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Role of Genetic Factors in the Pathogenesis of Lung Disease
This study is designed to evaluate the genetics involved in the development of lung disease by surveying genes involved in the process of breathing and examini…
Core A: The Hepato/Renal Fibrocystic Diseases Translational Resource (ARPKD Database Study)
Hepato-renal fibrocystic diseases (HRFD) is a term developed that encompasses rare diseases such as Autosomal Recessive Polycystic Kidney Disease (ARPKD), and …
A Phase 2 Randomized, Double-blind, Placebo-controlled Study of the Safety and Efficacy of MTX-463 in Participants With Idiopathic Pulmonary Fibrosis (IPF)
A Phase 2a, Randomized, Double-blind, Placebo-Controlled Study of the Safety and Efficacy of MTX-463 in Participants with Idiopathic Pulmonary Fibrosis (IPF)
A Comparison of a Medication Adherence Platform (FORTISKAP™) vs. Usual Care in Subjects on Oral Medications for the Treatment of Interstitial Lung Disease, Sarcoid and Pulmonary Hypertension
This study will evaluate whether participants with serious lung diseases such as idiopathic pulmonary fibrosis, sarcoidosis, and pulmonary hypertension who use…
Allogeneic Hematopoietic Cell Transplantation With Pegylated Interferon Alfa-2a for Primary and Secondary Myelofibrosis (ATIOM)
This is a single site, open-label, dose de-escalation, Phase 1 study of pegylated interferon alfa-2a administered after alloHCT in subjects with primary or sec…
Molecular Imaging Probes to Inform Heterogeneity in Idiopathic Pulmonary Fibrosis
The purpose of the study is to see if imaging with fluorine-18 Fluorodeoxyglucose (\[18F\] FDG) and fluorine-18 Displacement Per Atom (\[18F\]DPA-714) using po…
A Multicenter, Open-Label, Phase 1 Study of AJ1-11095 Administered as Oral Monotherapy in Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (PPV-MF), or Post-Essential Thrombocythemia Myelofibrosis (PET-MF) Who Have Been Failed by a Type I JAK2 Inhibitor (JAK2i)
AJX-101 is a first-in-human (FIH), phase 1, non-randomized, multi-center, open-label clinical trial designed to investigate the safety, tolerability, pharmacok…
Effect of GLP-1 Agonist Therapy on Insulin Secretion in Adults With Pancreatic Insufficient Cystic Fibrosis and Abnormal Glucose Tolerance: a Randomized, Open-label, Cross-over Trial
Diabetes is a major co-morbidity in pancreatic insufficient cystic fibrosis (PI-CF) and associated with worse outcomes. While reduced β-cell mass contributes t…
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 …
Feeding Study to Optimize Endocrine Dysfunction in Cystic Fibrosis
The goal of this study is to determine the extent to which excess dietary sugars serve as a precipitating factor in glucose intolerance in adults with cystic f…
A Medical Nutrition Supplement for Cystic Fibrosis
The goal of this study is to learn if one nutrition supplement formula works better than a different formula in adults with cystic fibrosis. The main question …
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.