View clinical trials related to Hemophilia A.
Filter by:IHBDH-GTHA-2020 is an open- label, non- randomized study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of GS001 in hemophilia A subjects with <1 IU/dl residual FVIII levels.
Research question: Whether there are the changes in quality of life in patients with haemophilia A after switching from SHL FVIII prophylaxis to efmoroctocog alfa prophylaxis? A 12-month prospective open-label, single-arm multicentre study. Evaluation of parameters will be carried out on the backdrop of patient treatment in the settings of routine medical practice. No medical examinations/ procedures/ treatment(s) on the top of regular medical practice are planned, except fixed time of examinations.
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.
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.
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.
This study aims to investigate the effects of an individually planned exercise program, counseling and family education on the followings in patients with hemophilia (PWH); 1. To examine the joint range of motion, muscle strength and functional status. 2. To evaluate the level of physical activity and daily living activities 3. To aim to determine the amount of change after participation.
This is a prospective, randomized control trial in which each patient will be randomly assigned to receive either extended half-life factor VIII based replacement therapy or non-FVIII based replacement therapy, which are both standard of care treatment for persons with Hemophilia A.
To evaluate the safety and tolerability of OP-724 in liver cirrhosis patients caused by HIV/HCV co-infection with hemophilia.
This Phase I/II clinical study will evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe haemophilia A and inhibitors to FVIII. Part A of the study will involve subjects who have active inhibitors to FVIII, and Part B involving subjects with a prior history of inhibitors.
This non-interventional study concerns a safety data collection based on adverse event data from a third-party registry (European Haemophilia Safety Surveillance System, EUHASS) that includes information about adverse events from patients with haemophilia A treated with turoctocog alfa pegol. There is no extra burden to the patients by participating in this registry-based data collection.