Active clinical trials for "Thrombocytopenia"

Primary Objective: To evaluate the safety and efficacy of human umbilical cord-derived mesenchymal stem cells（hUC-MSCs） to treat refractory immune thrombocytopenia（ITP）. Secondary Objective: To observe the changes of immune function in refractory ITP patients with human umbilical cord-derived mesenchymal stem cells（hUC-MSCs） after infusion, and to explore and reveal the mechanism of hUC-MSCs in treating ITP.

The main purpose of this study is to evaluate the efficacy and safety of iptacopan in participants with autoimmune benign hematological disorders such as primary immune thrombocytopenia and primary cold agglutinin disease.

Circulating blood platelets are small cellular elements that help to control bleeding (a process called hemostasis) and to avoid hemorrhage when blood vessels are injured. Platelets originate from cells in the bone marrow, the megakaryocytes (MKs), following a complex process of morphological transformation and maturation, which finally leads to the production of blood platelets. Multiple genes are implicated in this process. Constitutive thrombocytopenia (CT) are rare hematological diseases characterized by a decreased number of circulating platelets that are often larger than normal, that may lead to more or less severe hemorrhagic events. However, CT can be difficult to diagnose and differentiate from various forms of acquired thrombocytopenia. The ultimate diagnosis for CT is thus based on the molecular diagnosis, obtained by identifying and characterizing the abnormal gene and protein. About 40 genes / proteins have been identified so far as causal in CT, however, in about half of the patients suspected to have CT, genomic analysis does not detect a variant in one of these genes, and etiology of CT thus remains unknown. But insuring the diagnosis of CT is important: it will avoid misdiagnosis and inefficient or deleterious therapeutic interventions, while allowing a proposal of an adapted curative/preventive medical action. At the Resource and Competence Center for Constitutional Hemorrhagic Diseases (CRCMHC) (University Hospital Robert Debré, Paris, France), the investigating team has evidenced in unrelated patients presenting with familial forms of thrombocytopenia and no known molecular diagnosis, variants of genes not yet described as formally implicated in the occurrence of CT. Molecular genetic evidence must be completed by functional studies. Such functional studies are conducted in a research laboratory from the National Institute for Health and Medical Research (Inserm), "Innovative Therapies in Haemostasis (IThEM)" (Faculty of Medical Sciences, University of Paris, Paris, France), and include: an evaluation of how blood progenitor cells mature into MKs, by comparing cells obtained from patients to those of members free of the disease (the latter taken as normal control subjects); an evaluation of platelet functionalities, such as ability to form a blood clot similar to what happens during hemostasis, with the aim to detect not only quantitative (number and size) but also any qualitative (functions) defects; an evaluation of the ultrastructure (the structure of intracellular components) and biochemistry of MKs and platelets, focusing on the molecular pathways the variant protein is implicated in. This clinical trial is aimed to precisely delineate the mechanism of action of newly identified CT genetic variants, and will fulfill the aims of (1) offering the patient(s) a formal molecular diagnosis of CT, (2) ameliorating patients' medical support, both for diagnosis and therapy, (3) providing patients and family members with a pertinent genetic counseling, and (4) expanding the validated panel of genes implicated in CT to be explored in new suspected cases of CT. It will also help in extending the basic knowledge of the process of MK and platelet formation.

Randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of Hetrombopag in subjects with chemotherapy-induced thrombocytopenia receiving chemotherapy for the treatment of solid tumors.

This is an open-label long-term multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in adult patients with primary ITP.

This is a phase 3, multicenter, randomized, double-blinded, placebo-controlled, parallel-group trial to evaluate the efficacy, safety, and effect on QoL/PRO of efgartigimod PH20 SC treatment in adult patients with primary ITP.

Myelodysplastic syndromes (MDS) prevail in older age and are characterized by ineffective erythropoiesis and peripheral cytopenias. Supportive therapy is the main therapeutic option for most patients. Quality of Life (QoL) is mainly deteriorated by anemia and by the limitations associated with thrombocytopenia, neutropenia and transfusion dependence. The only available treatment for severe thrombocytopenia, in the presence of bleeding, is platelet transfusion. Eltrombopag is an orally bioavailable agonist of the thrombopoietin receptor. In adult patients with chronic immune thrombocytopenia (ITP), Eltrombopag rapidly increases platelet counts and significantly reduces bleeding episodes during treatment. Eltrombopag is well tolerated. In 2007, Eltrombopag has received the Orphan Drug Designation for the treatment of ITP (EMEA/OD/031/07), and in 2008 the Food and Drug Association approved Eltrombopag for the treatment of ITP refractory or resistant. It has been shown that in patients affected by MDS and by acute myeloid leukemia, Eltrombopag neither increases the proliferation, nor the clonogenic growth capacity of bone marrow blasts. Furthermore, Eltrombopag induces an increase in the megakaryocytic differentiation and in the formation of normal megakaryocytic colonies. These results provide the rationale for pursuing further research on Eltrombopag for the treatment of thrombocytopenia in case of MDS. The study is open to adult patients with myelodysplastic syndrome (MDS) with thrombocytopenia and low- or intermediate-1 IPSS risk (Index Prognostic Score System). Severe thrombocytopenia associated with MDS may lead to death from hemorrhage, even in low prognostic risk patients. The benefit of platelet transfusion is short-termed. Patients become refractory in the long term. The availability of a treatment that induces the increase of platelet count is extremely important, either in terms of quality of life, and in overall survival.

This clinical trial studies the use of reduced intensity chemotherapy and radiation therapy before donor stem cell transplant in treating patients with hematologic malignancies. Giving low doses of chemotherapy, such as cyclophosphamide and fludarabine phosphate, before a donor stem cell transplant may help stop the growth of cancer cells. It may also 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 cells (graft-versus-tumor effect). Reducing the intensity of the chemotherapy and radiation may also reduce the side effects of the donor stem cell transplant.

Evaluation of the Efficacy and Safety of Oral Azacitidine plus Best Supportive care versus Placebo and Best Supportive care in subjects with red blood cell (RBC) transfusion-dependent anemia and thrombocytopenia due to International Prognostic Scoring System (IPSS) lower risk myelodysplastic syndromes (MDS).

The purpose of this study is to see if the study drug, romiplostim, helps low platelet count caused by standard chemotherapy treatment for lymphoma. This study will also look at whether romiplostim can prevent the need for chemotherapy dose delays, chemotherapy dose reductions, and platelet transfusions. In addition, we will determine how safe it is to give romiplostim to people with lymphoma who have low platelet count from chemotherapy.