Clinical trials for your baby


  • Clinical trials help researchers learn about the safety of treatments and how well they work to help improve your baby’s health.

  • Clinical trials often test off-label drugs that aren’t approved by the FDA for use with babies. Most drugs used for babies are off-label.

  • You can help researchers design the trial and tell them what you think is most important for them to learn from the trial.

  • Having your baby in a clinical trial doesn’t mean that a treatment will work or that your baby will get better.

  • Talk to your baby’s health care provider to learn about benefits and risks of being in a clinical trial.

What are clinical trials?

Clinical trials are research studies that help health care providers understand if treatments, including medicines and medical equipment, are safe and helpful with certain health conditions. Clinical trials may show if treatments help people with health conditions, don’t help them or even harm them. Clinical trials also help researchers find out if a new kind of treatment is safe or if it works to help prevent or treat a health condition.

There’s a difference between a clinical trial and the regular health care that your baby gets. The trial does not replace your baby’s regular health care. Even in a trial, your baby still sees his regular health care provider for medical checkups and care.

How can you find out about clinical trials that may help your baby?

If your baby has a health condition, his provider may suggest that your baby be in a clinical trial. Or you can look for clinical trials for your baby’s condition on your own at Clinical trials often study treatments for conditions that affect babies, such as: 

  • Premature birth. This is birth that happens early, before 37 weeks of pregnancy.
  • Low birthweight. This is when a baby is born weighing less than 5 pounds, 8 ounces. 
  • Problems with the intestines, like necrotizing enterocolitis (also called NEC). Intestines are long tubes that are part of the body’s digestive system that helps break down food.
  • Lung and breathing problems, like bronchopulmonary dysplasia (also called BPD)

If your baby has any of these conditions, talk to his provider about having your baby in a clinical trial.

Why are clinical trials important for babies?

Health conditions and their treatments affect babies differently than older children and adults. Research from adults and older children can’t be used to treat babies. Clinical trials help researchers learn about which treatments work best for babies and how they can make treatments better and safe. For example, the kind and amount of medicine your baby needs may depend on his age, size and development. Medicine may need to be in lower doses for babies, and it may need to be given in different ways, like as a liquid, through an IV (through a tube into a vein) or in a shot in a baby’s muscle. Babies also may need smaller medical equipment than older people or equipment that’s been modified (changed) to work better with or in a baby’s body. 

What are off-label drugs and how are they used in clinical trials?

Most medicines providers use to treat babies in the NICU are off-label, and clinical trials often test the use of off-label drugs. Off-label means the drug is approved by the Food and Drug Administration (also called FDA) to treat certain conditions, but not the condition that your baby’s being treated for. Examples of off-label use of a drug include:

  • Using it to treat a group of people that it’s not approved for. For example, a drug is approved for use in adults but not in babies. 
  • Using it in a different way. For example, a drug is approved for use as a pill, but a provider gives it as a liquid.
  • Using it in a different amount. For example, a drug is approved for use once a day, but a provider gives it every 4 hours. 

If your baby is being treated with an off-label drug, you can advocate (ask for) a clinical trial that tests the medicine. You also can help the researchers design the trial and tell them what you think are the most important things for them to learn from the trial. For example, many trials compare different kinds of treatment for certain health conditions, but they often don’t focus on results needed to get FDA approval for the medicine to be used with babies or children. And FDA approval doesn’t always include information about the safety and effects of the drug on babies and children. If your baby is in a clinical trial, you can ask if the trial will focus on these things to learn the most about treatments that work and to be able to get drugs approved for use with babies (including premature babies) and children.

For more information about being more involved your baby’s clinical trial, talk to your baby’s health care provider or visit KIDS/iCAN. KIDS stands for Kids and Families Impacting Disease through Science; iCAN is the International Children’s Advisory Network. These are groups of parents and families who want to improve medicine and research for children. It’s important for you to know about your baby’s trial, what researchers are trying to learn from it and how the trial affects FDA approval of medicine for children. 

How do clinical trials work?

If your child is younger than 18, you have to give permission for her to be in a clinical trial. Before your baby joins a trial, the researchers give you complete information about the trial, including the purpose of the trial, the risks to your baby and what they want to learn about your baby and his condition. 

If you agree to have your baby in the trial, you give informed consent. This means you sign a form that says that you’ve received all the information about the trial, including the kind of treatment your baby will get and the benefits and risks it may have for your baby’s health. It also means that you’ve had all your questions about the trial answered, including questions about cost. The cost of the trial (like for medicines and treatment) may be covered by your health insurance, the organization that’s sponsoring the trial or the research institution that’s running the trial. Ask your baby’s provider or a member of the research team if you have questions about the cost of the trial. 

