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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 …
A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-121/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Through 11 Years of Age
The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF pa…
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…
Outcomes Related to Body Composition in Teens and Adults With Cystic Fibrosis (ORBIT-CF)
Nutrition and body composition, the amount of muscle and fat in the body, has a role in overall health. This study wants to learn more about how nutrition and …
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…
A Phase 1, Randomized, Double-blind, Placebo-controlled, Dose Escalation Study Evaluating Safety and Pharmacokinetics of VX-581 in Healthy Subjects
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of single and multiple ascending doses of VX-581.
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…
A Phase 1, Study of VX-828 in Healthy Subjects and in Subjects With Cystic Fibrosis
The purpose of the study is to evaluate safety, tolerability, and pharmacokinetics of VX-828 and VX-828 in triple combination (TC) with Tezacaftor (TEZ)/ VX-11…
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…