Clinical trials are research studies that help doctors find better ways to prevent, detect or treat diseases. They can also improve daily life for children with long-term illnesses.
For children, the main goal of a clinical trial is to learn whether a treatment is
safe,
effective and
well-tolerated. These studies may involve new medicines, vaccines, medical devices, procedures or new ways of using treatments that already exist.
Before you decide whether a clinical trial might be right for your child, it's important to understand what the study involves, including possible risks and benefits.
Why kids need their own clinical trials
Children are not simply "small adults." They grow, develop and respond to treatments differently. They may also have health conditions that don't affect adults. This means children need research designed just for them.
Without pediatric clinical trials, doctors often must rely on adult research to guide children's care. Many medicines used for children today were originally tested only in adults. When a medication is used in a child even though it was studied only in adults, this is called
off-label use.
Laws now
require clinical trials for certain childhood conditions. As a result, more research studies for children are available than in the past.
How clinical trials for children work
Every clinical trial follows a detailed plan, called a protocol. It explains:
Who can participate.
How long the study will last.
What treatment or approach is being tested.
How information will be collected.
Protocols are in place to lower risks as much as possible and make sure the study can answer important health questions. (Note: Before any new treatment is tested in people, it is first studied in laboratories and in animals to check for safety.)
In many trials, the treatment is compared to something called a placebo. A placebo looks like the treatment but does not have the active ingredient.
Sometimes, a treatment with a different active ingredient is already available and the goal is to create a new one. In this case,
one group may be given the older treatment as a placebo. Often, the researchers and the participants do not know who is getting the treatment or the placebo until the study ends. This is called a double-blind study. It is another way to help keep the results fair and to avoid bias.
Possible benefits and risks of a pediatric clinical trial
Clinical trials exist because doctors don't always know the best treatment—or a new treatment may work better than what is available now.
Being part of a trial may offer benefits. Your child may get access to medical specialists who are experts in their field. They may be able to try new therapies and learn more about their health condition. With a trial, kids may also have closer monitoring or extra testing that are not part of regular care. Financial or other rewards may be offered. And your child will have the opportunity to be part of something that may help other children.
However, there may also be risks. such as side effects or the chance that the treatment may not work.
The clinical trial may take a lot of time. There might be complex medicine dosing or procedures. Or you may spend extra time on visits to the study site, more tests, treatments or stays at the hospital.
What does consent mean in clinical trials?
Before you and your child agree to join a clinical trial, it's important that you know all the facts.
Informed consent is part of the clinical trial process. Informed consent ensures that parents fully understand the trial. Informed consent gives permission but is not a contract. Parents and children can leave a clinical trial at any time. If a child leaves during the trial, doctors may recommend taking certain safety steps before stopping the treatment.
"Assent" from children in many clinical trials is also needed. Assent is when a child agrees to be in a clinical trial. The child must be old enough to understand the basic facts and ask questions about the trial.
In some cases, the risks and benefits are not fully known at the start of the study. Your care team is here to help you and your child make those important decisions.
Questions to ask about the clinical trial
The following questions can help you and your child learn as much as possible about the process before you join a clinical trial:
What is the purpose of the trial?
Is the trial approved by the institutional review board (IRB) and/or (if the trial involves American Indian or Alaska Native participants/communities) tribal IRB?
Is there an age range?
Who is sponsoring or funding the trial? (For example, medical institutions, pharmaceutical companies or federal agencies.)
Why do the researchers believe that the treatment in the study could be better than others used now?
What tests and treatments are involved? Are they risky or harmful? How often do they need to be performed?
How will the doctor know if the treatment is working? When will we be told about the results of the trial?
What are possible benefits?
What are the side effects or risks and how do they compare with the standard treatment?
If my child receives a placebo, could that be harmful to their health?
Will my child be unable to have other treatments if they join the clinical trial? What will happen to my child's illness or disease without treatment?
Will I have access to my child's test results during the trial?
How will my child's health information be kept private? Who has access to this information?
If the clinical trial involves collecting a body specimen (for example: blood, skin or other tissue, sputum (spit) sample, etc.), will it be destroyed after the trial ends? How do I know if my child's specimen will not be used for any other research after the trial?
Will I have to pay for any of the treatments or tests? What costs will my health insurance cover, and who can answer questions about insurance?
Who pays the medical bills if my child is injured in the trial?
Words used in pediatric clinical trials
assent—When a child agrees to be in a study.
masking—When participants and/or the study team do not know which treatment participants are getting until the trial is over.
informed consent—Parents agree to allow their child to join a study after they have read an informed consent form and other details about the study, spoken with the investigators, and asked questions.
interventional study—Another name for a clinical trial.
investigators—People doing the research study. Each clinical study is led by a principal investigator, often a medical doctor.
Institutional Review Board (IRB)—An independent committee that ensures that clinical trials are ethical and that the rights of the people in the study are protected. Each clinical trial in the United States must be approved and monitored by an IRB. Tribal communities may also have a tribal IRB. If the trial includes American Indian or Alaska Native participants or communities, a tribal IRB may also be involved.
placebo—A treatment that looks the same and is given in the same way as the active treatment that is being studied.
protocol—Detailed plan of the research study.
randomization—A way to choose what treatment each person in a study gets so that there is less chance of bias. It's like flipping a coin or rolling dice.
Remember
Joining a clinical trial is a big decision. It's OK to take time to talk it over with your child. Your child's care team is there to support and help sort through information. Ask questions and make sure you both understand the benefits and risks before volunteering.
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