It’s OK if you decide you don’t want your baby to be part of a clinical trial, and you can remove your baby from a clinical trial at any time. 

Clinical trials are monitored (watched closely) by these groups:

  1. The FDA reviews all clinical trials that use new medicine or other treatments. It reviews the details of the trial and decides if the trial can be approved. It also gives advice about how the researchers should do clinical trials. 
  2. An Institutional Review Board (also called IRB) is a group of people, including medical professionals, researchers and community members, who are part of an institution like a university or hospital. All clinical trials that involve human participants, that test drugs or medical equipment that’s regulated (controlled) by the FDA and that are funded by (get money from) from the federal government have to be reviewed and approved by an IRB. If your baby’s in a clinical trial, the IRB helps protect your baby by reviewing the purpose of the trial, the risk and benefits to your baby and how the trial may help improve your baby’s health. For a trial to be approved, risks must be minimal (small or few), and benefits must outweigh the risks. 
  3. The Office for Human Research Protections (also called OHRP). The OHRP protects people who participate in research (like a clinical trial) that’s being done or funded by the U.S. Department of Health and Human Services (also called HHS). The OHRP helps people understand how research is done to help them make decisions about participating in (or having their baby participate in) a clinical trial. It also works with researchers to make sure that clinical trials follow rules and laws that help protect participants. 
  4. A Data Safety Monitoring Board (also called DSMB). Some clinical trials are reviewed by a DSMB. This is a group of experts that monitors a clinical trial to make sure it’s safe for participants. It can recommend changes to or stop a clinical trial if it thinks participants are at risk. 

Each clinical trial has a protocol (set of rules) that explains what happens in the trial. The protocol includes things like:

  • The number of people in the trial. The number depends on what the trial is studying. A trial may have very few participants (20 or less) or several thousand. 
  • Who can be in the trial. Each trial has rules about who can participate. If the trial includes babies, it may need babies of a certain age, of a certain sex or with a certain health condition. 
  • The kinds of medicines and treatment participants get. The protocol describes what kind of medicine and equipment will be used in the trial and how often participants get the medicine. 
  • How long the trial lasts. Depending on what the trial is studying, it may last a few months or several years.

During the clinical trial, your baby still sees his regular health care provider. Your baby may see other health care providers who are part of the research team that’s working on the trial. The research team may include doctors, nurses, and other health care professionals. Any member of the team can help you get information about your baby’s condition and understand what’s happening in the trial. Your baby’s health is always the most important focus of the trial and guides any decisions you make about your baby’s care.

Your baby may have more medical tests and checkups than if she weren’t in the trial. If you decide not to have your baby be part of a trial, he gets his medical care from his regular health care providers. 

Clinical trials have phases that help researchers learn about your baby’s health condition and how treatments are working. In beginning phases, researchers use treatments on a small number of participants. Later phases include more participants to help researchers decide if treatments help, don’t help or harm participants compared to other types of treatments. In each phase, researchers check to see if the treatments are working, if they’re safe and if there are any side effects. A side effect is an effect of a treatment that is not the intended result. For example, your baby may have a fast heartbeat, diarrhea or get fatigued (very tired) as a side effect of medicine being used in the trial. Side effects can be different for each baby and depend on the kind of treatment he gets. 

What are the benefits and risks of having your baby in a clinical trial?

Researchers can learn a lot about treatments from clinical trials. Many clinical trials for babies compare treatments already being used to find out which is safer and helps your baby more. Having your baby in a clinical trial, though, doesn’t mean for sure that a treatment will work or that your baby will get better. Talk to your baby’s provider about having your baby in a clinical trial to help you decide if it’s right for your family.  

Benefits of having your baby in a clinical trial include: 

  • Your baby may get treatment that helps him get better.
  • Your baby may get new kinds of treatment that aren’t yet available to everyone.
  • Your baby’s health condition is closely followed by researchers and health care providers. 
  • Your baby may be helping researchers find treatments that help other babies with the same health condition.

Risks of having your baby in a clinical trial include:

  • Your baby may have side effects from the treatment. Sometimes side effects can be severe. 
  • The treatment being studied may harm your baby, or it may not work. In some cases, treatment in a trial may even make your baby’s condition worse.
  • The treatment may not be better than treatment that providers are already using.
  • Your baby may need more medical checkups than if he weren’t in a trial. 

More information

Last reviewed: April, 2017