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Showing 1-20 of 40 trials
NCT06706206
The investigators will use state-of-the-art imaging to look at heart disease in people with haemophilia. Haemophilia is an inherited disorder in which blood does not clot properly because of lack of a key 'glue' blood component (chemicals known as factor VIII or IX). People with haemophilia are 40% less likely to die of heart disease, but it is not known exactly why this is. Understanding heart disease in people with haemophilia is important because better treatments for haemophilia mean that these patients are now living longer, but doctors still don't know if the risk for heart disease in these patients as they age is the same as that for the general population. If these processes are better understand (perhaps less blood clotting is actually protecting the heart from blockage-causing clots), scientists might be able to reduce the risk of heart attacks for everybody. The UK's first photon-counting detector cardiac CT scanner generates detailed images of the heart and its blood vessels by counting individual X-ray photons. Together with artificial intelligence tools, it is possible to extract a lot of information from these images. As people age, fat is deposited in the vessels which supply blood to the heart which forms plaques. Plaques cause narrowing of the vessels, reducing blood flow to the heart, and can also burst (rupture), leading to a blood clot and heart attack. The new CT scan will show the type and amount of plaques, and quantify the risk of plaque rupture, in people with haemophilia; and the investigators will compare this to people without haemophilia. Understanding the role of factor VIII/IX in heart attacks will improve management of heart disease in people with haemophilia, and may also lead to new prevention and treatment strategies that benefit heart health for everyone.
NCT04590950
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.
NCT02137850
This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of turoctocog alfa pegol (N8-GP) in previously untreated patients (PUPs) with haemophilia A.
NCT02141074
This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of nonacog beta pegol (N9-GP) in previously untreated patients with Haemophilia B.
NCT06752850
The rationale for conducting this open-label phase 4 study is to assess whether once-weekly prophylaxis with efanesoctocog alfa (50 IU/kg) improves the disease course of existing synovial hypertrophy and prevents the risk of joint bleeds in patients with moderate or severe haemophilia A. The use of imaging assessments will allow for objective detection and monitoring of synovial hypertrophy, and thus expand on the previous findings demonstrating positive effects of once-weekly prophylaxis with efanesoctocog alfa (50 IU/kg) on joint health.
NCT05681845
Novo Nordisk is developing the study medicine Mim8 for the treatment of haemophilia A. The study aims to show similar levels of Mim8 in blood when using a new pen injector, called DV3407-C1 pen injector, and when using a syringe and cartridge. The new pen injector is intended to facilitate the administration of Mim8 for patients with haemophilia A. The participants will get Mim8 as injection under the skin (subcutaneously) of the belly using the DV3407-C1 pen injector and a needle (hereinafter referred to as pen injector) or using a needle and syringe from a cartridge (hereafter called syringe and cartridge). The participants will receive one injection with Mim8, either with the DV3407-C1 pen injector or with a syringe and cartridge. The study participation will last up to 20 weeks. Only healthy men can take part in the study.
NCT06285071
The purpose of the study is to investigate the safety and effectiveness of Alhemo® in all participants under real-world clinical practice in Japan. Participants will get Alhemo® as prescribed by the study doctor. The study will last for about 2 years.
NCT06831734
The purpose of the study is to investigate the safety and effectiveness of Alhemo in participants under real-world clinical practice in Japan. Total duration of this study is about 6 years. Participants enrolment will be completed in the first 4 years. The observation period of this study will last for about 2 years for each enrolled participant.
NCT03619863
This is a real-world study of the safety of the treatments used for people with hemophilia. The study will follow people with hemophilia A or B from across the country for about 4 years as they receive treatment. The hemophilia treatment center (HTC) physician and participant will decide on the FDA-approved treatment to be used which may include non-factor products, bypassing agents, or clotting factor replacement products. The goal of this research is to study the use of hemophilia treatment products and their outcomes.
