Children With CF and Clinical Trials: What a Parent Needs to Know

Medical treatments that have been tested in adults often act differently in children. Medications require different dosages and sometimes produce different side effects in children than adults. More studies are needed focusing on children's health to develop treatments, drugs, and devices specific to children.

Patient Safety Is a Priority

Many different people are monitoring your child's safety in a clinical trial, just as they monitor the safety of adults in clinical trials. Clinical trials involving children must follow all the safety precautions in adult clinical trials. In addition, researchers must first collect and review safety data in adults before testing adolescents and then, finally, children.

Informed Consent and Assent

Parents have to give legal consent for their child to participate in a research study. Children also can be asked to assent, or agree, to participate, although some studies do not require assent. This means that children are given basic facts about a research study and are asked to be part of the decision. Children as young as 7 years old can be asked to give assent.

If you are thinking of enrolling your child in a study, you may want to ask how your child feels about it. Some children will want to be part of the process, while others may not. Some may be uncertain or afraid, worried about pain or concerned about how it will affect school or friendships. Some will be interested in helping other kids.

“My kids are very vocal about their interest in participating. They don't want to see another child go through [the sickness that] they went through.”
- Elizabeth Bishop, mother of 10-year-old twins with CF

At any age, the important thing is that your child is comfortable and his or her questions are answered. When children are asked if they want to join a study, it shows that you respect them. Children understand a lot, and it's important that their voices are heard.

The research team may be able to help if you and your child disagree about participation. Some studies have advocates and ethics experts involved who can help with questions.  

Ask Questions

Your study team is prepared to answer your questions, so don't hesitate to ask the principal investigator or the research coordinator questions. There are no bad questions. Some questions you may want to ask include:

• What is the purpose of the clinical trial?
• Why do researchers think that this particular CF drug or treatment might work?
• Who is paying for and supporting the study?
• Who has reviewed and approved the study?
• Who will be in charge of my child's care?
• How long will the study last?
• What will my responsibilities be if my child participates?
• What kinds of therapies, procedures, or tests will my child have during the trial?
• Will any of the therapies, procedures, or tests hurt, and, if so, for how long?
• How do the tests in the study compare with those my child would have outside of the trial?
• Will my child be able to take his or her regular medications while in the clinical trial?
• What are the risks?
• What are the benefits?
• How do the possible risks, side effects, and benefits compare with my child's current treatment? 
• How could being in this study affect the daily life of my child or my family?
• Can I talk to other people who are participating in the study?
• Will I be compensated for my child's participation in the clinical trial and/or travel expenses?
• What is the time commitment for us?
• Will results of the clinical trial be given to me and, if so, when?
• Whom should I communicate with during the trial: the research team, my CF care team, or both?

Write down your questions before you talk with your doctor or members of the research team to make sure you remember to ask them all. Also, try to write down the answers or record them with your smartphone, so that you can review them whenever you want.

Costs and Compensation

As a parent, you may be concerned about the cost of participating in a clinical trial. The sponsor of the clinical trial typically covers costs so there are no fees to participate. 

In the informed consent document, a section on compensation will tell you how much money you will get to participate. The compensation is usually given to pay for your time and to help offset the costs of participating. 

Some studies offer additional reimbursement for expenses such as meals, mileage, and parking. If you anticipate other costs, let your research team know ahead of time to see if they can reimburse you for those expenses.

Here are some questions you may want to ask:

• Will I have to pay for travel (gas, bus, subway, cab) and parking?
• Are there funds to help me if I have to miss work without pay?
• What if there is an overnight stay?
• Will meals be provided?
• What will the study pay for, and what is my insurance expected to pay for?
• What if I don't have health insurance? What will I have to pay for?
• Will I need to pay for the study medication once the study is complete?

Accommodating Your Schedule

To encourage participation, care centers and researchers often can work together to coordinate schedules so that regular care center visits can take place at the same time as the research visit. Often appointments can be moved to the beginning or end of the day to accommodate your work day. 

What You Can Expect

You should expect that your child will undergo more testing than would be done during clinical care visits because researchers must document changes in your child's health. These tests could include blood tests and extra breathing tests. 

Your child's care center will continue to be responsible for your child's primary care. If your child experiences any problems, you should let your care team and the research coordinators know.

When a study ends, it may take a while for the researchers to analyze data and publish results. If your child was testing a medication, he or she may have to stop the study medication at the end of the trial unless there is an open-label extension to the trial, or the drug becomes approved by the U.S. Food and Drug Administration (FDA). 

An open-label extension study means the doctors and participants know which treatment is being provided. Participants are invited to enroll, so that additional safety information can be gathered about long-term use while the FDA reviews the treatment for approval.

We call people with CF who participate in clinical trials, and all those who support them, trailblazers. Help us blaze a trail to better treatments and a cure for CF.

Resources

In this webcast, Warren and Lyndsey Love discuss why they enrolled their son, what types of questions and concerns they had initially, and how care center staff helped them fit the trial into their schedules. Dr. David Orenstein, a pediatric pulmonologist and CF doctor, talks about the layers of safety built into the study.

Read a short story for school-age children about one girl's experience joining a clinical trial. 

Learn more about children and clinical trials on the Children and Clinical Studies website launched by the National Heart, Lung and Blood Institute, a part of the National Institutes of Health.