Clinical Trials: Protecting and Treating Children with Key Advancements in Medical Science

Vaccinations, medications and treatments go through a series of tests and approvals before they are available to be used in practice. Le Bonheur Children’s Hospital is currently participating in several clinical trials for kids. Dr. Dennis Black, Vice President of Research at Le Bonheur Children’s Hospital and Scientific Director of the Children’s Foundation Research Institute, describes the process and purpose of clinical trials.

“A clinical trial is a formal way to test a new drug or treatment in humans,” he explains. “They establish the safety of that drug or treatment in humans, and they establish the efficacy of the drug or treatment. This is how well that drug or treatment functions to treat or improve a disease state. Because of those reasons, they’re extremely important for getting new treatments and new drugs on the market for several diseases, including those in children.”

Children: A Vulnerable Population

Data from clinical trials in adults can’t be transferred directly to children. Young bodies metabolize drugs differently and may have different diseases than adults. While a drug may be considered “effective” in adults, children have specific safety concerns to be considered. “We try to keep the adverse events associated with the drug to a minimum, and then we try to maximize the efficacy or effectiveness of the drug,” notes Dr. Black.

Since efficacy and safety of treatments may vary with age, several categories of children need to participate in clinical trials. This includes a wide set of developmental stages, encompassing toddlers, school-age children, adolescents and young adults.

Each clinical trial has several regulatory bodies providing oversight to protect children from harm while generating the most benefit with new treatments. The Institutional Review Board, an independent data safety monitoring board (DSMB) and the U.S. Food & Drug Administration (FDA) all play a part in monitoring a clinical trial, reviewing the data for safety and efficacy.

Safety Is a Top Priority in Children’s Clinical Trials

Clinical trials are very carefully monitored. Safeguards are built in to halt studies if potential harm could be caused by the drug or treatment. “The average time from that very first step in development to a drug being approved and marketed can run 10 to 12 years,” Dr. Black mentions. “Anywhere along the way, if something goes wrong, then the drug, the study, the process can be terminated, and the drug won’t be approved.”

Every drug or treatment goes through a discovery and development stage where the drug is investigated on a molecular level. Preclinical research involves testing the efficacy of the drug on cultured cells. The drug is then registered with the FDA. Safety concerns and potential adverse effects are top of mind at all phases.

Once the FDA has reviewed all of the data and believes it’s safe to proceed, the treatment is tested in healthy adults. Even treatment designed for children is tested on adults first. It may then be tested on adults with the disease intended for treatment—checking how the drug is metabolized, watching for benefits and side effects. Based on the results, it may then be tested on children.

Every clinical trial has a screening process for participants. Factors that could put the patient at great risk will exclude them from the study. Speak with your pediatrician if you would like to know more about a clinical trial for your child.

“We want to make sure that the patients are followed closely, that there’s an open chain of coordination between the parents and the study coordinators who are conducting this study. And that if there’s ever a concern or question on the part of the parent, they need to feel they can call or contact the study personnel anytime to get answers about a concern or other question,” assures Dr. Black. “Really a key part of the whole process is open communication. And then, just making sure all the components of the protocol are properly carried out all the way from the beginning to the conclusion of the study.”

To learn more about Le Bonheur’s active clinical trials, visit https://www.lebonheur.org/research/clinical-trials/

**To listen to an in-depth conversation on this topic with Dr. Dennis Black, Vice President of Research at Le Bonheur Children’s Hospital and Scientific Director of the Children’s Foundation Research Institute, follow this link: https://radiomd.com/lebonheur/item/45692