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Pharmacokinetic and Pharmacodynamic Effects of Dorzagliatin in Pancreatic Insufficient-Cystic Fibrosis: A Randomized Double-blind, Cross-over Trial
This study is designed to determine the pharmacokinetic and pharmacodynamic response of dorzagliatin 50 mg twice daily following 7-day administration in indivi…
Skeletal Health and Bone Marrow Composition in Adolescents With Cystic Fibrosis
The investigators will be evaluating bone marrow composition via magnetic resonance imaging in adolescents diagnosed with cystic fibrosis (CF) compared to heal…
Personalized Theratyping Trial
The purpose of this study is to explore the use of off-label CFTR modulators that may affect CFTR function in patients with CFTR mutations that are not current…
Advancing Telemedicine in Pulmonology: Acoustic-waveform Respiratory Evaluation (AWARE) Via Sensing and Machine Learning on Smartphones
The study will evaluate the feasibility of using smartphone speakers and microphones to evaluate the caliber of the airways, detect airway obstruction, aid in …
The Measurement and Analysis of Maximal Expiratory Flow Volume Loops at Low Lung Volumes in Children With Cystic Fibrosis and Normal Routine Lung Function.
Goal is to physiologically detect unsuspected small airways obstruction in children and adults with treated heterozygous and homozygous cystic fibrosis. Unsusp…
An Open-label, Single Center Study Assessing Tenapanor as a Non-CFTR-mediated Treatment of CF-related Constipation in People With CF.
Tenapanor is the newest FDA-approved drug for IBS with constipation (IBS-C). This study seeks to understand tenapanor as a treatment for cystic fibrosis-relate…
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patie…
Remote Sputum Collection in Adults With Cystic Fibrosis
Elexacaftor/Tezacaftor/Ivacaftor or Trikafta improves lung health in people with cystic fibrosis (CF), including decreased cough and mucous production. Diagnos…
Assessing the Feasibility of Home Nighttime Cough Monitoring in Children with Cystic
Cystic fibrosis (CF) is a disease characterized by chronic airway infection and impaired mucociliary clearance, which predisposes those affected to recurrent p…
Health Outcomes of Parents With Cystic Fibrosis-Aim 2
The goal of this observational prospective study is to determine the health impact of parenthood on United States (US) people with CF in the era of CF transmem…
Kidney Function in People With Cystic Fibrosis in the Era of HEMT
The purpose of this study is to find out what causes kidney disease in people with CF. The investigators will study biomarkers in the blood and urine that can …
Studying the Presence of CFRD Complications With Thoughtful Recruitment (SPeCTRuM)
This multicenter cross-sectional study will include a diverse population of adolescents and adults with CF. The overall Aim is to describe prevalence of diabe…