Pharmacokinetics, Safety and Efficacy of Recombinant Von Willebrand Factor (rVWF) in the Treatment...
Von Willebrand DiseaseThe purpose of this Phase 3 study is to assess the pharmacokinetics of rVWF:rFVIII and rVWF, and to assess the safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding events in subjects with severe hereditary von Willebrand disease (VWD).
Phase II Study of IL-11 (Neumega) in Von Willebrand Disease
Von Willebrand DiseaseThis study is testing the use of rhIL-11 (recombinant interleukin 11, Neumega) in individuals with Von Willebrand disease. The purpose is to evaluate: if rhIL-11 corrects VWF (Von Willebrand Factor) levels to normal if rhIL-11 and DDAVP together will boost VWF levels even higher the onset, peak, and duration of rhIL-11 effect if rhIL-11 is safe in individuals with Von Willebrand Disease
Heavy Menstrual Bleeding Progestin Treatment in Bleeding Disorders Study
Heavy Menstrual BleedingBleeding Disorder1 moreThe goal of this multicenter prospective observational study and registry of U.S. adolescents and young adults with heritable bleeding disorders is to determine the bleeding outcomes, satisfaction, hemostatic parameter changes, and patient reported quality of life after 6 months of use of either of two commonly used hormonal treatments for menstrual suppression - levonorgestrel intrauterine device (LNG-IUD) and norethindrone acetate (NETA). Under this application we will compare the two treatments and compare outcomes after LNG-IUD treatment results to a control group without a bleeding disorder, with the goal of determining the benefits and expected outcomes of these treatment options for this population.
Study of Voncento® in Subjects With Von Willebrand Disease
Von Willebrand DiseaseThis is a multi-centre, open-label, single-arm, phase 4, post-marketing study to further investigate the efficacy and safety of Voncento in subjects with Von Willebrand Disease (VWD) in whom treatment with a Von Willebrand Factor (VWF) product is required as on-demand therapy, for prophylactic therapy, or during surgery. Subjects will be treated with Voncento either as an on-demand regimen (eg, to treat a non-surgical spontaneous or traumatic bleeding event) or prevention regimen (eg, to prevent an anticipated bleeding event) at a dose prescribed by the Investigator in accordance with the Voncento Summary of Product Characteristics (SmPC), or with a prophylaxis regimen (regular treatment with Voncento at a frequency of 1-3 times per week). Voncento will also be given to prevent and treat any surgical bleeding events.
Study of Biostate® in Children With Von Willebrand Disease
Von Willebrand DiseaseThis is an open-label study to investigate the pharmacokinetics (PK), efficacy, and safety of a von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, in children with Von Willebrand disease (VWD) in whom treatment with a VWF product is required for prophylactic therapy, haemostatic control during surgery, or control of a non-surgical, spontaneous, or traumatic bleeding event.
Efficacy and Safety of IL-11 in DDAVP Unresponsive
Hemophilia AVon Willebrand DiseaseThe purpose of this study is to determine the biologic efficacy and safety of rhIL-11 when given subcutaneously in adults with moderate or mild hemophilia A or Von Willebrand disease unresponsive to DDAVP. Biologic efficacy will be measured by the number and percent increase of VWD coagulation tests (FVIII:C, VWF: Ag, VWF: RCo, closure time, APTT, and VWF multimers) to the normal range, or at least to 1.5-3 time baseline, following dosing of rhIL-11 when given daily for 4 days, and boosted by DDAVP infusion on day 4, in those in whom DDAVP is not contraindicated. Safety will be measured by the frequency of adverse events, including fever, headache, fatigue, myalgias, arthralgias, fluid retention, or edema.
ARC1779 Injection in Patients With Von Willebrand Factor-Related Platelet Function Disorders
PurpuraThrombotic Thrombocytopenic1 moreTo evaluate the overall safety and tolerability of ARC1779, a therapeutic oligonucleotide ("aptamer") in patients with three types of von Willebrand Factor related platelet disorders.
Phase I Study of Human Von Willebrand Factor for Von Willebrand's Disease
Von Willebrand's DiseaseOBJECTIVES: I. Evaluate the effect of a new von Willebrand factor concentrate on bleeding time, in vivo recovery, and circulating half-life of the infused factor in patients with von Willebrand's disease. II. Assess the safety of von Willebrand factor in these patients.
The Lived Experience of People With Von Willebrand Disease
Von Willebrand Diseasesvon Willebrand disease (vWD) is reported to be the most common bleeding disorder, with prevalence estimated at 1% of the general population. Despite this, little is known about its natural history, or of the impact it has on affected individuals and their families. The Haemnet vWD360 programme is a mixed-methods, natural history study designed to gain a greater understanding of vWD and its impact on individuals and their families. It comprises both qualitative and quantitative approaches and is designed to include the perspectives of individuals with a diagnosis of any subtype of vWD. The vWD360 study includes three components: Quantitative cross-sectional survey Qualitative one-to-one interviews with affected individuals 30-day bleed diary.
Recombinant Von Willebrand Factor in Subjects With Severe Von Willebrand Disease Undergoing Surgery...
Von Willebrand DiseaseThe purpose of the study is to assess the efficacy and safety of recombinant von Willebrand factor (rVWF) with or without ADVATE in major and minor elective surgical procedures in adult patients with hereditary severe von Willebrand disease (VWD).