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A Phase 3, Randomized, Double-blind, Active-control Study of Pelabresib (DAK539) and Ruxolitinib vs. Placebo and Ruxolitinib in Adult Patients With Myelofibrosis Who Are JAK Inhibitor Naive
The purpose of this trial is to evaluate whether treatment with pelabresib in combination with ruxolitinib leads to improved clinical outcomes compared to ruxo…
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 …
A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Tolerability and Efficacy of Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03) in Patients With Idiopathic Pulmonary Fibrosis
Rationale: LTI-03 is an experimental medication breathed into the lungs using an inhaler. It is being studied for the treatment of Idiopathic Pulmonary Fibrosi…
A Multinational, Long-Term, Safety and Tolerability, Open-Label Extension Study of Subjects Who Have Participated in Avalyn Pharma Studies of Inhaled Antifibrotic Agents (AP-LTE-008 [SAIL])
This is an open-label extension study for participants who were previously enrolled in and completed an Avalyn Pharma Sponsored study with an inhaled antifibro…
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…
A Phase 1b Trial of CMV-MVA Triplex Vaccine in Haploidentical Stem Cell Donors and Recipients to Enhance CMV-Specific Immunity and Prevent CMV Viremia in Recipients of Hematopoietic Stem Cell Transplant
This phase Ib trial tests the safety, side effects, and how well cytomegalovirus (CMV)-modified vaccinia Ankara (MVA) Triplex vaccine works in enhancing CMV-sp…
A Phase 2, Multicenter, Randomized, Active-controlled Study to Assess the Safety and Efficacy of ANG003 at Two Different Dose Levels in Subjects With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
In this study, ANG003, a pancreatic enzyme replacement therapy (PERT; commonly called "enzymes"), is being investigated as a potential treatment for exocrine p…
Natural History of Bronchiectasis
Background: * Bronchiectasis is a disease characterized by airways that are inflamed, abnormally dilated, and chronically infected. Individuals with bronchiec…
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…
An Open-label, Multi-center, Long-term Extension Study to Evaluate the Long-term Safety and Tolerability of Admilparant in Participants With Pulmonary Fibrosis
The purpose of this study is to evaluate the long-term safety and tolerability of Admilparant in participants who completed participation in parent studies IM0…
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…