Two researchers at the IU School of Medicine are co-leading a nationwide clinical trial in the fight against cystic fibrosis that will focus on an at-home observational study of patients. Dr. Cynthia Brown and Dr. Clement Ren are overseeing the first-of-its-kind study that uses a smartphone app to track CF outcomes.
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, a prescription medication approved by the U.S. Food and Drug Administration in 2019.
“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.”
IUSM says the medication targets genetic mutations in CF patients. The Cystic Fibrosis Foundation says approximately 90% of CF patients have the mutation.
The genetic disease causes persistent lung infections and limits the ability to breathe.
The foundation says data from clinical studies suggest the medication is an effective treatment, but they don’t know how it impacts day-to-day life for patients.
“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,” said Dr. Bruce Marshall, chief medical officer for the Cystic Fibrosis Foundation.
IUSM says Dr. Brown also led the school’s effort to test a COVID-19 vaccine.