View clinical trials related to Blood Platelet Disorders.
Filter by:To evaluate the safety and feasibility of collecting hematopoietic stem cells (HSC) in participants with RUNX1-FPD.
This is an open-label, multicenter study to evaluate the safety, tolerability, and efficacy of HMPL-523 in adult subjects with ITP.
To learn about the safety and effects of low-dose sirolimus in participants with RUNX1-FPD.
Background: Runt-related transcription factor 1 (RUNX1) gene regulates the formation of blood cells. People with mutations of this gene may bleed or bruise easily; they are also at higher risk of getting cancers of the blood, bone marrow, and lymph nodes. Objective: To test a drug (imatinib) in people with RUNX1 mutations that cause symptoms. Eligibility: Adults aged 18 and older with RUNX1 mutations. Healthy people without this mutation, including family members of affected participants, are also needed. Design: Participants with the RUNX1 mutation will be screened. They will have a physical exam with blood and urine tests. They will have a test of their heart function. They may need a new bone marrow biopsy: A sample of soft tissue will be removed from inside a bone. Imatinib is a tablet taken by mouth once a day, every day, at home. Affected participants in different parts of the study will take imatinib for either 28 days or up to 84 days. Participants will visit the clinic once a week for the first 28 days that they are taking the imatinib. Then they will come once every 2 weeks if they are taking the drug for 84 days. Blood, urine, and tests of heart function will be repeated. They may opt to have the bone marrow biopsy repeated after they finish their course of imatinib. Participants will have a follow-up visit 30 days after they stop taking imatinib. Participants who do not have the RUNX1 mutation will have 1 clinic visit. They will have blood tests. They will fill out questionnaires. They may opt to have a bone marrow biopsy....
This study aims to improve platelet function testing during bleeding investigations. To this end, the study will evaluate the diagnostic accuracy of novel platelet function tests in patients with confirmed or suspected platelet function disorders. Study participants will be recruited from patients that are referred to or treated at the Coagulation Unit, Karolinska University Hospital, and Pediatric Coagulation Unit, Astrid Lingren Children's Hospital.
A cross-sectional study will be conducted, which will include 80 young patients with psoriasis, aged 30-45 years, treated with five different types of antipsoriatic treatment, and 20 healthy patients. All 100 subjects will be subjected to anthropometric measurements, blood will be collected for laboratory tests, and an imaging test will be performed to determine the function of the endothelium and arterial stiffness. The results will then be statistically analyzed.
Non-cardiac acute and chronic inflammatory conditions are associated with high risk of acute myocardial infarction. Specifically, there are reports of high prevalence of AMI and cardiac death in chronic conditions such as Rheumatoid arthritis, chronic gum disease, psoriasis and Chronic airway disease. Furthermore, there are intriguing temporal links between acute non-cardiac conditions, including fractured neck of femur and admission for chest infection in the elderly and subsequent risk of AMI within the next few weeks. Finally, a more recent association has been reported between COVID vaccination and acute thrombotic events. In Summary, a link between acute non-cardiac inflammatory conditions and subsequent AMI in a near term envelope is established, but unexplained, and circumstantial evidence so far suggests a possible mechanism in terms of dynamic alteration in platelet reactivity. It is this concept we wish to explore further in the proposed set of experiments. Our experiments may provide some insight into a potential mechanism of such an association, which could have implications for future tailored therapeutic interventions. We will recruit 5 groups of patients, consistent with the data produced previously and the literature regarding disease models of non-cardiac inflammations. Aiming to recruit 20 patients per group with 100 candidates in total. Groups including: 1. Fracture neck of femur. 2. Patients >70 years age admitted with chest infection. 3. Healthy volunteers receiving fourth COVID booster vaccine. 4. Patients admitted with AMI within 6 weeks of (fractured neck of femur, chest infection Rheumatoid arthritis flare up, exacerbation of psoriasis and exacerbation of inflammatory bowel disease). 5. AMI secondary to stent thrombosis. Study will be undertaken within the Cardiothoracic unit at University Hospital Southampton, the sponsor will be UHS Research and Development Department, UHS.
The purpose of this study is to develop a highly sensitive method for assessing the functional activity of platelets for use in the differential diagnosis of thrombocytopenia and platelet defects.
This project seeks to perform whole genome sequence (WGS) and whole transcriptome sequence (WTS) analysis on 350 patients with suspected inherited bone marrow failure syndromes and related disorder (IBMFS-RD) in order to increase the genomic diagnostic rate in IBMFS.
The purpose of the study is to evaluate the efficacy and safety of avatrombopag for the promotion of platelet engraftment after Allo-HSCT.