A protocol to test the impact of discontinuing chronic therapies in people with cystic fibrosis on highly effective CFTR modulator therapy (SIMPLIFY)
CF SIMPLIFY Site
What is the goal of the study?
This prospective, randomized study will determine if the discontinuation of standard therapies used by patients with cystic fibrosis (CF) is non-inferior to continuation of these therapies, specifically hypertonic saline of Pulmozyme. Individuals with CF who are 12 years of age and older, may be eligible for enrollment after initiation of treatment with a CFTR modulator (Trikafta). Study participants will have a run-in period to capture adherence to the therapies being studied and will be randomized to continue of discontinue one therapy if eligibility based on adherence is demonstrated. Following randomization, study visits at Week 2 and Week 6 will collect safety data including adverse events, pulmonary exacerbations, and pulmonary function. A phone call for research assessments will occur at Week 4. Participants will complete patient reported outcome measures electronically weekly from Screening through Week 6. A subset of participants will also complete a Multiple Breath Washout (MBW) procedure to measure the Lung Clearance Index (LCI). Study participants who use both hypertonic saline and Pulmozyme are eligible to re-enroll in the other study arm after the Week 6 visit.
Who can participate in the study?
Please contact the study team listed below to learn more.