Clinical Trials in Children: What You Should Know
Jun 02, 2021 Amy Paturel, MS, MPH
If your child is suffering from a serious condition such as asthma, irritable bowel syndrome or even seasonal allergies, you may wonder if they're eligible to participate in a clinical trial. It's important to note that clinical trials for children are different than trials for adults.
"Your child isn't the same as an adult—they're not even the same as other children. Infants react differently to interventions than their school-aged counterparts, and adolescents respond differently than those who are in puberty," says Dr. Irina Dralyuk, a pediatric pulmonologist in Cedars-Sinai's Maxine Dunitz Children's Health Center.
But like clinical trials for adults, pediatric trials could offer children a chance to receive beneficial treatment—a new drug, therapy, device or procedure—before it becomes widely available.
"Your child isn't the same as an adult—they're not even the same as other children. Infants react differently to interventions than their school-aged counterparts, and adolescents respond differently than those who are in puberty."
Clinical trials explained
The goal of clinical trials is to advance scientific knowledge, but not all clinical trials are created equal. Some are noninvasive and require little involvement from study participants. Others test the effects of new therapies, vaccines and procedures and may call for multiple clinic visits, uncomfortable tests and treatments that produce side effects.
All studies follow an established protocol that outlines what the trial aims to uncover, who is an ideal candidate, which treatments (if any) are being tested and how the results will be assessed. In randomized and controlled clinical trials performed in adults, researchers separate participants into two groups: an experimental group that receives the treatment under investigation and a control group that receives standard treatment or a placebo (water pill).
A randomized, double-blind clinical trial, for example, randomly distributes study participants to receive either the placebo or the experimental treatment. In these trials, neither the patients nor the researchers know who received the treatment and who received the placebo until the study is complete. At the end of the trial, researchers analyze the results to determine whether the new treatment is effective.
Why clinical trials in kids are different
Children are a complicated group to study. Researchers can't lump all children into one bucket—testing a treatment on a 17-year-old is not the same as testing a treatment on an infant. That's just one reason for the seven-year lag time (on average) between an adult approval and a pediatric approval for the same agent—that means adults have access to effective treatments years before they're available to children.
"Principal investigators for pediatric trials provide focused care and attention to kids in these trials simply because children's bodies and minds are still developing," says Dr. David Ziring, associate director of the Pediatric Inflammatory Bowel Disease Program at Cedars-Sinai.
So, while clinical trials for children are run in a similar manner as those for adults, there are a few key caveats:
- Informed consent: Depending on the age of the child, both the participating child and the child's parent need to consent to participate in the study. Older children and teenagers complete a separate informed consent. "We need to know they're willing to participate in the trial," Dr. Ziring says.
- Children's trials come last: Studies that are randomized and controlled set out to identify effective treatments for a particular condition. These trials occur only after preliminary research shows the agent is safe and effective in the lab, then in animals and finally in adults. "Kids are always the last to get tested with a new treatment," says Dr. Dralyuk. "By the time researchers begin trials for children, we know the intervention is safe and effective in adults."
- Placebo use: Clinical trials for children generally don't include placebos. "If there's evidence that a treatment works well in adults, it's not ethical to expose a child to a placebo and withhold a treatment that could improve their condition," says Dr. Ziring.
Investigators who conduct clinical trials in children also pay particular attention to child development, including bone growth, hormonal changes, social and emotional development and other considerations that are specific to children.
How to find trials
Finding clinical trials that are relevant for your child may require some investigating. The first step for many parents is the government's repository of health trials at clinicaltrials.gov.
"That's the most comprehensive source of information about clinical trials," says Dr. Ziring.
If that process feels too overwhelming, talk to your child's doctor and ask about relevant trials at Cedars-Sinai and other major centers. Often, your child's physician is better equipped to identify appropriate trials.
For children who have a specific condition, such as cystic fibrosis, leukemia or cerebral palsy, parents may be able to tap into national foundations and nonprofit organizations dedicated to the disease for information about relevant clinical trials.
"If you're interested in clinical trials testing a specific drug, you can also do an internet search of the medication's name," Dr. Dralyuk says. "In many cases, pharmaceutical companies provide information or links to clinical trials investigating their treatments."
What you should know before you enroll
Clinical trials provide an avenue for patients to help advance scientific knowledge. Your child's participation could result in scientists discovering a more effective treatment for diseases that affect children all over the globe—and it could treat their disease more effectively than standard protocols.
Still, there are risks involved with participation, too. In addition to potential side effects of experimental therapies, children may be subjected to the emotional and mental toll of frequent clinic visits, blood draws and assessments.
"That's one reason these trials include incentives," Dr. Ziring says. "It's not to entice parents to enroll their child in the study, but rather to compensate them for their time and effort, and for the discomfort and inconvenience involved with participation."
Before you decide to enroll your child in a clinical trial, make sure you get answers to three key questions:
- What are the potential risks and side effects of this protocol/therapy?
- How many visits will be required?
- Can you describe any invasive and/or potentially uncomfortable treatments so I can decide if the trial will be too taxing for my child?
Then, if you really want to know the specifics, you can delve deeper with this set of questions:
- What tests, medicines and therapies will be part of the trial?
- Is this treatment approved for adults with similar conditions?
- Has this intervention been tested on other kids?
- How many clinic visits, blood draws and other procedures will be required throughout the course of the study?
- Will we receive reimbursements for participating in the trial?
- How does this protocol differ from my child's current regimen?
- Will my child need to be hospitalized?
- Who will cover the costs if my child suffers from complications during the trial?
- How long will the trial last?
- Who will support my child and me during the trial?
- Will we have access to the results of the trial?
- Will the trial include follow-up care?
Learning as much as you can before enrolling your child in a trial offers the best odds of success.
"When you review the informed consent with the physician who is running the trial, it's important to ask not only about the risks and benefits of participation, but also the risks and benefits of not participating—and what alternatives are available to your child," Dr. Ziring says.
Remember, too, that every trial, invasive or not, is completely voluntary. You or your child can decide to withdraw from a clinical trial at any time, and for any reason. Your child's health—mental, emotional and physical—always comes first.