Observational study supported by the CF Foundation will monitor real-world experience with Trikafta

Today, the Cystic Fibrosis Foundation announced the initiation of the HERO-2: Home Reported Outcomes Study, a first-of-its-kind, at-home observational study that aims to characterize the range of outcomes people experience while using Trikafta® (elexacaftor/tezacaftor/ivacaftor). The study will rely on observations that individuals living with cystic fibrosis make about their health in their daily lives to understand the impact of Trikafta when taken in the real world.

“Data from clinical studies suggest that Trikafta and other highly effective modulators have a transformative effect on pulmonary and nutritional outcomes of cystic fibrosis, yet we still have much to learn about how these therapies impact day-to-day life with CF,” said Dr. Bruce Marshall, chief medical officer and senior vice president of clinical affairs for the Cystic Fibrosis Foundation. “Understanding real-world experiences with Trikafta will inform the continued evolution of CF care and aid in the prioritization of future research and support programs to meet the needs of people on modulators. We are grateful to our collaborators at Indiana University School of Medicine and Folia Health for enabling this important research.”

For the first time in CF research, the HERO-2 study will empower individuals with CF to actively track their health and any changes they make to their daily therapies while on Trikafta through self-reported updates on the smartphone Folia Health app. Participants will use the app to enter weekly updates on their treatment use and symptoms and complete monthly check-ins over the course of 12 months. Utilizing remote data entry through an app provides a novel way to measure outcomes in CF and learn about how personalizing daily care works in the real world.

The study design was developed by an interdisciplinary group of patients and caregivers, clinicians, scientists, and information technologists over a period of over eight months, where they focused on ways to ensure the design would work for the widest group of patients and families.

People ages 12 or older living with cystic fibrosis, who are taking Trikafta are eligible to participate.

The HERO-2 study is being conducted by Indiana University School of Medicine with funding from the Cystic Fibrosis Foundation. The app powering the study was developed by digital health company Folia Health, and the data will be linked to the CF Foundation’s Patient Registry, a robust database that informs research and care of CF.

To learn more about virtual participation and see details about the study protocol visit to the CF Foundation’s Clinical Trials Finder and www.clinicaltrials.gov (NCT04798014).

About the Cystic Fibrosis Foundation

The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. To advance its mission in finding a cure for all people living with cystic fibrosis, the CF Foundation launched its $500 million Path to a Cure initiative. This ambitious research agenda aims to accelerate the next generation of transformative CF therapies and deliver a treatment for every person with CF. The CF Foundation is a donor-supported nonprofit organization. For more information, visit cff.org.

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