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Clinical Trials

Medically reviewed by: Amy W. Anzilotti, MD

If your child has a serious illness like cancer or a (ongoing) condition like asthma, your doctor might talk to you about enrolling your child in a clinical trial.

What Is a Clinical Trial?

A clinical trial is a study that researchers do to see whether a treatment (such as a new medicine, therapy, or surgery) works well and is safe. Clinical trials follow a specific plan of action, called a protocol. The protocol says what kind of trial it will be, what it will study, who should be in the study, what treatments will be used, and how results will be measured.

The type of study used for most clinical trials is called a randomized, controlled clinical trial

What Happens in a Randomized, Controlled Clinical Trial?

In a randomized, controlled clinical trial, researchers often are trying to find out if a treatment works. These trials test the effects of drugs, therapies, vaccines, vitamins, or procedures. They try to find the best treatment with the fewest side effects.

In this type of study, people are randomly separated into groups:

  • a group that will get the treatment being tested
  • a group that will get the usual treatment (often, this is what they’re already taking) 
  • a placebo group that will get a treatment that contains no medicine at all

In clinical trials on children, a placebo group is used only if not giving the medicine won’t put a child’s health at a risk.

These clinical trials are "blinded." This means that patients don't know if they’re getting the treatment or the placebo until the trial is over. That way, their response can't be influenced by whether they think they have taken the real drug or not. In a double-blind study, neither the patients nor the researchers know who got the drug and who got the placebo until the study is over.

When the study is over, the results from the different groups are compared to see if the treatment being tested works and is safe.

Who Can Be in Clinical Trials?

Who can be part of a clinical trial depends on the study. For example, someone with an illness who’s not getting better with their current treatment might be in the trial to see if the new treatment could work. Sometimes, people who have a very aggressive illness or one that came back after treatment go into a clinical trial.

No one is forced to be in a clinical trial — it is always a choice. And someone who starts a trial can leave it at any time, for any reason. All information collected during clinical trials is confidential. It can’t be shared with anyone else without your permission.

Why Do People Go Into Clinical Trials?

People go into clinical trials for different reasons, such as

  • They’re looking for a better outcome for their illness.
  • They want the chance to try the latest treatments.
  • They want to help doctors develop new treatments.

What Else Should I Know?

Being in a clinical trial might be a way to help your child. You can learn more about a clinical trial by talking to your doctor and health care team.

Some questions you might want to ask are:

  • Why is my child a good candidate for this trial?
  • What are the possible benefits to my child?
  • What are the risks/potential side effects (both short-term and long-term) of this trial?
  • What tests, medicines, or therapies will be given?
  • How does this treatment differ from what my child is currently getting?
  • Will my child need to be in the hospital or see doctors at different locations?
  • How long will the trial last?
  • Is there any cost involved?
  • If costs are involved, will my insurance be billed?
  • Who will provide information and support to my child and me during the trial?
  • How will we know if the treatment is working?
  • What happens if the treatment does not seem to be working?
  • Will we be told about the trial results?
  • What happens after the trial ends? Will my child continue on the treatment if it's working well?
  • When the trial is over, will my child have the option to continue the treatment?
  • Is the trial being paid for by a particular company?

You also can find information online at ClinicalTrials.gov and search for current and upcoming clinical studies around the world.

Deciding what care is best for your child can be hard. Talk with your doctor, your child, your family, and others in your support system. Having as much information as possible will help you make the right decision for your child.

Medically reviewed by: Amy W. Anzilotti, MD
Date reviewed: January 2021