When Laura Elliott adopted Julian at 5 years old, she knew he had hemophilia, but when she brought him home to Georgia, she learned more about the severity of his bleeding disorder: hemophilia A with inhibitors.
Even when Julian’s condition was well-controlled, he could spend about one week per month in a wheelchair. “I was open to any opportunity to change that,” including participating in clinical trials, Elliott says.
In the US, drug medications must go through clinical trials to determine their safety and effectiveness. Treatments are tested on volunteer participants and must be approved by the US Food and Drug Administration before going to the public.
Although any experimental treatment carries some risk, researchers work to limit it, especially for children. Medications usually are approved for adults before being tested on children, explains Robert Sidonio Jr., MD, director of hemostasis and thrombosis clinical operations at the Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta.
Also, children with hemophilia won’t receive a placebo—a substance with no therapeutic value used as a control—so parents shouldn’t worry about their child being without medication, Sidonio says. More commonly, a child would try a new treatment and receive lab monitoring for bleeding and quality of life before and after.
It is important to note that even when a product has been evaluated in adults, it doesn’t mean the results will be the same for children. It’s possible, for example, that young participants may develop a reaction to a modified factor product, ranging from mild itchiness to anaphylaxis, because their immune systems are less developed.
One benefit to children who participate in clinical trials is getting access to a drug that isn’t on the market yet and that may better control bleeding than their current treatment.
Julian, for example, continues to take the medication that was the focus of his trial. “His bleeds are well enough controlled that he can go anywhere,” Elliott says. “Things are possible that weren’t possible before.” Now age 13, Julian no longer needs a wheelchair.
Another benefit is the chance to contribute to the greater good.
“I learned how limited the research was in this field,” Elliott says. “The opportunity to add to that bank of knowledge was very appealing.”
She continues, “I am incredibly grateful for the people who participated in studies before us. Because of them, my kids have options that weren’t even dreamed of 20 years ago.”