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Active clinical trials for "Hemophilia B"

Results 231-239 of 239

Epidemiological Study on Haemophilia Care and Orthopaedic Status in Developing Countries

Congenital Bleeding DisorderHaemophilia A3 more

This study is conducted in Africa and Asia. The aim of this study is to evaluate in the participating countries the orthopaedic status and the degree of arthropathy of severe haemophilia patients in general.

Completed5 enrollment criteria

Pre-clinical Models for Mesenchymal Stem Cell Therapy in Hemophilic Arthropathy

Hemophilia AHemophilia B1 more

Hemophilia is a constitutional coagulation disorder responsible for a hemorrhagic phenotype in patients from an early age. Hemarthrosis is one of the most frequent complications in hemophiliacs and leads to the development of severe and early arthropathy, sometimes as early as childhood. To date, there is no curative treatment for these joint disorders and preventive treatments are insufficient to completely prevent joint degradation. Mesenchymal stem cells have been shown to be of therapeutic interest in the management of pathologies such as osteoarthritis and inflammatory arthritis through their anti-inflammatory, regenerative and anti-apoptotic effects. Hemophilic arthropathy is a separate condition at the border of these two diseases Our study aim to show pre-clinical interest of mesenchymal stem cell therapy in hemophilic arthropathy

Unknown status4 enrollment criteria

Dosage and PD Study of Eftrenonacog-alfa

Severe Haemophilia B

The purpose of this study is to evaluate the performance of different methods for measuring factor IX activity levels in haemophilia B patients treated with eftrenonacog-alfa and assess its pharmacodynamics (PD) in a real-life setting.

Unknown status3 enrollment criteria

Natural History Study of Factor IX Treatment and Complications

Factor IX Deficiency

This study will examine two groups of subjects with factor IX (FIX) deficiency: 1) those with a current or history of inhibitors to FIX, and; 2) groups of two or more affected brothers, with or without inhibitors. The overall goal is to characterize the study groups in terms of their medical history, their patterns of bleeding, their care, quality of life, and complications including the development of joint disease, inhibitory antibodies to FIX, use of immune tolerance induction (ITI) and outcome.

Unknown status6 enrollment criteria

Global Hemostatic Methods in Hemophilia and Von Willebrand's Disease

Hemophilia AHemophilia B1 more

Patients with hemophilia who have the same level of deficient factor(s) may express different severity of clinical presentation and bleeding tendency. Therefore a test which could determine overall hemostasis rather than simple concentration of a single deficient factor may correlate better with clinical phenotype in these patients. The investigators will therefore study the usefulness of global hemostatic methods (endogenous thrombin potential (ETP), overall hemostatic potential (OHP), fibrin clot structure) and microparticles in the prediction of severity of bleeding and estimation of response to the treatment in patients with hemophilia. Since hemophilia patients on prophylactic treatment virtually do not bleed, additional patients who are treated on demand only will be included enabling to study possible modulatory effects of different hemostatic factors (particularly prothrombotic and thrombin activatable fibrinolysis inhibitor (TAFI)) on clinical presentation. The investigators will correlate both those factors and clinical severity with global hemostatic methods. The investigators expect to prove that individual tailoring of the treatment, which may enable lowering the prophylactic dose of factor concentrate without increasing the risk of bleeding, is justified in some hemophilia patients. This approach would reduce the amount of necessary factor concentrate in certain patients and decrease the cost (which represents extensive burden for health care systems) of treatment without potential risk for the patients.

Unknown status2 enrollment criteria

UK - EHL Outcomes Registry

Hemophilia AHemophilia B

Severe haemophilia A and B (SHA, SHB) are inherited bleeding disorders affecting male patients and are characterised by low levels of circulating clotting factors VIII and IX respectively. Clinically low levels present with multiple recurrent bleeds into joints and muscle from the first couple of years of life. In addition patients may present with spontaneous and potentially fatal bleeding into any organ. The mainstay of treatment is replacement with the missing factor in the form of intravenous injections of factor VIII and IX. Clotting factors can be given to treat a bleed or can be given to prevent a bleed, and the latter is termed prophylaxis. Regular prophylaxis is the current standard of care and aims to decrease spontaneous bleeding events and resulting joint damage, and this requires patients to self-infuse factor into their veins two to four times week. Patient's compliance with prescribed regimen and recommendations has a significant influence on outcomes. Advances in biomolecular and protein engineering have extended the duration of the effect of clotting factor VIII and IX through multiple mechanisms. This extension of the duration of the effect presents the clinician and patients with opportunities to tailor the treatment to their particular needs, circumstances and body other characteristics. It has been suggested that decreasing the frequency of infusions will improve adherence and thus contribute to improved outcomes. In rare disorders, it is an accepted fact that post-marketing studies are crucial to understand the generalisability of the efficacy and safety outcomes and identify any new safety and efficacy concerns in relation to specific population group. The investigators propose the development of a registry for systematic collection of information with the dual aim of analysing the relationship between patient and treatment characteristics, and outcomes, and simultaneously identify areas for practice development that can improve the overall quality of life experienced by the haemophilia patient community.

Unknown status4 enrollment criteria

Medication Adherence and Non-adherence in Adults With Rare Disease

Cystic FibrosisHemophilia A4 more

The purpose of this study is to use the Medication Adherence Reasons Scale (MAR-Scale) to determine the extent of non-adherence to specific medications indicated to treat cystic fibrosis, hemophilia (A or B), idiopathic pulmonary fibrosis, myasthenia gravis, and sickle cell disease, and to identify the top patient-reported reasons for non-adherence. Internal reliability of the MAR-Scale will also be assessed in each condition.

Withdrawn12 enrollment criteria

Mutation p.Ile112Thr : Discrepancy Between Factor IX Level and Bleeding Phenotype

Hemophilia B

It appears that the mutation p.Ile112Thr in the factor IX gene confers a discrepancy between mild factor IX level and severe bleeding phenotype. Databases and litterature analysis are poor on this matter. The goal of this study is to compile bleeding phenotype in patients with this specific mutation to prove the clinico-biological discordance in order to improve patient care and follow-up.

Unknown status1 enrollment criteria

A Study of Factor Inhibitors in Adult Patients With Hemophilia and Von Willebrand's Disease in Upper...

HemophiliaVon Willebrand Diseases1 more

Hemophilia A and B are bleeding disorders caused by deficiency of factor VIII and IX, respectively. The deficiency of one of these coagulation factors is due to a mutation on the X chromosome. Accordingly replacement of the deficient factor is currently the main treatment for these disorders. The most disappointing complication of replacement therapy in hemophilia is the development of inhibitors. Unlike haemophilia , inhibitor development in patients with V Willebrand's Disease (VWD) is a rare complication of treatment. Studies on inhibitors whether on hemophilia or VWD are limited in our region. This study aims to To estimate the frequency of factor inhibitors in hemophilia and VWD patients in our region. To investigate modifiable risk factors associated with development of inhibitors in both diseases. To correlate the level of inhibitor with the clinical presentation of the patients. To assess influence of factor inhibitors on quality of life in patients who developed factor inhibitors in both diseases.

Unknown status4 enrollment criteria
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