If your son or daughter is struggling with a serious illness like cancer or has a chronic condition like asthma, your doctor might talk to you about enrolling your child in a clinical trial.

About Clinical Trials

A clinical trial is a research study that follows a predefined protocol, or plan of action.

Some clinical trials observe people in certain situations (for example, how many overweight or obese kids become depressed) and then record the outcomes for study investigators to analyze. Others test the effects of potential new drugs, therapies, vaccines, vitamins, or procedures. These types of trials, called randomized, controlled clinical trials, try to find the best treatment with the fewest side effects. They begin only after preliminary research has been conducted and there is reason to believe that a potential new treatment is effective.

All clinical trials are voluntary and information collected during them is confidential. Participants can withdraw from a trial at any time, for any reason.

How They Work

All studies are led by a protocol, which establishes what the trial will study, who is a good candidate for the study, what treatments (if any) will be used, and how results will be measured.

In a randomized, controlled clinical trial, children are separated into groups. The experimental group(s) will receive the treatment that is to be tested, while the control group(s) will receive conventional treatment (usually what a child is already taking) or placebo (a fake treatment or sugar pill that contains no medicine at all).

In clinical trials on children, placebo is used only if the lack of treatment is short (perhaps a few days) and poses minimal risks, or if the therapy being tested is used to only treat uncomfortable symptoms (like watery eyes) and not a severe illness.

In these types of trials, patients are "blinded," which means they don't know who is getting the treatment and who is getting the placebo until the trial is over. That way, their perceived response to the drug or placebo can't be influenced by whether they think they have been taking the real drug or not. In a double-blind study, neither the patients nor the researchers know who has taken the drug or the placebo until the study is over.

Once the study is over, the outcomes of the experimental group and the control group are compared. Researchers analyze the data to determine if the potential new treatment is effective.

Who Can Participate?

The criteria that determine who is a candidate for a clinical trial differ from study to study. Often, those with an illness that isn't responding to current treatment and who may benefit from a potential new treatment may be candidates. Those with an aggressive illness, such as a recurring form of cancer or a disease for which there are few treatments, may be good candidates for a clinical trial.

Your doctor can help determine if your child is a candidate for a particular trial or may refer you to a physician associated with the trial. Once deemed a suitable candidate, your child must meet certain criteria (such as age, gender, and medical history) to participate.

Weighing Pros and Cons

Clinical trials are conducted in the hopes of finding new, more effective treatments and they're often very promising. However, a few factors should be considered when deciding if a clinical trial is right for your child.

The most compelling benefit of a clinical trial is the potential for a more effective treatment and better outcome for your child — and ultimately a cure for the illness. Often, trials enable patients to receive new drugs, therapies, or treatments before they become widely available. Kids involved in trials often receive treatment from the leading physicians in the field and their health is closely monitored. In addition, many families get a deep sense of satisfaction from the knowledge that their child is contributing to the development of better treatments for his or her illness.

Conversely, the new drugs, therapies, or treatments used in a trial might not be as effective as current treatment options or may not work at all. There is also a risk of potential side effects, all of which may or may not be known at the time of the trial.

Being a trial participant also might require your child to make more trips to the doctor's office or hospital (possibly overnight), see new doctors or specialists at different locations, or adhere to more involved or complex treatment requirements. Keep these factors in mind when deciding if a trial is good idea for your child.

Asking Questions, Voicing Concerns

Participating in a clinical trial may be a promising step for your child. But asking questions and communicating with the doctor and the health care team will help you feel confident in your decision.

Some questions you might want to ask are:

• Why is my child a good candidate for this trial?
• What are the benefits to my child?
• What are the risks/potential side effects (both short-term and long-term) of this protocol?
• What tests, medicines, or therapies will be administered?
• How does this treatment differ from what my child is currently undergoing?
• Will my child need to be hospitalized or see doctors at other locations?
• How long will the trial last?
• Is there any cost involved?
• Will my insurance be billed for expenses not covered by the sponsor?
• Who will provide information and support to my child and me during the trial?
• Can I speak to someone else not involved in the trial?
• How will we know if the treatment is working?
• Will we have access to trial outcomes?
• What happens after the trial has ended? Will my child continue on the investigational treatment if it's working well?
• Will follow-up care be offered after the trial is over?
• Is there a group that oversees the trial?

Asking questions and finding out as much as you can before making a decision is called the informed consent process. Getting the information you need is your legal right and responsibility. If you decide to enroll your child in the trial, you will be asked to sign a consent form.

It's Your Decision

Deciding the best course of care for your child can seem overwhelming. Before making a decision, take time to learn more about your child's illness, gather information, and ask questions. Discuss all of your options with the doctor and your family or others in your support system. Having accurate information will help you make the right decision for your child.

Reviewed by: Carlos D. Rosé, MD
Date reviewed: October 2012