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Nucleoside Therapy in Patients With Telomere Biology Disorders
The goal of this clinical trial is to learn if a combination therapy of deoxycytidine (dC) plus deoxythymidine (dT) is safe in patients with telomere biology d…
Streamlined Treatment of Pulmonary Exacerbations in Pediatrics Randomized Controlled Trial (STOP PEDS RCT)
The STOP PEDS RCT is a multicenter, parallel, open label randomized controlled trial evaluating the long-term (one year) and short-term safety and efficacy of …
A Phase 1/2, Open-Label, Dose-Escalation, Safety, Pharmacokinetic, and Pharmacodynamic Study of Oral Nuvisertib (TP-3654) in Patients With Intermediate or High-Risk Primary or Secondary Myelofibrosis
This study is a Phase 1/2, multicenter, dose-escalation, open-label trial to assess safety, tolerability, pharmacokinetics and pharmacodynamics of nuvisertib (…
Quantitative MRI for Myelofibrosis - MRI Parameters as Biomarkers for Analyzing Extent of Disease and Measuring Response to Treatment
This study is for the development and validation of functional magnetic resonance imaging (MRI) parameters as biomarkers for analyzing extent of disease and qu…
A Prospective Multicenter International Randomized Controlled Trial Comparing Surgical and Medical Therapies in the Treatment of Advanced Metabolic Dysfunction Associated Steatohepatitis
Metabolic dysfunction-associated steatotic liver disease (MASLD), formerly known as non-alcoholic fatty liver disease (NAFLD), a major global public health con…
Comparison of Continous Glucose Monitor and OGTT as a Screen for Cystic Fibrosis Related Diabetes and Impaired Glucose Tolerance
Cystic Fibrosis (CF) related diabetes (CFRD) is a unique form of diabetes mellitus, different from type 1 diabetes and type 2 diabetes. The diagnosis of CFRD i…
A Phase 1, Open-Label, Multi-Center, Safety and Efficacy Study of PRT12396 in Participants With Polycythemia Vera and Myelofibrosis
This is a first-in-human, open-label, multi-center Phase 1 study designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of P…
Phase Ib/II Study Of Revumenib As Monotherapy Or In Combination With Jak Inhibitors In Patients With Myelofibrosis
This is a Phase Ib/II 2 study investigating the safety and efficacy of revumenib in two cohorts of participants with myelofibrosis. COHORT-1 will investigate t…
A Phase IIa, Randomised, Double-blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AZD2389 in Adult Participants With Steatotic Liver Disease and Advanced Fibrosis (BRAVO)
The purpose of this study is to evaluate the safety, tolerability, and pharmacodynamic effects of AZD2389 in adult participants with steatotic liver disease (S…
Natural History of Noncirrhotic Portal Hypertension
Background: \- Noncirrhotic Portal Hypertension (NCPH) is caused by liver diseases that increase pressure in the blood vessels of the liver. It seems to start…
A Phase 1/2 Study of Combined JAK/ERK Inhibition in Patients With Myelofibrosis
The researchers are doing this study to find out whether the combination of ruxolitinib and ulixertinib is a safe and effective treatment for people with myelo…
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…