Why Participate in a Cystic Fibrosis Clinical Trial?

There are many different types of CF clinical trials, and one could be right for you. When you are deciding whether to participate in a clinical trial, it's important to know the potential risks and benefits.

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Summary

We can only develop new treatments for CF with the help of clinical trial volunteers. As a participant, you will be leading the way to new therapies and advancements in CF care.
Participating in a trial doesn't just help other people with CF – you may also gain access to new treatments not available to the public and get expert medical care at leading health care facilities.
There are some risks to participating in a trial, such as potential side effects from the treatment being studied. It's important to discuss participation with your care team. Your health and safety always come first.

Why Clinical Trials are Important

Incredible breakthroughs in the treatment of cystic fibrosis have been achieved through clinical trials. Drugs and treatments that have added tomorrows — and make living with CF today better for thousands — are now available thanks to this research and everyone who volunteered to be a part of it. In order to keep promising new research moving forward, we need people with CF to participate in clinical trials.

Watch this video to hear Dr. David Orenstein, a pediatric pulmonologist and CF doctor at Children's Hospital in Pittsburgh, talk about why clinical trials are so important.

The Benefits of Participating in a Clinical Trial

There are many possible benefits to participating in a trial, including:

Taking a more active role in your own health care
Gaining access to new treatments not available to the public
Getting expert medical care at leading health care facilities
Helping others by contributing to medical research
Receiving a treatment that works for you

"With these trials, we really don’t know the long-term effects, but I do know the long-term effects of cystic fibrosis. So the possible benefits kind of outweigh the possible negative risks associated with the study." — Brady Brock, an adult with CF who participated in a genetic therapy clinical trial

Brady Brock, an adult with cystic fibrosis who has two nonsense mutations, shares his experience participating in a clinical trial for a genetic therapy.

What About the Risks?

Although there are many possible benefits to participating in a clinical trial, there are also potential risks. Possible risks include:

Experiencing side effects or adverse reactions to medications or treatments
Receiving a treatment that doesn’t work for you

Your health and safety are always the priority. The research team will continuously monitor your health throughout the trial, whether you are receiving the drug being studied or a placebo.

If the research team notes any worsening of your health during the trial, they will notify your regular CF care team and the study sponsor to determine if it is related to the study drug. You should always contact the research team if you have any concerns during the clinical trial.

Remember: By signing an informed consent document, you are not signing a contract. You can leave a trial at any time. In addition, those who are monitoring the trial for safety — such as the principal investigator or the study medical monitor — can pull you from a study if they believe your health is declining.

If you have questions about participating in a CF clinical trial, reach out to our Clinical Trial Navigator for help. They can answer your research-related questions and help you find opportunities to participate.

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