Active clinical trials for "Hematologic Diseases"

To demonstrate the safety and effectiveness of orally-administered pyridoxal isonicotinoyl hydrazone (PIH) for the chronic treatment of iron overload.

Diamond Blackfan anemia (DBA) is a condition in which the bone marrow is underdeveloped. DBA is considered a congenital disease, meaning patients are born with it. In DBA there is a lack of cells that give rise to red blood cells. The other elements produced in the bone marrow, such as white blood cells and platelets, are normal. Standard treatments used for this disorder such as steroids and bone marrow transplants are associated with failure, relapse, side-effects, increased morbidity, and even death. Two drugs, antithymocyte globulin (ATG) and cyclosporin have been used to treat DBA, but have only provided occasional responses. No study has ever combined these two drugs for the treatment of DBA. This study is designed to explore the combined use of ATG and cyclosporine as a rational approach to the treatment of DBA.

The trial is an open-label, 4 cohorts, sequential, dose-escalating, single dose trial.

This study will randomized hematology oncology patients with active diarrhea and a NAAT positive/toxin EIA negative to either 14 days of oral vancomycin capsules or placebo. The study is designed to include 30 patients (15 per arm). Outcomes will include C. difficile load using qPCR, VRE loads, structural and functional microbiome changes and frequency of bowel movements. All endpoints will be measured at several time points including days 0, 14, 21 and 90.

To characterize the safety and tolerability of 1) MBG453 as a single agent or in combination with PDR001 or 2) PDR001 and/or MBG453 in combination with decitabine or azacitidine in AML and intermediate or high- risk MDS patients, and to identify recommended doses for future studies.

The overall purpose of this proposed study is to improve management of vaso-occlusive episodes (VOEs) in adult EDs. We aim to implement NHLBI recommendations for VOE treatment by embedding Individualized Pain Plans (IPPs) in the electronic health record (EHR). The EHR-embedded IPP will serve as a record of patients' SCD genotype and will include analgesic medication recommendations developed by the SCD provider. In this project, we will provide access to the IPP for both adult patients with SCD and ED providers. The proposed multisite study will use a pre-post study design, with a core set of mandatory intervention components and strategies for each participating site and optional components and strategies to allow for intervention adaptation to local needs and resources. The EHR-embedded IPP will be available for all adult ED providers to use as their routine practice, and patients will be invited to participate and enroll in the study. We will use a simplified Technology Acceptance Model to explain the use of the IPP and the RE-AIM framework to assess the Reach, Effectiveness, Adoption, Implementation, and Maintenance of the intervention.

PURPOSE: To investigate the effect of the disease and HSCT on muscle dysfunction and to investigate the prognostic role of muscle dysfunction at critical decision points in patients with hematological diseases referred to hematopoietic stem cell transplant (HSCT). HSCT: Patients diagnosed with malignant hematological diseases who are referred to myeloablative HSCT, to a myeloablative "reduced toxicity conditioning" regime with Fludarabine and Treosulfane (FluTreo) or to non-myeloablative HSCT.

To establish a population pharmacokinetic and pharmacodynamic model of rituximab in children with hemopathy. To optimize the administration of rituximab in the treatment of children based on pharmacokinetic model.

Allogeneic stem cell transplantation may provide long-term remissions for some patients with hematological malignancies. However, allogeneic transplantation is associated with a significant risk of potentially life threatening complications due to the effects of chemotherapy and radiation on the body and the risks of serious infection. In addition, patients may develop a condition called Graft versus host disease that arises from an inflammatory reaction of the donor cells against the recipient's normal tissues. The risk of graft versus host disease is somewhat increased in patients who are receiving a transplant from an unrelated donor. One approach to reduce the toxicity of allogeneic transplantation is a strategy call nonmyeloablative or "mini" transplants. In this approach, patients receive a lower dose of chemotherapy in an effort to limit treatment related side effects. Patients undergoing this kind of transplant remain at risk for graft versus host disease particularly if they receive a transplant from an unrelated donor. The purpose of this research study is to examine the ability of a drug called CAMPATH-1H to reduce the risk of graft versus host disease and make transplantation safer. CAMPATH-1H binds to and eliminates cells in the system such as T cells that can cause graft versus host disease (GvHD). As a result, earlier studies have shown that patients who receive CAMPATH-1H with an allogeneic transplant have a lower risk of GvHD. In the present study, we will examine the impact of treatment with CAMPATH-1H as part of an allogeneic transplant on the development of GvHD and infection. In addition, we will study the effects of CAMPATH-1H on the immune system by testing blood samples in the laboratory.

The purpose of this study is to compare standard therapy (transfusions and chelation) with alternative therapy (hydroxyurea and phlebotomy) for the prevention of secondary stroke and management of iron overload in children with sickle cell anemia (SCA).