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
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 early research has been done 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 get the usual 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 used to 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 decide if your child is a candidate for a particular trial
or may refer you to a health care professional associated with the trial. If
your child is a suitable candidate, he or she will also have to 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 things 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. Trials may let patients 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 watched. And,
many families get a deep sense of satisfaction knowing that their child is helping
to develop better treatments for his or her illness.
On the other hand, a new drug, therapy, or treatment 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 things 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
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
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.