Phase 2 Study of SAR302503 in Patients With Myelofibrosis
Hematopoietic NeoplasmPrimary Objective: - To evaluate the efficacy of daily oral doses of 300 mg, 400 mg, and 500 mg SAR302503 for the reduction of spleen volume as determined by magnetic resonance imaging (MRI). Secondary Objectives: To evaluate the safety of SAR302503. To evaluate the pharmacokinetics (PK) of SAR302503 after single and repeat doses. To evaluate the pharmacodynamics of SAR302503 as measured by changes in JAK2V617F allele burden in those patients with JAK2V617F mutation, changes in substrate phosphorylation in the JAK-STAT signal transduction pathway, and the expression of cytokines. To measure improvement in baseline Myeloproliferative Neoplasm (MPN) associated symptoms, as well as overall impact in quality of life (QOL), through serial administration of the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF). To measure generic health-related quality of life (HRQL) and utility values using the EQ-5D questionnaire.
Study of Ruxolitinib (INCB018424) Sustained Release Formulation in Myelofibrosis Patients
MyelofibrosisThe purpose of this study is to determine the safety and tolerability of ruxolitinib (INCB018424) sustained release (SR) formulation in participants with primary myelofibrosis (PMF), post-polycythemia vera MF (PPV-MF), and post-essential thrombocythemia MF (PET-MF).
Efficacy and Safety of Simtuzumab in Adults With Primary, Post Polycythemia Vera or Post Essential...
MyelofibrosisThis study is to evaluate the efficacy and safety of simtuzumab (GS-6624) on bone marrow fibrosis either alone or in combination with ruxolitinib in participants with primary myelofibrosis (PMF) and post polycythemia vera or post essential thrombocythemia myelofibrosis (ET/PV MF). The study is designed as a two-stage trial. In the stage 1, participants will be randomized into two cohorts to receive either 200 or 700 mg of study drug. In the stage 2, participants on ruxolitinib will be randomized to receive either 200 or 700 mg of study drug.
A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological...
Adult Acute Lymphoblastic Leukemia in RemissionAdult Acute Myeloid Leukemia in Remission46 moreThe purpose of this research study is to compare the survival rates of patients with better risk disease undergoing hematopoietic stem cell transplant (HSCT) to the survival rates reported in the medical literature of similar patients undergoing reduced intensity HSCT from matched related donors.
INC424 for Patients With Primary Myelofibrosis, Post Polycythemia Myelofibrosis or Post-essential...
MyelofibrosisThe primary objective of this study was to collect additional safety of INC424 in patients with Primary Myelofibrosis, Post Polycythemia Myelofibrosis or Post-essential Thrombocythemia Myelofibrosis, who either received prior treatment with commercially available agents or who have never received treatment.
A Long-Term Study of the Effects of Orally Administered SAR302503 in Patients With Myelofibrosis...
MyelofibrosisThe purpose of this study is to evaluate the long-term effects of orally administered SAR302503 (TG101348) in patients with myelofibrosis who have completed the MF-TG101348-001 study.
Fludarabine Phosphate and Total Body Irradiation Followed by a Donor Peripheral Stem Cell Transplant...
Atypical Chronic Myeloid LeukemiaBCR-ABL1 Negative14 moreThis phase II trial studies the side effects and best dose of total-body irradiation when given together with fludarabine phosphate followed by a donor peripheral stem cell transplant in treating patients with myelodysplastic syndromes (MDS) or myeloproliferative disorders (MPD). Giving low doses of chemotherapy, such as fludarabine phosphate, and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. Giving chemotherapy or radiation therapy before or after transplant also stops the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil after the transplant may stop this from happening.
CC-4047 in Treating Patients With Myelofibrosis
Chronic Myeloproliferative DisordersSecondary MyelofibrosisRATIONALE: Biological therapies, such as CC-4047, may stimulate the immune system in different ways and stop cancer cells from growing. CC-4047 may also stop the growth of cancer cells by blocking blood flow to the cancer. PURPOSE: This trial is studying the side effects and best dose of CC-4047 and to see how well it works in treating patients with myelofibrosis.
Sibling Donor Peripheral Stem Cell Transplant or Sibling Donor Bone Marrow Transplant in Treating...
Chronic Myeloproliferative DisordersGraft Versus Host Disease4 moreRATIONALE: Giving chemotherapy before a donor peripheral stem cell transplant or bone marrow transplant using stem cells from a brother or sister that closely match the patient's stem cells, helps stop the growth of cancer or abnormal cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer or abnormal cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving colony-stimulating factors, such as G-CSF, to the donor helps the stem cells move from the bone marrow to the blood so they can be collected and stored. Giving methotrexate and cyclosporine before and after transplant may stop this from happening. It is not yet known whether a donor peripheral stem cell transplant is more effective than a donor bone marrow transplant in treating hematologic cancers or other diseases. PURPOSE: This randomized phase III trial is studying filgrastim-mobilized sibling donor peripheral stem cell transplant to see how well it works compared with sibling donor bone marrow transplant in treating patients with hematologic cancers or other diseases.
CEP-701 for PH-negative Myelofibrosis
LeukemiaMyelofibrosisThe goal of this clinical research study is to find out if CEP-701 can help control myelofibrosis (MF). The safety of CEP-701 will also be studied.