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Quantitation and Spatial Registration of Airways Dysfunction With Dynamic 19F MRI in Cystic Fibrosis
The purpose of this study is to look at lung ventilation in people with cystic fibrosis over time (1 year) using magnetic resonance imaging (MRI) with an inhal…
Gastrointestinal Response of Pediatric Cystic Fibrosis Patients on Mediterranean Diet
There is no study to date that has evaluated the impact or effect of a Mediterranean diet in children with CF. The goal of this study would be to help provide …
Study to Enable New Diagnostics for Pulmonary Microbes in People With CF (SEND-CF)
Sputum culture has been the best approach to detect harmful bacteria in the lungs of people with cystic fibrosis (CF). With the widespread use of new CF therap…
Implementing Digital Retinal Exams Into Comprehensive Pediatric Diabetes Care
The purpose of this study is to determine if use of a nonmydriatic fundus camera using autonomous artificial intelligence software at the point of care increas…
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…
Evaluation of Ventilation Defects Downstream of Mucus Plugs in Patients With Muco-Obstructive Lung Disease
In this study, xenon MRI will be used to evaluate regional functional consequences of mucus plugs in the lungs of patients with muco-obstructive pulmonary dise…
Health-Related Quality of Life and Metabolic Outcomes in PCOS: A Feasibility Study of Comprehensive PCOS Education and Lifestyle Modification Support Delivered Via Synchronous Virtual Groups
The primary outcome of interest is change in PCOS health-related quality of life, while the secondary outcome of interest is change in adiposity, cardiometabol…
A Phase 1/2 Study of CB-103 (Oral Pan-NOTCH Inhibitor) With Abemaciclib or Lenvatinib in Combination in Patients With NOTCH Activated Adenoid Cystic Carcinoma (CALCulus)
The goal of this study is to treat patients with NOTCH active advanced adenoid cystic carcinoma (ACC) tumors with a combination or two different oral medicatio…
A Phase 1/2, First-in-human, Multicenter Study of Emiltatug Ledadotin (Emi-Le) in Participants With Solid Tumors
A Study of Emi-Le in Participants with Solid Tumors
Glucose Metabolism in CFRD: Exploring the Role of CFTR Modulators in Metabolic Dysfunction
The study aims to test whether use of CFTR modulators (ETI) improves fasting and post prandial glycemia by enhancing disposition index (DI) in individuals with…
A Phase 1/2, Multicenter, Open-label Study of REM-422, a MYB mRNA Degrader, in Patients With Recurrent, Metastatic, or Unresectable Adenoid Cystic Carcinoma
The goal of this study is to determine the safety and antitumor effects of REM-422, a MYB mRNA degrader, in people with advanced Adenoid Cystic Carcinoma (ACC)
A Randomized Trial of the Insulin-only Bionic Pancreas in Cystic Fibrosis Related Diabetes
This multi-center randomized controlled trial (RCT) will compare efficacy and safety endpoints using the insulin-only configuration of the iLet Bionic Pancreas…