The Cystic Fibrosis Foundation announced the initiation of an at-home observational study that aims to characterize the range of outcomes people experience while using Trikafta (elexacaftor/tezacaftor/ivacaftor). The research, called the HERO-2: Home Reported Outcomes 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.  

“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 Bruce Marshall, MD, 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 the CF Foundation’s Clinical Trials Finder and (NCT04798014).