NCT04675541
Record real life data of patients with Hemophilia A and treated with Afstyla® to assess the effectiveness and the safety of the treatment used as prophylaxis, prevention of bleeding (e.g. surgery) or on-demand treatment during 3 years after patient inclusion
NCT04165135
This multicenter, non-interventional, prospective study will collect information about activity status, bleeds, health-related quality of life (HRQoL), health status, and safety in participants with moderate or severe haemophilia A without factor VIII (FVIII) inhibitors, who are being treated in accordance with normal clinical practice.
NCT03276130
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.
NCT04584892
This is a study on Italian patients with haemophilia A in prophylaxis treatment with Turoctocog alfa under routine clinical conditions. The purpose of the study is to investigate the therapeutic scheme in a population of patients treated with Turoctocog alfa and to investigate the participation in recreational activities, the level of physical activity, and quality of life. Participants will get NovoEight® (Turoctocog alfa) as prescribed to them by the study doctor. The study will last for about 12 months. Participants will be requested to fill in the Questionnaires investigating the participation in recreational activities, the level of physical activity, and quality of life.
NCT03075670
This trial is conducted in Europe and the United States of America. The aim of this trial is to compare the pharmacokinetics (the exposure of the trial drug in the body) of nonacog beta pegol (N9-GP) and ALPROLIX® in patients with haemophilia B.
NCT02229331
Haemophilia is a rare X chromosome-linked coagulation disorder resulting from a congenital deficiency or absence of circulating factor VIII (Haemophilia A) or factor IX (Haemophilia B).As a consequence, patients with haemophilia are unable to generate adequate thrombin resulting in abnormal bleeding. Approximately 80-90% of bleeding episodes occur in the musculoskeletal system, especially in the large synovial joints and muscles. Repeated haemarthrosis induce joint cartilage damage and irreversible degenerative joint disease. Regular intravenous administration of coagulation factor concentrates starting after the first joint bleed and/or before the age of 2 ('primary' prophylaxis) is now the evidence-based, first-choice treatment in children with severe haemophilia. This primary prophylaxis has radically decreased the incidence of arthropathy in patients with haemophilia. Despite the positive effect of primary prophylaxis on arthropathy at several joints, the ankle joint seems to be an exception to the rule, as patients with haemophilia treated with primary prophylaxis still experience ankle arthropathy. As such, the ankle now is the main affected joint in patients with haemophilia under the age of 20. This makes the scientific community facing a new challenge. Determining aetiologic/contributing factors associated to the ankle arthropathy pathophysiological cascade in children with haemophilia (CwH) is therefore a primary objective in the haemophilic research community nowadays. Loading of the ankle is crucial as the ankle plantar flexors provide the main propulsive power during gait, the tibiotalar joint caries a complex distribution of joint stresses as a consequence of talar morphology and kinematics and, from a biomechanical and biochemical viewpoint, differs significantly from other major lower limb joints. Previous biomechanical studies focused on kinematic and strength measures, however, they omitted to incorporate fundamental measures of joint loading (joint kinetics) and joint structural integrity (JSI, assessment of soft tissue and osteochondral integrity through MRI). This makes it impossible to draw firm conclusions on biomechanical contributing factors. Furthermore, few, low-quality studies focused on conservative treatment strategies (e.g. strength training, mobilisation, proprioceptive training) in patients with ankle arthropathy. In a minority of the cases, important adverse effects (joint bleeding during strength and proprioception training) have been reported. Because of the low quality of the intervention studies and the lack of biomechanical studies focusing on joint loading and joint integrity, it is impossible to pinpoint the aetiology of these side effects. One assumption might be that side effects originate from inappropriate conservative approaches as a consequence of lacking knowledge on joint loading and joint integrity. An innovative approach within the domain of ankle arthropathy in CwH is therefore to study foot and lower limb kinetics during gait. The quantification of foot joints kinetics encompasses considerable challenges and, until recently, only simplified single-segment foot models have been used. Those models typically underestimate the mechanical contribution of the different foot joints and, of equal importance, overestimate the kinetic contribution of the tibiotalar joint. To overcome the above mentioned shortcomings, a valid 3D Multisegment Foot Kinetic Model (3DMFKM) should be developed and introduced within the population of CwH. This is important as it allows to quantify increased loading at the tibiotalar joint (or other joints) that may help to explain this ankle arthropathy in these patients. This will have been missed by previous research due to the consideration of the foot as one entire segment. Providing a classification system based on these kinetic data would, in a second stage, be a pertinent and valuable approach as this provides a rationale for designing randomized controlled trials. In this perspective, it is also recommended to assess the relationship with other biomechanical and anatomical determinants, especially if one aims at developing optimal management and rehabilitation strategies.
