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The Role of Conformational Diseases on Macrophage Function
The purpose of this study is to look at how Alpha-1-antitrypsin (AAT) deficiency and Cystic Fibrosis (CF) affect white blood cells in the lungs, called macroph…
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-710 in Adults With Cystic Fibrosis
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
Targeting Inflammation With Losartan to Improve Response to Modulator Therapy in Cystic Fibrosis
The goal of this clinical trial is to test use of losartan in those with cystic fibrosis (CF) on modulator therapy. The main question it aims to answer is if t…
In Vitro Maturation of Human Eggs
CCRM Fertility, a global pioneer in fertility treatment, research and science, is seeking participants for a new study on in vitro maturation (IVM). IVM requir…
Measuring Fasting Insulin and HOMA-IR by Age, Sex, Race/Ethnicity, BMI, and PCOS Diagnosis
The study aims to investigate the relationship between fasting insulin and Homeostatic Model Assessment of Insulin Resistance (HOMA-IR) across various demograp…
A Cross-sectional Pilot Study of 19F MRI in Healthy Children and Children With Mild Cystic Fibrosis Lung Disease
This study capitalizes on the emerging technology of 19F MRI, using conventional 'thermally' polarized perfluorinated gas (perfluoropropane, or PFP) mixed with…
Early PKD Observational Cohort Study
This observational study will collect blood and urine and clinical information from individuals with early-stages of polycystic kidney disease (PKD), their una…
Glucose Monitoring in Youth With Cystic Fibrosis During Pulmonary Exacerbations (GeM-PEx)
The goal of this study is to investigate the prevalence of dysglycemia with continuous glucose monitoring (CGM) obtained during pulmonary exacerbations, both o…
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…
Role of Semaglutide in Restoring Ovulation in Youth and Adults With Polycystic Ovary Syndrome
Girls and women 12-35 years old with obesity and polycystic ovarian syndrome who are on or off metformin, will receive a glucagon like peptide-1 receptor agoni…
NOX4, Mitochondria and Related Biomarkers in Autosomal Dominant Polycystic Kidney Disease
To determine the value of NOX4, markers of mitochondria injury and function, and oxidative stress as real-time biomarkers to assess disease severity in patient…
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…