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.
This study is a randomized, double-blind, placebo-controlled, multicenter study evaluating the safety and tolerability of twice daily inhaled bacteriophages (BX004-A) for up to 10 days in 32 outpatient adult Cystic Fibrosis subjects with chronic PsA pulmonary infections who are currently receiving inhaled antibiotics. This study consists of a screening period and a treatment period, divided into 2 parts. In Part 1, subjects will receive the study drug over 7 days, in part 2 over 10 days. Subjects in both parts will be included in a 6-month safety follow-up. Final safety assessments will include phone calls 28 days and 6 months after the last dose of study medication. Questionnaires will be administered through 28 days after the last dose of study medication. A Data Monitoring Committee (DMC) will monitor safety during both parts of the study.
This is a prospective, multicenter open-label study in adults with CF who are colonized with NTM. Subjects will receive two 5-day infusion cycles of IV gallium. The study will evaluate the safety and antimycobacterial effect of two 5-day infusions of IV gallium. Subjects will be on study for up to 148 days, with a screening period of up to 7 days and active study period up to 20 days. About 40 people with CF who are 18 years of age or older will take part in this study at about 10 hospitals and clinics around the country.
This is a prospective, multi-center, observational study in pregnant women with cystic fibrosis (CF) to characterize forced expiratory volume in 1 second (FEV1) changes based on exposure to highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators. The key factors contributing to the change in lung function during pregnancy and for 2 years post-delivery will be evaluated along with assessment of fetal and maternal outcomes.
The total duration of participation for each participant is expected to be about 3 years (up to 35 months). Women will be enrolled in the first trimester of pregnancy and assessed every 3 months during pregnancy and during the first year after delivery, then every 6 months for an additional year. Over the course of the study, CF clinical data, patient-reported outcomes, questionnaires, obstetrical outcomes, infant growth, child development outcomes, baseline CF-related therapies and co-morbidities will be collected to enable evaluation of changes from before pregnancy to during pregnancy and post-delivery.
MAYFLOWERS participants will be provided an opportunity to participate in an optional continuous glucose monitoring (CGM) sub-study to evaluate glucose control in pregnancy. Participants will undergo CGM sensor placement and data collection as part of the MAYFLOWERS study.
This is an observational study which will collect sweat chloride values from approximately 1000 cystic fibrosis subjects who are currently on cystic fibrosis transmembrane conductance regulator (CFTR) modulators. This study will require one visit to an MUSC clinic which will last approximately 2 hours. Compensation is provided.
This study has two parts and will assess your hearing and the potential effects of antibiotic-induced ototoxicity. Ototoxicity is a potential effect of drugs used to treat your lung infections, and its effect on your ears (oto=ears), particularly structures in your inner ear that enable hearing and balance. Part One of the study will assess your inner ear function that may be associated with hearing loss and / or loss of balance in cystic fibrosis patients taking tobramycin. Part Two is looking to determine if SPI-1005, an investigational drug, influences hearing loss, due to mediations (tobramycin).