Clinical studies are a major part of CF research. They take place at Cystic Fibrosis Foundation-accredited care centers all over the United States and enroll people with CF of all ages.

The two main types of studies are interventional and observational.

Interventional Studies

An interventional study tests (or tries out) an intervention — a potential drug, medical device, activity, or procedure -- in people. It is also commonly referred to as a clinical trial.

Examples of interventions in CF clinical trials include:

• A new potential drug, such as the compounds on the drug development pipeline
• A medical device, such as a vibration vest
• An activity, such as exercise or therapy

New potential drugs need to pass through three phases of interventional testing to show that they are safe and effective before receiving approval from the U.S. Food and Drug Administration (FDA).

Placebos in Interventional Studies

Many interventional studies are placebo-controlled. This means that some participants receive the treatment in question, while others receive a placebo. Researchers can compare the two groups to evaluate the treatment's effectiveness.

Some interventional studies are open-label. This means that all participants receive the treatment in question, and no one receives a placebo.

Observational Studies

Observational studies don't test potential treatments. Instead, participants stay on their normal treatment plan. Researchers observe participants and track health outcomes over time.

These studies help us understand CF by showing trends in survival and complications. They can also help us understand how effective a treatment is in a day-to-day or “real-world” setting. For example, after a new drug is approved, the FDA often requires that the pharmaceutical company continue to monitor the effectiveness and safety of the drug in what is known as a Phase 4 study.

Observational studies often take less time to participate. Participation could be as simple as a single blood draw or filling out a questionnaire. Unlike interventional studies, you can be in more than one observational study at the same time because the studies do not involve any changes to your regular treatment plan.

You can find both interventional and observational studies using the Clinical Trial Finder.