This is a intervention longitudinal study for children with CF and caregivers. Children aged 2-5 who are diagnosed with CF and are eligible for TRIKAFTA will be invited to participate in this study. The treating physicians will manage children with CF in accordance to their medical judgement and data will be collected throughout the study. Children with CF and caregivers may be enrolled up to 3 months before their child initiates TRIKAFTA and site personnel will record date for the study in that time (such as pulmonary exacerbations, medical history, and medication history.) Caregivers will also report PROS (patient reported outcomes) and CGRO (caregiver-reported outcomes).
The purpose of this study is to evaluate the safety and tolerability of single ascending doses (SAD) of VX-52 in patients with Cystic Fibrosis and the CFTR genotype who have not been responsive to CFTR modulator therapy. This is a first in human study.
This is a prospective, multicenter observational study to investigate clinical markers of nutritional status, physical frailty, and sarcopenia in adults with CF spanning a range of lung impairment and to identify barriers and risk factors to optimize nutrition and physical functioning in this patient population. Repeat measurements of body composition and physical function by various methods will be taken during the study and compared to clinical outcomes, as well as with each other. Smaller sub-set studies will be performed to assess bioimpedance analysis (BIA) and/or ultrasound as measures of body composition. Two cohorts matched by age, sex, race, and CFTR genotype severity will be enrolled: (1) those with FEV1 <60% and (2) those with FEV1 ≥60%.
This is a cross-sectional, multi-center study comparing stool-based testing (multi-target DNA-based test and quantitative FIT test) to standard of care colonoscopy for CRC screening in PwCF. The study includes an enrollment visit, two stool samples completed at home, a clinical screening colonoscopy, and three participant surveys which will be completed at enrollment and remotely.
The total duration of participant participation will be 3-12 months, depending on the timing of the completion of the stool sample and the participant's colonoscopy. The targeted participant timeline is 3 months from enrollment to the completion of a clinical screening colonoscopy. To allow for varying site-specific clinical colonoscopy scheduling delays and COVID delays in scheduling, the duration of time between stool sampling and screening colonoscopy may extend up to 12 months for inclusion of data in the study. However, greater than 3 months duration between stool-based testing and colonoscopy will be considered a protocol deviation.
The primary objective of this platform trial is to evaluate the efficacy and safety of differing treatments in CF pulmonary exacerbations during a planned 14 day course of IV antimicrobials. Primary efficacy will be evaluated as the difference in mean ppFEV1 (pulmonary function testing) changes from Visit 1 to Visit 2 (Day 28 ± 2 days) between intervention arms. The study will compare treatment of an intravenous aminoglycoside and β-lactams (AG) versus intravenous β-lactams only (non-AG) in people with CF diagnosed with a pulmonary exacerbation who will be treated for Pa.