The CHEC-SC Cohort Study: CHaractErizing CFTR Modulated Changes in Sweat Chloride and their Association with Clinical Outcomes.
Study ID: STU 012018-076
Summary
This is a multicenter, cross-sectional, cohort study which will collect contemporary SC values from approximately 1000 CF patients prescribed and currently receiving commercially approved CFTR modulator therapies. eligible subjects who have been prescribed and chronically taking a commercially approved CFTR modulator for at least 3 months will be enrolled for a single visit to collect sweat to be analyzed for SC at their local site laboratory. Limited clinical data obtained at this visit will be augmented by retrospective and prospective data obtained from the CFFPR. Study subjects who have been prescribed and switch to an alternative commercially approved CFTR modulator will be approached to re-enroll in the study after being on the alternative modulator for at least 3 months so that a new SC value can be obtained.
To comprehensively describe patterns of SC response across a representative cohort of CF patients prescribed and receiving CFTR modulator therapy, adjusting for treatment duration, and characterizing these response patterns according to different CFTR modulators and across different patient groups defined by genotype, age, weight, disease severity, and disease stage. The secondary objectives are to: Characterize the association between varying levels and duration of CFTR-modulated SC and subsequent clinical outcomes including rate of lung function decline and frequency of pulmonary exacerbations. among the cohort of subjects who have experienced a change in their CFTR modulator therapy, characterize the observed changes in SC response to these changes and associations with clinical outcomes. assess data quality and consistency, feasibility, and utility of CHeC-SC relative to key findings to determine scalability to a greater portion of the CF population.
