View clinical trials related to Hemorrhagic Disorders.
Filter by:The investigators try to improve the screening of bleeding disorders in children by identifying symptoms, laboratory abnormalities and clinical scores discriminating patients congenital bleeding disorders in order to create a simple screening algorithm applicable in pediatrics, aiming for use in pre-anesthetic consultation and in consultation by pediatricians and general practitioners.
Inherited bleeding disorders (IBD) consist of a heterogeneous group of diseases including coagulation and/or platelets defects and more rarely vascular dysfunctions. A family of four patients suffering from unexplained excessive bleeding has been followed clinically in France for many years. Recently, whole exome sequencing (WES) of DNA allowed the identification of a heterozygous genetic variant which segregated to family members with bleeding diathesis. The aim of the study was to better characterize the phenotype by studying VWF and platelets in affected family members ultimately contributing to the pathogenesis of a bleeding diathesis.
In parallel with the growth of American Thrombosis and Hemostasis Network's (ATHN) clinical studies, the number of new therapies for all congenital and acquired hematologic conditions, not just those for bleeding and clotting disorders, is increasing significantly. Some of the recently FDA-approved therapies for congenital and acquired hematologic conditions have yet to demonstrate long-term safety and effectiveness beyond the pivotal trials that led to their approval. In addition, results from well-controlled, pivotal studies often cannot be replicated once a therapy has been approved for general use.(1,2,3,4) In 2019 alone, the United States Food and Drug Administration (FDA) has issued approvals for twenty-four new therapies for congenital and acquired hematologic conditions.(5) In addition, almost 10,000 new studies for hematologic diseases are currently registered on www.clinicaltrials.gov.(6) With this increase in potential new therapies on the horizon, it is imperative that clinicians and clinical researchers in the field of non-neoplastic hematology have a uniform, secure, unbiased, and enduring method to collect long-term safety and efficacy data. ATHN Transcends is a cohort study to determine the safety, effectiveness, and practice of therapies used in the treatment of participants with congenital or acquired non-neoplastic blood disorders and connective tissue disorders with bleeding tendency. The study consists of 7 cohorts with additional study "arms" and "modules" branching off from the cohorts. The overarching objective of this longitudinal, observational study is to characterize the safety, effectiveness and practice of treatments for all people with congenital and acquired hematologic disorders in the US. As emphasized in a recently published review, accurate, uniform and quality national data collection is critical in clinical research, particularly for longitudinal cohort studies covering a lifetime of biologic risk.(7)
This study will find the maximum tolerated dose (MTD) of CYNK-001 which contain NK cells derived from human placental CD34+ cells and culture-expanded. CYNK-001 cells will be given post Autologous Stem Cell Transplant (ASCT). The safety of this treatment will be evaluated, and researchers will want to learn if NK cells will help in treating Multiple Myeloma.
The improvements observed in the care of patients with hemophilia or Willebrand disease have led to an increase in their life expectancy, which today approaches that of the general population. This increase in life expectancy leads in these patients to the development of comorbidities related to aging (cardiovascular and neurological diseases, cancers and kidney diseases) (e.g "Franchini & Mannuccio", BJH, 2009). The care of these comorbidities represents a new challenge for the medical teams. Toward multiple comorbidities, polypharmacy is often associated. Many studies about medication exposure and management in older patients were published but no study was conducted to explore the medication management of older patients with hemophilia or Willebrand disease.
The aim of the study was to investigate the possible relationship about blood gas and bleeding disorders in healthy volunteers of blasters and no blasters groups of the Italian National Mountain and Cave Rescue who handled nitrogen compounds and nitroglycerine and then they were exposed to combustion products from an accidental uncontrolled detonation of micro-charges during a cave unblocking procedure.
Treatment of hemorrhoidal disease includes a conservative approach (dietary and behavioral measures, venotropic and topical medication), office-based treatments and surgery. Rubber banding is currently considered the instrumental method of choice in the treatment of hemorrhoidal disease grades I to III (Goligher's classification). However, its use in patients with bleeding disorders is not recommended. Sclerotherapy can be performed in these patients since the hemorrhagic risk is very low. The most commonly used agent for sclerotherapy is liquid polidocanol. Polidocanol foam seems to be more effective than the liquid formulation and is safe in the treatment of hemorrhoidal disease even in patients with coagulation disorders. This study is aimed to evaluate the efficacy and safety of polidocanol foam sclerotherapy in the treatment of hemorrhoidal disease grades I to III in patients with bleeding disorders.
The aim of this prospective, observational study is to establish a dataset on the frequency of bleeding events, as well as other characteristics of bleeding events and FVIII infusions, in patients with clinically severe hemophilia A receiving prophylactic FVIII replacement therapy as standard of care. The data collected from this study may assist in providing baseline information for comparison to the Spark's investigational hemophilia A gene therapy in future Phase 3 studies.
Characterizing the impact and treatment of reproductive tract bleeding on women and post-menarchal girls with bleeding disorders. Objectives: This is a cross-sectional observational study of women and girls (WG) with bleeding disorders enrolled in the American Thrombosis and Hemostasis Network (ATHN) dataset. Based on the investigators' study of currently available data of WG with bleeding disorders in the ATHNdataset, the investigators hypothesize that the information currently captured in the core data elements of the ATHNdatasest does not adequately capture data specific to WG with bleeding disorders. Further, the investigators hypothesize that it is feasible for Hemophilia Treatment Centers (HTCs) to include data points specific to WG with bleeding disorder when enrolling participants in the ATHNdataset. This hypothesis will be evaluated through the following specific aims: Specific Aim 1: Characterize reproductive tract bleeding in a cohort of WG with bleeding disorders cared for at US HTCs. Specific Aim 2: Characterize the treatment strategies for and the impact of heavy menstrual bleeding in a cohort of females with bleeding disorders cared for at HTCs. Specific Aim 3: Evaluate the feasibility of adding female specific core data points to the ATHNdataset.
SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with severe hemophilia A and no measurable inhibitor against FVIII.