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Routine Validation and Reproducibility Testing of Laboratory Assays and Research Techniques Used for Endocrine, Cardiometabolic, and Musculoskeletal Disorder Research (VALD)
The purpose of this research study is to validate (check the accuracy of) laboratory assays, intravenous catheter insertion, and equipment or devices and their…
Characteristics of Gastroesophageal Reflux in Children With Cystic Fibrosis
The purpose of this study is to evaluate the prevalence of gastroesophageal reflux disease (GERD) symptoms in pediatric patients with cystic fibrosis using the…
Testing Drug Efficacy in Cystic Fibrosis Through N-of-1 Trials
The purpose of this study is to validate and utilize a personalized medicine approach to identify potential treatments with current FDA approved CFTR modifiers…
The Effects of a Low Glycemic Load Diet on Dysglycemia and Body Composition in Adults With Cystic Fibrosis-Related Diabetes
This study will evalute the effect of a low glycemic load (LGL diet on dysglycemia, insulin requirements, DXA-derived body composition, gastrointestinal sympto…
A Phase 1, Randomized, Double-blind, Placebo-controlled, Dose Escalation Study Evaluating Safety and Pharmacokinetics of VX-272 in Healthy Subjects
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of single and multiple ascending doses of VX-272.
Regional Monitoring of CF Lung Disease After Changes in Mechanical Airway-clearance Treatment
The main reason for this research study is to learn more about some new tests that are being developing for patients with Cystic Fibrosis (CF) to measure chang…
A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN Study)
This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) mo…
Exploring Caregiver Needs While Supporting People with CF Learning About Lung Transplant
Lung transplant is an option for treating end-stage lung disease in cystic fibrosis (CF). In the United States, more people with CF and low lung function die e…
Hyperpolarized 129Xe MR Imaging of Lung Function in Healthy Volunteers and Subjects With Pulmonary Disease
The purpose of this study is to develop and evaluate the usefulness of hyperpolarized (HP) 129Xe gas MRI for regional assessment of pulmonary function.
Impact of Highly Effective Modulator Therapy on Sinonasal Microbial Communities, Host Mucosal Inflammatory Environment and Quality of Life in Adults With Cystic Fibrosis
The goal of this observational study is to learn about the effects of a specific cystic fibrosis therapy (Cystic Fibrosis Transmembrane Conductance Regulator M…
ACT With CF Self-Help Toolkit
Acceptance and Commitment Therapy (ACT) tailored to meet the needs of adults living with cystic fibrosis (ACT with CF) is a newer form of talk therapy that has…
A Single-Site Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical resear…