Active clinical trials for "Hemophilia A"

This investigation will utilize a systematic determination of anti-FVIII antibody specificity in PTPs (> 50 ED) with hemophilia A who have developed inhibitors in response to treatment with any FVIII product(s). A group of patients with hemophilia A, who have no evidence of current or prior FVIII inhibitor will be included for comparison. The objective of this study is to describe the patterns of antibodies and associated epitopes in the study population.

Haemophilic arthropathy is one of the major complications of severe haemophilia. In order to maintain plasma clotting factor activity levels above 1% and avoid spontaneous joint bleeds and other serious bleeding events, prophylactic factor replacement therapy is used. Because of the high cost and limited availability of clotting factor concentrates, dosing is a crucial issue for prophylaxis therapy. Several studies reported a better correlation between clinical bleeding tendency of patients with haemophilia and thrombin generation assay results compared to FVIII/FIX levels. However there is no specific data showing that thrombin generation may be a better indicator of the clinical efficacy of prophylaxis compared to the conventional FVIII measurement. The main objective of this open, multicentre, prospective study is to compare trough thrombin generation capacity and FVIII levels in severe haemophilia patients and compare these two laboratory results with: the number of spontaneous haemarthroses and other spontaneous serious bleeds occurred in the last 6 months the number of additional FVIII units used in the last 6 months This project requires no change in term of type of treatment : During the study, each patient will be treated by his usual clotting factor at the usual regimen (frequency and dosage). The clinical outcome with the usual prophylaxis regimen will be correlated to TGA results

In persons with severe haemophilia A (HA) infused factor VIII (FVIII) half-life and other pharmacokinetic parameters can vary according to determinants such as blood group, von Willebrand factor (VWF) level or age. However, FVIII pharmacokinetics (PK) has not been thoroughly studied in patients with severe HA as a function of daily physical activity. Patients with severe HA (FVIII <1%) are predisposed to prolonged bleeding following even minimal musculoskeletal injuries. Potential consequences of repeated musculoskeletal bleeding are pain, arthropathy and physical disability. The key standard of care for HA patients is prophylactic infusions of FVIII concentrates (25-50 IU kg-1 infused 2-3 x/week), depending on individual response. The level of infused FVIII decreases as a function of time according to both specific PK features of each product and biochemical/genetic characteristics of the patients or different clinical conditions. Some critical points remain still unraveled, for instance, whether or not FVIII AUC is significantly affected by physical activity/exercise, in response to increased metabolic rate or subclinical/microhaemorrhages in patients with severe HA. It is known that vigorous-intensity physical activity/exercise can transiently but significantly increase circulating levels of endogenous VWF and consequently FVIII in normal subjects and in patients with moderate or mild haemophilia A. The proposed study is a Proof of Concept one as it will be aimed at investigating the relation between daily physical activity, measured by SenseWear® armband device, as number of daily steps, and PK variability of infused rec-FVIII concentrate. This kind of investigation has never been done and it is a great interest also for the evaluation of patients' quality of life.

This is a randomized, prospective, multicenter study to examine whether or not the current recommended factor dosing strategy - i.e., dosing by actual body weight - in overweight and obese patients with Hemophilia A may deliver excessive clotting factor to achieve the desired result of bleeding prevention and cessation. This study also examines ways to prevent delivering excessive factor by using a patient's ideal body weight as a new dosing strategy compared to the current dosing strategy. The hypothesis being tested is that factor dosing based on ideal body weight will result in protective factor levels.

Commercial one and two-stage factor VIII assays may not detect some clinically significant inhibitor antibodies. The purpose of the proposed study is to standardize and validate a platelet-based factor VIII activity assay with greater sensitivity to clinically important inhibitory antibodies. Investigators will evaluate the platelet-dependent inhibitory activity vs. conventional inhibitory activity in stored patient plasmas and correlate to bleeding histories

This study aims to develop a systematic genetic screening strategy for (potential) female carriers of haemophilia by identifying as many female carriers as possible within the families of haemophilia patients regularly followed at Cliniques universitaires Saint-Luc (CUSL) and to search for differences between female carriers of haemophilia A (HA) and B (HB).

The purpose of this study is to seek to understand the patient's perspective around deciding to participate in a clinical trial for a Hemophilia therapy

This study is conducted in the United States of America (USA). Tha aim of this study is bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S).

To assess and describe real-life treatment choices for rFⅧ contained regular prophylaxis/bleeding prevention treatment in pediatric hemophilia patients in China (2007-2013)

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.