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Umbilical Cord Blood Transplantation Using a Myeloablative Preparative Regimen for the Treatment of Hematological Diseases
This is a treatment guideline for an unrelated umbilical cord blood transplant (UCBT) using a myeloablative preparative regimen for the treatment of hematologi…
BEGIN Novel ImagiNG Biomarkers
To determine the treatment effect of triple-combination therapy in 6-8 year olds after presumed FDA approval, using rapid structural and functional pulmonary a…
The Role of Quantitative CT and Radiomic Biomarkers for Precision Medicine in Pulmonary Fibrosis
This observational study involves obtaining 2 chest CT scans; a historical baseline CT within ±1 year of enrollment into PRECISIONS, and a follow-up CT (either…
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 Randomised, Double-blind, Placebo-controlled, Multicentre, Phase III Trial Evaluating Long-term Efficacy and Safety of Survodutide Weekly Injections in Adult Participants With Noncirrhotic Non-alcoholic Steatohepatitis/Metabolic Dysfunction-associated Steatohepatitis (NASH/MASH) and (F2) - (F3) Stage of Liver Fibrosis
This study is open to adults who are at least 18 years old living with obesity and have: * a confirmed liver disease called non-alcoholic steatohepatitis (NAS…
An Open Label Pilot Trial of the Combination of Tagraxofusp With Pacritinib in Patients With Intermediate-1 or Higher Myelofibrosis, Who Have Had Prior Therapy With the Approved JAK Inhibitors or in Which Therapy With the Approved JAK Inhibitors is Not Appropriate, Contraindicated or Declined
The goal of this open-label, single-center, pilot trial is to test the combination of Tagraxofusp (TAG) with Pacritinib (PAC) in patients with intermediate-II …
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…
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…
An Open Label, In-Clinic Study to Assess Respiratory Function and Safety of Escalating Doses of Nalbuphine Extended-Release Tablets (NAL ER) in Subjects With Idiopathic Pulmonary Fibrosis (IPF)
The primary purpose of this study is to assess the safety and tolerability of escalating doses of NAL ER and to evaluate its effect on respiratory function in …
High Dose Ruxolitinib and Allogeneic Stem Cell Transplantation in Myelofibrosis Patients With Splenomegaly
To learn if giving ruxolitinib and busulfan before a stem cell transplant can help to reduce spleen size and help the transplant to succeed.
Non-Invasive Biomarkers for Metabolic Liver Disease (NIMBLE) Study 2.0 - An FNIH Biomarkers Consortium Study
The Non-Invasive Biomarkers for Metabolic Liver Disease (NIMBLE) study is a comprehensive, multi-year collaborative effort to standardize, validate and advance…
PET Study of 68Ga-FAPi-46 in Patients With Interstitial Lung Disease: an Exploratory Biodistribution Study With Histopathology Validation.
This is a prospective exploratory biodistribution study in patients with interstitial lung disease (ILD). The purpose of this research study is to determine w…