Active clinical trials for "Von Willebrand Diseases"

The purpose of this study is to determine the efficacy and safety of rhIL-11, when given subcutaneously for six consecutive months, in reducing menstrual blood loss in women with type 1 von Willebrand disease and refractory menorrhagia. Efficacy will be measured by subjective bleeding severity scale and pictorial bleeding chart. Safety will be measured by the frequency of adverse events, including fever, headache, fatigue, myalgias, arthralgias, fluid retention, or edema.

To evaluate the effect of ARC1779, a therapeutic oligonucleotide ("aptamer") in patients with Type2B von Willebrand Disease.

The purpose of the study is to determine whether oral contraceptives, desmopressin acetate, and/or tranexamic acid are effective in the treatment of women with menorrhagia who are diagnosed with a bleeding disorder.

Cerebral microbleeds (CMBs) are haemosiderin deposits, resulting from the leakage of erythrocytes from small cerebral vessels, which can be detected noninvasively using susceptibility-sensitive magnetic resonance imaging (MRI) techniques. CMBs are commonly observed in daily practice: their prevalence range from five percent in healthy individuals over 65 years old to 50% in patients with a history of stroke. CMBs are associated with intracerebral hemorrhage (ICH) and also cognitive impairment and dementia. The pathophysiology of CMBs is thought to primarily involve damage to brain microvasculature but the exact underlying cascade of events, including a potential role for haemostasis, has yet to be elucidated. Haemostatic defects (congenital or acquired) may contribute to an increased number and importance of CMBs. Congenital bleeding disorders such as haemophilia or von Willebrand disease (vWD), populations at high risk of ICH, are unique conditions that may give us further insights into a potential role of haemostatic defects in the pathophysiology of CMBs. CMBs might be the missing link between severe haemostatic defects, ICH risk and cognitive function. We hypothesized that severe congenital haemostatic defects could contribute to an increased prevalence and number of CMBs, with an impact on cognition in adulthood.

The study aims to study the expression of numerous proteins involved in angiogenesis in 70 patients with von Willebrand disease in order to try to identify markers of interest. Secondly, the investigators plan to investigate whether there is a relationship between the proteins tested, the distribution of multimers and the clinical phenotype of the patients, in particular by looking for the presence of bleeding linked to the presence of angiodysplasias.

The objectives of this study are to evaluate the immediate tolerability and safety of rVWF:rFVIII in subjects with Type 3 Von Willebrand Disease after administration of various dosages of VWF:RCo.

Primary Objective: -To characterize the pharmacokinetics (PK) of BIVV001 after a single intravenous (IV) administration, as assessed by factor VIII (FVIII) activity determined by the one-stage activated partial thromboplastin time (aPPT) clotting assay, as well as, BIVV001 capture chromogenic Coatest FVIII activity assay Secondary Objective: -To assess the safety and tolerability of a single IV dose of BIVV001 in adult patients with type 2N and 3 VWD

During treatments with extracorporeal circuits such as extracorporeal membrane oxygenation (ECMO) degradation of high molecular weight (HMW) of von Willebrand factor (vWF) multimers occur leading to an acquired von Willebrand disease. This disease is associated with increased bleeding and requirement for the transfusion with allogenic blood products especially packed red blood cells (PRBCs). A continuous treatment with von Willebrand factor concentrate (vWFC) may restore the multimers and bleeding can be avoided. Therefore a randomized, double-blind, prospective, controlled, two-arm clinical trial was designed, comparing patients receiving vWFC versus placebo.

Von Willebrand Disease (VWD) is defined as an inherited bleeding disorder that is caused by deficiency or dysfunction of von Willebrand factor (VWF), a plasma protein that mediates the initial adhesion of platelets at sites of vascular injury and also binds and stabilizes blood clotting factor VIII (FVIII) in the circulation. The most severe forms of VWD are usually easy to diagnose (obvious hemorrhagic symptoms and major VWF deficiency), whereas the mild forms of the disease are still difficult to confirm. It is indeed reported that about 1% of the population carry mild biological VWF deficiency without any bleeding tendency and any "actual disease". On the contrary, some patients with severe bleeding history can carry a true VWF abnormality, well-confirmed by genetic studies, without any VWF deficiency when evaluated with standard biological methods, such as Ristocetin Cofactor activity (VWF:RCo). However, in these patients, the use of alternative methods, such as PFA-100 (Platelet Fonction Analyzer-100), the study of Factor VIII (FVIII:C) to VWF (FVIII:C/VWF) ratio or the evaluation of VWF activity using more specialized methods such as VWF:CB (VWF-Collagen Binding) assay can detect the VWF deficiency and possible hemorrhagic predisposition. In this project, the investigators plan to assess the performance of VWF:CB in the diagnosis of VWF deficiency in patients with unexplained bleeding history.

Von Willebrand disease (VWD) is the most common constitutional bleeding disorder in the world, caused by missing or defective von Willebrand factor (VWF). In France, VWD affects approximatively 7,000 patients. There are many types of VWD. The severest forms are characterized by the occurrence of extremely serious bleedings, requiring in-stays with clotting factors (CF) treatments in specialized hospital units and/or an ambulatory substitutive therapy; both of them are highly expensive. In France, Hemostasis Treatment Centers (HTC) have the opportunity to record these kinds of data in a database called NHEMO (Net-Hemostasis = care database for constitutional bleeding disorders). Further ahead, the data can be coded, dumped into and extracted from the research database BERHLINGO and analyzed. The HOPSCOTcH-WILL study will be a retrospective, non-interventional, multicenter (national) cohort study & will provide an overview of the real-life management of patients with VWD in western France requiring a substitutive treatment with VWF, as well as a description of the characteristics of their hemorrhagic events. Model : Observationnal, real world evidence study. Time Horizon : 2015-2018. HTC (France): Western University Hospitals (BERHLINGO network) = Nantes University Hospital (promotion), Angers University Hospital, Brest University Hospital, Le Mans Regional Hospital & Rennes University Hospital