Active clinical trials for "Hemophilia A"

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:

This study aims to examine the real-life experience and impact of using emicizumab in a cohort of patients with haemophilia and inhibitors, who were prescribed emicizumab as part of the early access to medicine schema (EAMS),those who have been in clinical trials and those now receiving emicizumab as part of routine haemophilia care. The Investigators also intend to capture the impact of emicizumab use on the lives of close family members (parents/carers/children/partners/siblings). Each participant and his family members will be deemed a study 'dyad'. This is a prospective, observational cohort qualitative research study to be conducted among patients using emicizumab in routine clinical practice. The study is designed to allow English-speaking patients and their families to tell their own life stories through narrative accounts. The narratives represent a true sharing of experiences and therefore offers insight into how these patients and families cope with haemophilia.

Hemophilia is a bleeding disorder and repeated joint bleeding leads to hemophilic arthropathy. Among patients with hemophilia, vitamin D deficiency and hemophilic arthropathy have been associated with osteoporosis in several clinical studies.There is no data on the prevalence of osteoporosis in hemophilia patients in Taiwan or Asia. To the best of our knowledge, no previous studies have reported the prevalence of sarcopenia and correlation with osteoporosis in hemophilia adult patients. This study will investigate the prevalence and corelation of sarcopenia and low BMD in patients with hemophilia. patients in Taiwan. The study will estimate the prevalence of sarcopenia and body composition in the hemophilia population and compared baseline demographic and clinical characteristics between the non-sarcopenia and sarcopenia individuals, with particular emphasis on the overlap with osteoporosis and hemophilic arthropathy.

Joint damage secondary to recurrent haemarthroses and chronic synovitis represents the commonest clinical manifestation of haemophilia. Incapacitating pain, loss of joint stability and mobility, axial deviation, deterioration and decreased function are the most frequent complains in patients with severe haemophilic arthropathy In this context, the purpose of our study is to examine and analyse specific changes in gait in patients suffering from haemophilic arthropathy and determine the impact on health-related quality of life in terms of conceptual components of pain and stiffness, psychosocial stress, disability, and activity limitations as recommended by the International Classification of Function (ICF) recently published by the World Health Organization.

This study is conducted in Europe. The aim of this prospective, observational study are to describe the different clinical practice and detailed haemostatic management of activated recombinant human factor VII (NovoSeven®) in patients with acquired haemophilia in France. The secondary aim is to assess the primary haemostasis disorder associated with the coagulation disorders, if available.

Understanding how often the bleeding events occur in the subjects who voluntarily decide to switch from prophylaxis to on-demand and in those subjects who remain on prophylaxis. Also look into the consequences of switching treatment in QoL (quality of life), development of target joints, activity level and reasons that might influence the desire to switch.

Analyze the clinical and therapeutic approach to treatment of haemophilia A and B in habitual clinical practice conditions in Spain.

To correlate the Human Leukocyte Antigen type and genetic defect with hemophilia A.

This study programme aims to examine the real-world experience and impact of gene therapy in a diverse community of people and families affected by haemophilia who have been or will be exposed to gene therapy.

The aim of this prospective, observational study is to establish a dataset on the frequency of bleeding events, as well as other characteristics of bleeding events and FVIII infusions, in patients with clinically severe hemophilia A receiving prophylactic FVIII replacement therapy as standard of care. The data collected from this study may assist in providing baseline information for comparison to the Spark's investigational hemophilia A gene therapy in future Phase 3 studies.