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Nationwide cystic fibrosis study led by two IU School of Medicine researchers launched

5179-Brown, Cynthia

5179-Brown, Cynthia

The IU School of Medicine researcher who led the school’s effort to test a COVID-19 vaccine is now co-leading a nationwide clinical trial about cystic fibrosis. Cynthia Brown, MD, has several years of experience with clinical trials, including a decade seeing and treating patients with cystic fibrosis. This latest study is the first of its kind, using a smartphone app to track cystic fibrosis outcomes.

“I have been seeing patients with cystic fibrosis for ten years and know the toll it can take on families,” said Brown, who is an associate professor of clinical medicine at IU School of Medicine. “We are looking forward to finding more answers for people who have CF through this unique study.”

HERO-2: Home Reported Outcomes Study is an at-home observational study looking at the range of outcomes people experience while using Trikafta (elexacaftor/tezacaftor/ivacaftor). Trikafta was approved by the US Food and Drug Administration in 2019 as the first triple combination therapy to treat patients with the most common cystic fibrosis mutation. Trikafta works by targeting the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Experts estimate about 90 percent of cystic fibrosis patients have that mutation. According to the Cystic Fibrosis Foundation, cystic fibrosis is a progressive, genetic disease that causes persistent lung infections and limits the ability to breathe over time.

The HERO-2 study will allow people with cystic fibrosis to track their health and changes they make to their daily therapies while on Trikafta through self-reported updates on the Folia Health app on their smartphone over the course of a year. People who are 12 years or older living with cystic fibrosis who are taking Trikafta are eligible to participate.

“We want to learn how patients with cystic fibrosis are managing their daily care after starting Trikafta and how day-to-day symptoms change,” said Brown.

Clement Ren, MD, professor of clinical pediatrics at IU School of Medicine, is co-leading the study with Brown.

Abbie Volmar with her family

Abbie Vollmar (pictured with her family above) participated in a previous study about Trikafta through IU School of Medicine. Vollmar, who lives in Carmel, was diagnosed with cystic fibrosis when she was a month old. A couple years after her twin daughters were born, Vollmar started getting sick more often and feeling much worse. She was coughing all the time, exhausted from not being able to sleep and had to use IVs for medication at home. Back then, Brown, who is Vollmar’s doctor, suggested she participate in the Trikafta study.

“I spent my entire life doing different treatments, which were more like a Band-Aid, because they didn’t address cystic fibrosis itself, just the symptoms,” said Vollmar. “About a week into the Trikafta study, I noticed a difference.”

Vollmar said within a couple weeks, she wasn’t coughing much at all. By her next doctor’s appointment, her breathing had increased significantly.

“It was amazing how quickly that worked,” said Vollmar. “It’s not a cure, but it’s been an effective treatment for me.”

The HERO-2 study is being conducted by IU School of Medicine with funding from the Cystic Fibrosis Foundation.

“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.”

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.

Details about the study protocol are available on CFF Clinical Trials Finder and (NCT0479801).

The views expressed in this content represent the perspective and opinions of the author and may or may not represent the position of Indiana University School of Medicine.

Anna Carrera

Research Communications Manager

Anna Carrera is the research communications manager for Indiana University's Precision Health Initiative, IU School of Medicine and the Indiana Clinical and Translational Sciences Institute. She joined the team in June 2019 after working as a TV news rep...