Active clinical trials for "Von Willebrand Diseases"

Currently, the exploration of primary hemostasis (a physiological phenomenon used to stop bleeding) is imperfect because it is based on targeted tests for platelets or von Willebrand factor, without taking into account blood flow and other blood cells (red and white blood cells). Tests for whole blood and flow conditions exist, but there is currently no test that comes close to actual physiological conditions. An exploration of whole blood haemostasis in a device that is similar to a blood vessel and at different flow conditions (venous and arterial) could help to better identify the risk of bleeding in predisposed patients (von Willebrand factor deficiency, antiplatelet therapy). The objective of the study is to evaluate the performance of this new hemostasis test in whole blood and under flow conditions. Participation in the study is ad hoc and is limited to adding a maximum of 4 citrated tubes (20 mL or the equivalent of 4 teaspoons) and 1 EDTA tube (5mL) to a routine blood sample (for patients) or during blood donation (for controls).

ECMO has improved the outcome of heart or respiratory failure and carcinogenic shock and are increasingly used. However bleeding complications occurring in up to 50% of patients are poorly understood and worsen the overall results. The aim is to investigate the occurence of bleeding and its frequency according to the type of ECMO either veno-arterial or veno-venous. The investigators also want to assess the relation of bleeding with von Willebrand Factor defects.

The main objective of the study is to assess the pharmacokinetics of OPTIVATE® after a single dose of 80 IU/kg VWF:RCo. The secondary objectives of the study are to assess efficacy and safety of OPTIVATE® in long-term use over at least 12 months.

Due to increasing of life expectancy, patients with von Willebrand disease are exposed to age-related pathologies such as cancer or cardiovascular diseases. Management of thrombotic events is challenging given the inherent bleeding risk in von Willebrand disease. Few data are currently available in the literature. The aim of the study is to describe the frequency and nature of arterial and venous thromboembolic events and atrial fibrillation in patients with von Willebrand disease in the West of France. The investigators will perform a retrospective multicenter study conducted in the von Willebrand population of the French West. Von Willebrand adult patients followed in one of the French West medical centers participating in the study, who presented deep vein thrombosis, pulmonary embolism, ischemic stroke, transient ischemic attack, acute coronary syndrome, acute limb ischemia, atrial fibrillation, arteriopathy of the lower limbs, angina will be eligible.

Description of the long-term evolution of patients with von Willebrand disease and treated with Voncento® and of the hemostatic efficacy in the prevention and the treatment of non-surgical bleeding episodes and prevention of surgical bleeding during 2 years after patient inclusion.

International Registries and Prospective Study on Type 3 Von Willebrand's Disease (VWD3), aimed to assess number, types and risk factors for bleeding and the efficacy and safety of plasma-derived and/or recombinant Von Willebrand Factor (VWF) concentrates used to treat VWD patients.

An open, multi-centre study in patients with von Willebrand Disease (VWD) undergoing surgery.

The main objective of the study is to compare the pharmacokinetics of Optivate® with the subject's current FVIII concentrate when given as a bolus dose of 50IU/kg. The secondary objective is to compare the first and second pharmacokinetic assessments on Optivate® (and recovery if a subject changes batch) to evaluate Optivate® in terms of clinical tolerance and safety.

This is a prospective, non-controlled, international, multi-center phase 3 study investigating the efficacy and safety of Wilate in previously treated adult patients with VWD, to obtain additional data on the safety and efficacy of Wilate in previously treated patients with VWD undergoing regular prophylaxis.

BT200 is a PEGylated aptamer that binds to the A1 domain of human von Willebrand factor (VWF). At low doses, BT200 blocks the clearance of VWF antigen (VWF Ag) from the circulation and causes an increase in concentrations of both VWF Ag and Factor VIII (FVIII), but has negligible effect on the activity of either. At higher doses, BT200 blocks clearance of VWF and also inhibits its activity, but still does not inhibit FVIII activity. Therefore, low dose BT200 could potentially be used to correct deficiency of VWF and/or FVIII in patients with hereditary bleeding disorders. This study is designed as a "basket design" pilot study to determine the relevant dose and pharmacological activity of BT200 in such patients. In this open basket study up to 25 patients with the following congenital blood-clotting disorders are to be included: Patients with hemophilia A, heterozygous carriers of hemophilia A with subnormal FVIII levels; patients with von Willebrand syndrome (VWD) type 1, "Vicenza type", and with VWD type 2b. Participants will receive BT200 subcutaneously on day 0, day 4 and day 7 in the first week and then once a week for a total of five weeks - initially in a dose of 3 mg, then in week 3 individually after response in a dose of 3 to 9 mg. Subsequently, blood samples are taken once a week for a further three weeks (wash-out phase). Patients may be enrolled in an additional pharmacokinetics sub-study. For this purpose, approximately three blood samples are taken to estimate the half-life of substituted FVIII under the influence of BT200. The primary objective of this study is to obtain clinical proof of mechanism for BT200 in one or more hereditary bleeding disorders.