How to take part in cystic fibrosis medical research
Scientists do medical research to better understand what happens in cystic fibrosis (CF) and ways to prevent and treat its complications. Research has given us many advances in CF care and will continue to do so in the future. Promising treatments have to be tested with patients who volunteer to take part in clinical trials. You and your child can help make a difference by taking part in research.
Your child may qualify to take part in one or more research studies. Deciding whether or not to take part in a study is a personal choice for each child and his or her family. There is no obligation to enroll or participate in a CF study.
How do I know if my child is eligible for a clinical trial?
Each study has entry criteria for who may qualify to take part. For example, a study may enroll only patients with a certain level of lung function. Sometimes there are criteria that exclude people who have problems that could put them at higher risk of side effects. Getting the right patients in a clinical trial is very important. The research team will tell you if your child meets the entry criteria. If your child does not qualify for one study, it does not mean he or she might not be eligible for others. Usually a person can take part in only one clinical trial at a time.
Researchers want to enroll people in studies who will follow directions and use the study treatment as prescribed. Patients and families who are good CF managers make good study subjects. On the other hand, patients who do not take their medications or do other treatments as prescribed are not likely to be chosen to take part in a clinical trial.
What is informed consent?
People who volunteer to take part in research give their permission by signing a consent form. Researchers must tell you about the study and what you and your child have to do to take part in it. For studies about drugs or other treatments, you will be told about the drug or treatment being tested and possible benefits and risks. This is called informed consent. Ask questions until you feel that you have enough information to decide whether or not you want you and your child to take part in the trial. Of course, you can refuse. You can also change your mind and stop taking part in a study if it is not right for you. Even if you and your child decide to drop out of a study later, there will be no change in the regular medical care that your child receives from your CF center health care team.
Many CF trials include children who are younger than 18 years of age. The child’s parent or guardian must sign the consent form and state that the child understands the study and agrees to take part. For some studies, children 6 years and older may sign an assent (agreement) form, but the parent or guardian must also give consent.
What benefits might there be from taking part in research?
People who take part in research have the first chance to possibly benefit from new treatments. Whether a treatment works or not, the information will be helpful to scientists and CF health care providers. The research study could lead to future benefits for all people with CF. Some studies let your child continue to use a treatment if it is working well prior to the treatment being available to the public. Taking part in research may also help you learn new things about CF or about your child.
The visits and tests that are done during a clinical trial are usually free. You and your insurance company do not have to pay for study tests or treatments. You and your child are often compensated for your time and effort to take part in a study. You will still pay for your child’s regular medical CF care.
Are there risks to taking part in a clinical trial?
Yes. Clinical trials test new treatments and there can be unknown or unexpected side effects. Some possible side effects of a new drug or treatment are known and may be minimized and some are not. Most drugs that get to the clinical trial process are expected to have only mild or temporary side effects. Some side effects can be serious, even life-threatening, or permanent. It is very important that you talk with the researchers about the possible risks and side effects of a treatment before you or your child join a study.
Questions to ask about taking part in a clinical trial
Here are some questions you will want to ask. Many of these will also be answered in a study consent form. Write down your questions so you can remember them.
What is the purpose of the study?
What do I have to do in the study?
What tests will be required?
What treatment is being tested? What are other possible treatments?
What side effects might my child expect?
What benefits might my child gain from being in the study?
How long will the study last?
How many visits to the CF center will my child need to make?
What will my child and I get for our time and effort if we take part in the study?
Who should I talk to if my child or I have other questions or problems?
Where can I learn more about research for CF?
The CF Foundation has a drug development pipeline to help test new treatments. You can see the progress of potential treatments going through the clinical trial phases by going to the CF Foundation’s website.
You can also search for a CF clinical trial on the CF Foundation's website.
Most clinical trials for CF are done at accredited CF centers. Ask your CF health care team what trials are being done at your center.