NCT03301376
Severe haemophilia and subsequent arthropathy result in joint pain, disability and impaired health-related quality of life. Improvements in haemophilia care over the last decades might lead to expectation of a near-normal quality of life for adults with haemophilia. However, little is known about the impact of haemophilia and its treatment on social functioning. A study based on a national US cohort of 141 young men (18-34 y.o.) showed that, compared to the general US population, this cohort experienced social burdens such as unemployment and lower high-school graduation rates. Another study conducted in the framework of the HERO (Haemophilia Experiences, Results and Opportunities) international initiative showed that a majority of the 230 young adults aged 18-30 years (including 16 French participants) were employed at least part-time (62%) and most of them reported that haemophilia has had a negative impact on employment (76%) which was reported as moderate or very large by 47% of the participants. The replicability of these results could be limited by the specific socio-economic national context. In France, a survey was conducted in 2008 to assess quality of life and its determinants in children, adolescents and adults with moderate and severe haemophilia. Descriptive results on the socio-economic characteristics of the included adults showed that 51.3% of them had access to higher education (which was higher than the observed rate in the French general population in 2005, i.e. 32.5%), and that 52.1% were employed among whom 44.3% had an at-risk activity. In spite of these results which were presented in a descriptive report by the InVS (Institut de veille sanitaire - French Institute for Public Health Surveillance), some limitations could be discussed:
NCT03179748
The trial is conducted in North America. The aim of the trial is to assess the safety of turoctocog alfa under conditions of routine clinical care in patients with haemophilia A in Mexico
NCT04524481
The aim of this study is to evaluate the prevalence of osteoporosis in adult patients with haemophilia.
NCT03914209
Background. Hemophiliac arthropathy is the main long-term physical damage in patients with hemophilia, causing disability and functional limitations. Objective. Assessment of the efficacy of a prophylactic treatment with EHL products for improving the musculoskeletal health of adult patients with hemophilia. Study Design. Multicenter observational clinical study. Method. 40 patients with hemophilia A will be included in this study. Patients will be recruited from 5 centers located in various regions of Spain (Community of Madrid, Galicia, Community of Valenciana, Málaga and Vizcaya). The dependent variables will be: bleeding frequency (self-report of bleeding), changes in the dosage of factor VIII with EHL products (rFVIIIFc), pain (measured with the visual analog scale and a pressure Algometer), degree of kinesiophobia (Tampa Scale of Kinesiophobia), degree of adherence to treatment (Veritas-Pro scale), joint health (using the Hemophilia Joint Health Score), muscle strength (using a dynamometer) and functionality (using the 6-Minute Walking Test). Three evaluations will be carried out: baseline, at 6 months, and at the end of the study period, at 12 months. The evolution of quantitative variables shall be analyzed by parametric tests (t-student test) or non-parametric tests (Wilcoxon test). Pearson's correlation coefficient shall be used to obtain the correlation between the dependent and independent variables. By means of a linear regression analysis we will record the percentage influence of the clinical variables relative to the dependent variables measured in this study Expected results. The aim is to establish the effectiveness of the prophylactic treatment in the improvement of the state health, joint pain, muscle strength and functionality in patients with hemophilia.
NCT01811875
Primary objective: To assess post-marketing immunogenicity of Optivate® by monitoring plasma inhibitor levels for at least 100 Exposure Days (EDs) for each subject. Secondary objectives: To assess efficacy and tolerability by monitoring FVIII recovery and adverse events