Smartphone App Study to Assess How Trikafta Affects Daily Life
A first-of-its-kind, observational study will allow patients with cystic fibrosis (CF) to use their smartphones to monitor the ways in which Trikafta (elexacaftor/tezacaftor/ivacaftor) affects their daily lives.
Sponsored by the Cystic Fibrosis Foundation, the now-enrolling HERO-2 study (NCT04798014) will collect patients’ self-reported feedback for one year to track changes in their daily medications and symptoms while taking Trikafta.
Participants will be also asked to report instances of changes to their treatment plan and pulmonary exacerbations (the acute worsening of symptoms), as well as to provide a monthly overview using patient-reported outcome questionnaires.
“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,” Bruce Marshall, MD, chief medical officer and senior vice president of clinical affairs at the CF Foundation, said in a press release.
Patients’ feedback will be submitted using the Folia Health app for smartphones, which allows health information to be securely sent without the need to visit a clinical site. Data in the Folia Health app will be linked to the foundation’s Patient Registry database.
The study, conducted by the Indiana University School of Medicine, is looking to enroll 860 CF patients, ages 12 and older, taking Trikafta and with access to an internet connection. It is scheduled to end in December 2023.
Patients in the U.S. with any type of mutation in the CFTR gene, the cause of CF, are invited to self-enroll. For more information, please contact Lisa Bendy, clinical research manager at Indiana University School of Medicine, by phone at 317-278-7152 or by email [email protected].
However, anyone enrolled in the randomized phases of the SIMPLIFY (NCT04378153) trial underway in the U.S. may not take part in the HERO-2 study. SIMPLIFY is evaluating the health of CF patients, ages 12 and older, on Trikafta who stop a nebulized treatment inhaled — hypertonic saline or Pulmozyme (dornase alfa) — compared with those who continue with both these forms of treatment.
Those interested in joining the trial can do so using the free Folia Health app, which can be accessed on its website using an iPhone, android phone, or a computer. After completing an initial survey, participants log in to their Folia Health account where they can provide information on their symptoms and medication use. While they are encouraged to report daily, the app allows for a “batch track” to provide reports covering the previous seven days.
On a monthly basis, they will be asked to complete a survey regarding symptoms and medication.
“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,” Marshall said.
The study was designed over eight months by a team that included CF patients, caregivers, clinicians, scientists, and information technologists, the CF Foundation reports. The aim was to ensure that the trial would be open to the broadest group of patients and families.
“We are grateful to our collaborators at Indiana University School of Medicine and Folia Health for enabling this important research,” Marshall.
Trikafta, marketed by Vertex Pharmaceuticals, is approved in the U.S. to treat CF in patients, ages 12 and older, who have at least one copy of the F508del mutation in the CFTR gene — the most common disease-causing mutation.
The U.S. Food and Drug Administration is expected to soon decide on an application seeking the expansion of Trikafta’s use to children as young as 6.