Active clinical trials for "Hemophilia A"

This trial is conducted globally. The aim of this trial is evaluating the pharmacokinetics (the exposure of the trial drug in the body) of NovoEight® (turoctocog alfa) in relation to BMI (body mass index) in subjects with haemophilia A.

This is a single dose, open-label, randomized, crossover study in subjects with severe Hemophilia A to compare the bioavailability of 2 different strengths of Kogenate FS (BAY 14-2222).

Haemophilia is often associated with chronic pain, functional restrictions and negative consequences regarding the individual physical performance, especially on basic motoric skills, activities of daily life and quality of life. Caused by high benefits, physical activity is a fixed component and recommendation in the guidelines for the management of haemophilia. Several studies showed that goal-directed physical activity is feasible for patients with haemophilia (PwH) without any complications or bleeding episodes. Nevertheless, there are difficulties in the implementation of supervised sports therapy for PwH regarding local distributions and uncertainties concerning correct exercise execution on patients´ side and in the monitoring and adequate training regulation from long distances on the therapeutic side. Aim of this project is a patient-related and participative development and pre-evaluation of a mobile application to generate an online-guided, individualized home training for PwH, followed by pre-evaluation in terms of a twelve weeks interventional phase. The intention of this project is to show, that a mobile device is an appropriate way to increase physical activity of PwH. Due to the development of an innovative trainings application, adapted to the needs of PwH, a larger number of patients should be reached and supported later on in participating on supervised physical exercise to improve the individual physical performance and therefore the quality of life. Additionally, with an individualized exercise program also adapted to the specific interests, needs and physical conditions, PwH at any age can be addressed. Furthermore, in cooperation with the patient, the medical staff will get systematic information of patients´ exercise activities to generate optimal factor and overall treatment patterns. In addition, on the basis of the mobile device PwH will be able to comprehend their individual exercise performance and to get an overview of the individual trainings progress and improvements of physical performance with the purpose to enhance the processes of self-motivation. Detailed instructions and information of exercise execution, need of exercise and physical adaptions to be achieved, will minimize complications and support patients´ self-confident for participating on exercise.

Background. The recurrence of hemarthrosis in patients with congenital coagulopathies favors the development of a progressive, degenerative intra-articular lesion (hemophilic arthropathy) that mainly affects the knees, ankles and elbows. Disability and functional limitations are characteristic sequelae of these patients from an early age. Objective. To assess the joint status, the perception of quality of life, the performance of roles and the performance of daily life activities, in patients with hemophilic arthropathy. Study design. Observational, multicenter study. Method. 70 patients with hemophilia A and B will be included in this study. Patients will be recruited from 4 centers in different regions of Spain. The dependent variables will be: pain (measured with the Visual Analogue Scale), joint status (Hemophilia Joint Health Score), perception of quality of life (SF-36 questionnaire), list of roles, performance of daily life activities (Barthel scale), performing the instrumental activities of daily life (Lawton and Brody scale). The evolution of the variables will be analyzed using parametric (t-student or ANOVA) or non-parametric (Wilcoxon or Friedman) tests. A correlation analysis will be carried out between the dependent and independent variables, as well as a linear regression analysis between the observed determining variables. Expected results. The aim is to observe the relationship of pain and joint status in patients with hemophilic arthropathy, with their perception of quality of life, the development of the roles they play and the performance of basic and instrumental activities of daily living.

The primary objective of this clinical research study is to establish the bioequivalence of 2 treatments, rFIX and rFIX-R, when given as a 10-minute intravenous bolus infusion.

To determine if platelet dysfunction and/or pharmacologic drug interaction occurs in patients taking both AZT and ibuprofen, which might account for enhanced bleeding tendency.

Physical activity is lower in patients with haemophilia than in their healthy peers. Nevertheless, exercize is recommended for those patients and supported by evidence. Until 2013, scientific and medical evidence to encourage physical activity for patients with haemophilia were listed to increase their locomotor function, their metabolic status, their fitness level and their well-being. In 2013, an original publication by Groen et al. suggested that physical activity could also interact with the specific and biological disturbance of the disease. We propose to consider that regular endurance exercize prescription should be encouraged in patients with haemophilia not only for a healthier lifestyle but also because it could positively alter the specific biological blood disturbance seen in haemophilia. Nevertheless, before planning a well powered intervention trial we need to determine the acceptability of regular exercize and the expected value of factor Factor VIII, von Willebrand factor and von Willebrand propeptide after an endurance training program.

To quantify the seroprevalence of antibodies to AAV5, AAV6, and AAV8 and the seroconversion rate over varying follow-up intervals in subjects with hemophilia A

The objective of the trial is to demonstrate that 2-3 times per week prophylaxis therapy with BAY81-8973 is superior to on-demand therapy with BAY81-8973 in patients with severe Hemophilia A. The hypothesis is that prophylaxis will result in fewer bleeds than on-demand treatment.

This trial is conducted in Europe. The aim of this trial is to demonstrate bio-equivalence between the marketed activated recombinant human factor VII (NovoSeven®) (CP-rFVIIa) and a new formulation stable at 25°C (VII25).