D. from the University of Massachusetts Medical School.