Ruxolitinib Prior to Transplant in Patients With Myelofibrosis
Primary MyelofibrosisPost Polycythemia Vera Myelofibrosis1 moreThe purpose of this study is to find out if giving the study drug Ruxolitinib (INC424) prior to a combination of other chemotherapeutic drugs (Fludarabine and Busulfan) before infusing another person's hematopoietic stem cells (bone marrow transplantation) will be successful in people who have advanced primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF) or post-essential thrombocythemia myelofibrosis (PET-MF), collectively known as myelofibrosis (MF). MF is a disorder in which bone marrow tissue develops in abnormal sites because the bone marrow itself undergoes fibrosis or scarring. This study plans to evaluate whether adding the drug Ruxolitinib will further aid in reducing pre-transplant spleen size, improve physical performance levels and reduce adverse events (side effects) related to the transplant. Ruxolitinib is a drug that is approved by the FDA for the treatment of patients with advanced forms of myelofibrosis. Using Ruxolitinib prior to stem cell transplantation is experimental.
Anti-TGF-beta Therapy in Patients With Myelofibrosis
MyelofibrosisPrimary Myelofibrosis3 moreTGF-β is a cytokine that is found to be upregulated in the bone marrow of patients with myelofibrosis. This cytokine likely plays a dual role in promoting myelofibrosis and myeloproliferation, both of which are the bone marrow morphologic hallmark of MF. The investigators propose that inhibiting the TGF-β signaling pathway in MF will decrease the fibrogenic stimuli leading to myelofibrosis and concomitantly interrupt myeloproliferation. This is a novel approach to the treatment of patients with myelofibrosis.
129Xe MRI Cardiopulmonary
Interstitial Lung DiseaseChronic Thromboembolic Pulmonary Hypertension3 moreThe goal of this NIH-sponsored study is to characterize three biomarkers derived from 129Xe gas exchange MRI and to understand how they change in response to interventions.
Late Gestational Diabetes Mellitus Diagnosis in Obese Women
Gestational Diabetes Mellitus in PregnancyMacrosomia9 moreIn the current work, we aim to perform a prospective study that will investigate the relationship between maternal obesity (BMI >30 kg/m2) and morbid obesity (BMI >35 kg/m2) with a late GDM diagnosis (>32 weeks), with an emphasis on obstetric and neonatal outcomes.
Study to Assess the Self-administration of AOP2014 Using a Pen, Developed for the Treatment of Polycythemia...
Polycythemia VeraPolycythemia Vera (PV) is a disease of bone marrow stem cells that manifests in a drastic increase of red blood cells and frequently also of white blood cells. The "thickening" of the blood in relation with a modified function of the cells has several consequences like increased blood pressure, pruritus of the skin, fatigue, disturbed blood circulation in the brain as well as fingers and toes and an increased risk of arterial and venous thrombosis (thrombosis is the formation of a blood clot in a vessel); like stroke, cardiac infarction, deep vein thrombosis in the legs. In case of a strong increase of platelets there is an additional risk of bleedings. As the disease progresses the size of spleen and liver increased in most cases and the bone marrow shows signs of fibrosis. In some cases of PV a progression at a later time point to a leukemia (increased formation of white blood cells) can occur. The aim of this study is to assess the ease of AOP2014 self-administration using dedicated questionnaires. To assess safety and tolerability: adverse events (AEs), laboratory parameters, electrocardiogram (ECG) throughout study. To assess maintenance of the blood efficacy parameters Hct (Hematocrit), WBC (white blood cells) and PLTs (platelets) and spleen size (comparing values at Visit P7 vs. values at Visit P1). To assess the feasibility of AOP2014 self-administration: defined as the ability of the patients to use the pen as a self-administration tool (ease of handling, safety, tolerability and efficacy).
Efficacy and Safety of Ruxolitinib in the Treatment of Anemic Myelofibrosis Patients.
Primary MyelofibrosisPost-Polycythemia Vera-Myelofibrosis1 moreThis was a study of treatment with ruxolitinib in patients who presented with transfusion dependent or independent anemia. Starting dose was 10 mg BID. This dose was maintained for the first 12 weeks of the study and up-titrated thereafter unless the subject met criteria for dose hold or dose reduction
Efficacy and Safety of P1101 in Polycythemia Vera Patients for Whom the Standard of Treatment is...
Polycythemia Vera (PV)This is a Phase 2 single arm study to investigate efficacy and safety of P1101 for adult Japanese patients with PV.
Efficacy of Momelotinib Versus Best Available Therapy in Anemic or Thrombocytopenic Subjects With...
Primary Myelofibrosis (PMF)Post-polycythemia Vera (Post-PV)1 moreThis study is to determine the efficacy of momelotinib (MMB) versus best available therapy (BAT) in anemic or thrombocytopenic adults with primary myelofibrosis (PMF), or post-polycythemia vera or post-essential thrombocythemia myelofibrosis (Post-PV/ET MF) who were treated with ruxolitinib as measured by splenic response rate at Week 24 (SRR24). Participants will be randomized to receive either MMB or BAT for 24 weeks during the randomized treatment phase, after which they will be eligible to receive MMB in an extended treatment phase for up to an additional 204 weeks. After discontinuation of study medication, assessments will continue for 12 additional weeks, after which participants will be contacted for survival follow-up approximately every 6 months for up to 5 years from the date of enrollment or until study termination. For those subjects planning to continue treatment with MMB following the end of the study, the End of Treatment, 30-day, 12-Week, and survival follow-up visits are not required.
Long-term Safety and Efficacy of Momelotinib in Subjects With Primary Myelofibrosis, Post-polycythemia...
Primary MyelofibrosisPost-Polycythemia Vera Myelofibrosis3 moreThis open-label study is to determine the long-term safety and tolerability of momelotinib in previously enrolled study participants with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (post-PV MF), post-essential thrombocythemia myelofibrosis (post-ET MF), polycythemia vera (PV), or essential thrombocythemia (ET), who have tolerated and achieved stable disease or better with momelotinib treatment while enrolled in a previous clinical trial.
A Clinical Study of Ruxolitinib in Patients With Primary Myelofibrosis (PM), Post-polycythemia Vera...
Primary Myelofibrosis (MF)This is an open-label, multicenter clinical study in order to collect and examine data concerning the safety and efficacy of ruxolitinib in patients with Primary Myelofibrosis (MF), Post-Polycythemia Vera (PV) MF, Post-Essential Thrombocythemia (ET) MF.