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  • Understanding Study Results

Understanding Study Results | My Cayston Community™


Ongoing cystic fibrosis (CF) research efforts are directed at identifying a path to new treatments. Each of these studies helps us to learn more about CF and provides valuable information to the future of CF. Reading the study results can be confusing if you’re not familiar with common terms. As always, partnering with your care team and asking questions about things you may not understand is the best approach to take.

As always, partnering with your care team and asking questions about things you may not understand is the best approach to take.

In this article, I define some common terms to help you make sense of study data you come across:

Eligibility – Think of this as who could participate in the study. This can include things like a certain age range, forced expiratory volume (FEV1) range, or treatments being taken by participants.

Study Design – This describes how the study was conducted. Some studies are designed to determine if a difference can be shown between an active drug group and a control group, such as a placebo. A placebo is a solution with no medication and is given in place of an active drug to some of the study participants. This type of study is used most often to find out more information on the safety of a new medication and to help prove that it works as expected. You can read more about the study details for CAYSTON here.

You might also wonder what it means if a study is “blinded.” It doesn’t have anything to do with the study subject’s vision; it just means they are not told if they have been given active treatment or a placebo. A “double-blind” study is where neither the researcher nor the participant knows what a patient has been given. The opposite of this type of study is called an “open-label study,” where both the participant and the researcher know what treatment they are getting.

Methods/Procedures – This is what information was collected and what each participant was required to do while participating in the study. For example, with a new drug, this would include a description of any baseline testing (before starting the new medication) and then the follow-up testing and how often that testing was done.

Endpoints – This can be divided into two groups: primary and secondary. This is basically what the study is looking to learn. The primary endpoint is the main goal, and the secondary endpoint(s) is any additional information learned from a given study. In studies comparing two treatments, often the endpoint is whether one treatment is better than (superior to) or not worse than (non-inferior to) another treatment option. Additionally, endpoints may include looking at a measurement of something. In CF, we will often monitor respiratory symptoms and measure FEV1 to know if a treatment is working.

Statistical Analysis – I’ll admit, this can be a confusing area of a paper to read. Sometimes it reads as a lot of letters and numbers and seems to be in an unfamiliar language! This is an important part of the study that tells us whether the results are meaningful to patients. Often, these help to interpret the results that will either lead to approval of a new treatment or lead to termination of studying a given treatment.

With any research that is done, safety monitoring is very important. For this reason, study participants are asked to report any change in their baseline health to the research team. This is called adverse event reporting. The research team then consults with the clinical provider or principal investigator for the study to determine if the event is related to the treatment being studied. Adverse events and how often they occurred are reported as part of the study results and are helpful in determining whether a treatment option may be right for you.

Finally, if you are interested in participating in clinical research, talk to your CF care team or look on the Cystic Fibrosis Foundation website for the Clinical Trials Finder. If you’d like to learn more about the study details for CAYSTON, click here.