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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…
An Optimal Dose Finding Study of N-Acetylcysteine in Patients With Myeloproliferative Neoplasms
This is a phase I/II study evaluating the optimal dose of N-acetylcysteine (N-AC) in patients with myeloproliferative neoplasms (MPN).
Collection of Airway, Blood and/or Urine Specimens From Subjects for Research Studies
The purpose of this study is to obtain biologic materials from the blood, airways and/or urine of normal individuals and individuals with lung disease. The nor…
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…
Metformin to Attenuate Progressive Respiratory Decline in Idiopathic Pulmonary Fibrosis (MAVRIC)
This is a randomized, placebo-controlled trial of metformin in 400 participants with idiopathic pulmonary fibrosis (IPF) who are at high risk of adverse clinic…
Therapeutic RSK1 Targeting in Myelofibrosis
This is a phase Ib study evaluating PMD-026, an oral inhibitor of ribosomal protein S6 kinase A1 (RSK1), in participants with myelofibrosis (MF).The dose escal…
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…
A Phase I Open Label Peptide Based Vaccine in Patients With Myeloproliferative Neoplasm Harboring CALR Mutations
The primary objective of this study is to assess the safety and tolerability of administrating mutated-CALR peptide Vaccine to patients with MPN. The researche…
Myeloablative Allogeneic Hematopoietic Cell Transplantation Using a Related or Unrelated Donor for the Treatment of Hematological Diseases
This is a Phase II study of allogeneic hematopoietic stem cell transplant (HCT) using a myeloablative preparative regimen (of either total body irradiation (TB…
A Phase 3, Randomized, Double-blind, Add-on Study Evaluating the Safety and Efficacy of Navtemadlin Plus Ruxolitinib vs Placebo Plus Ruxolitinib in JAK Inhibitor-Naïve Patients With Myelofibrosis Who Have a Suboptimal Response to Ruxolitinib
This clinical trial is evaluating whether addition of navtemadlin to ruxolitinib treatment will provide more clinical benefit than ruxolitinib alone for patien…
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…
Hyperpolarized Xenon-129 MRI in Idiopathic Pulmonary Fibrosis
Idiopathic pulmonary fibrosis has a poor prognosis with limited treatment options. The Investigator hypothesize hyperpolarized Xe129-MRI can be performed in pa…