View clinical trials related to Hemophilia A.
Filter by:Severe hemophilia is a rare and chronic disease characterized by spontaneous bleedings from early childhood, which may lead to various complications especially in joints. The diagnosis of this disease, but also its long term care have an impact on the relatives of the affected persons, including the siblings who bear daily the cognitive, emotional and social impacts of the disease. Studies conducted in the framework of pediatric chronic diseases showed that siblings of affected children presented a higher prevalence of physical and psychological troubles (emotional distress, behavioral disorders, etc.) than siblings who were not concerned by a disease. Few studies have been conducted in the framework of severe hemophilia, and to our knowledge, no study addresses this issue in France.
Severe haemophilia A and B (SHA, SHB) are X - linked inherited bleeding disorders, characterised by factor VIII and IX levels of <1 IU/dL respectively. The mainstay of treatment in SHA and SHB is replacement therapy with intravenous infusions of factor VIII and IX. However, there is significant variability in the bleeding phenotype within severe haemophiliacs with some presenting with minimal bleeding episodes even on less intensive treatment regimens. A significant contributor to inter-individual variability in the bleeding phenotype is the coagulation phenotype, but there are no established assays in routine clinical practice that can be used to quantify this. This study aims to study novel assays and characterise the observed phenotypic heterogeneity.
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.
The puropse of this non-interventional register and survey study is to identify the patterns of prescribed pain, anti-depressive and anti-anxiety medication and management of pain, depression and anxiety for people with haemophilia. The study will be conducted in the Nordic countries (Sweden, Norway, Denmark, Finland) and the aim is to cover the entire haemophilia population in the register part of the study.
The purpose of this feasibility study is to find out if two clotting factor products, Eloctate [hemophilia A] and Alprolix [hemophilia B], can reduce the amount of menstrual bleeding in female hemophilia A and B carriers (14 years of age or older) who have severe or heavy bleeding. These products are FDA-approved for use in males with hemophilia A and B to prevent and treat bleeding. They are not approved specifically to reduce menstrual bleeding, but may be useful for this purpose. Both products have an "extended half life" which means they circulate in the body longer than other FVIII or FIX products. The study team will gather additional information about the safety of these drugs and how well they work. The results of this feasibility study will provide information for an upcoming larger study.
This is an investigator-initiated, multinational, retrospective, non-interventional pilot study conducted in five haemophilia treatment centres from different geographical regions that maintain a local (Algeria, Malaysia) or national (India, Iran, South Africa) haemophilia registry. Data from a randomly selected sample of patients from national or local registries are anonymously collated and analysed.The aims are to determine the feasibility of combining data from national and local registries in countries with developing healthcare systems and to assess how existing registries implemented current recommendations for data collection in terms of available fields and their completion.
Study Design A prospective observational, cross-sectional epidemiological study in U.S. site-based clinical practice settings. 30 sites will enroll approximately 300 patients Participating patients - or their caregiver in the case of patients under the age of 18 - will be consented to participate. Physicians complete a retrospective chart review on each enrolled patient. Patients will complete a one-time study questionnaire.
Ultrasound represents a promising technique for the assessment of joint health in persons with haemophilia (PWH) by non-imaging specialists. The Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) programme has been developed with the aim of integrating joint ultrasound in the routine assessment of PWH through the use of a simplified scoring system. The inter-operator reliability of the technique among European haemophilia treaters has been validated and described elsewhere. Further work is needed to assess the real-world impact of ultrasound on disease management and treatment decision-making.
This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor VIII into patients with hemophilia A, to evaluate the safety and efficacy of infusion of lentiviral gene modified autologous stem cells in patients.
The primary purpose was to assess the safety and tolerability of a single intravenous (IV) administration of BIVV001 in adult previously treated patients (PTPs) with severe hemophilia A.