Restarting Triple Therapy with Robust Monitoring for Adverse Events
CF RETRIAL
What is the goal of the study?
RETRIAL is a prospective, longitudinal, observational multi-site study designed to observe what happens when people with cystic fibrosis (PWCF) ages 6 and up start taking a newly approved CFTR modulator, vanzacaftor-tezacaftor-deuterated invacaftor (VTD), and have a history of: (1) New or worsening mental health (MH) symptoms while on a different CFTR modulator, elexacaftor ? tezacaftor-ivacaftor (ETI), requiring discontinuation or change from standard dosing OR (2) Liver-related intolerance to ETI requiring discontinuation or change from standard dosing. Participants will be enrolled for up to 13 months. Approximately 200 unique PWCF are expected to enroll in the study: ? RETRIAL-MH: 150 PWCF age 6 years and older who experienced new or worsening mental health (MH) symptoms while on ETI, requiring discontinuation or change from standard dosing will enroll in this approximately year-long study arm. Surveys will be completed at designated time points. ? RETRIAL-Liver: 50 PWCF age 6 years and older who experienced liver-related intolerance to ETI requiring discontinuation or change from standard dosing AND 50 PWCF enrolled in RETRIAL-MH will enroll in this approximately year-long study arm. Baseline labs will be collected prior to initiation of VTD with periodic surveys over the enrollment period. ? RETRIAL-Neuro: 60 PWCF enrolled in RETRIAL-MH, age 7 years and older, and who experienced new or worsening neurocognitive symptoms while on ETI, will complete two neuropsychological evaluations: before starting VTD and about 28 days after. ? RETRIAL-Biobank: All participants from RETRIAL-MH and RETRIAL-Liver will be invited to opt-in to provide blood, stool, and/or nasal swab samples before starting VTD and about 28 days after, to be biobanked for future analyses. No changes will be made to participants? existing care as part of their participation in the RETRIAL study. There is no clinical intervention, and no investigational products will be initiated under the study protocol.
Who can participate in the study?
Please contact the study team listed below to learn more.