View clinical trials related to Hemophilia A With Inhibitor.
Filter by:The goal of this observational study is to assess if the version A of the HemA EnzySystem, a novel portable coagulation testing platform, can be used in patients with hemophilia A treated with Factor VIII Bypassing Agent (FEIBA). The main question[s] it aims to answer are: - Can the version A of the HemA EnzySystem can record thrombin generation within a time frame of 60 min in fresh whole blood samples of patients with hemophilia A treated with FEIBA? - Are the TGA results of the version A of the HemA EnzySystem in agreement with the TGA results obtained with conventional methods in fresh non frozen plasma? Participants are asked to fill in a questionnaire regarding their general health and hemophilia treatment. Subsequently, blood will be drawn from the patients before, and at 30, 120, and 240 minutes after FEIBA administration. Whole blood is immediately tested using the Version A HemA EnzySystem, and plasma is generated for testing with the Ceveron s100 (Technoclone). Leftover samples are frozen for later additional coagulation testing.
This phase I study aims to evaluate the safety, PK/PD and immunogenicity of SS109 in hemophilia A or and B with inhibitors. Twenty -seven patients are enrolled in study, and divided into five dose cohorts, from 30μg/kg to 360μg/kg. Dose 1 cohort enrolls three patients, each other dose cohorts enroll six patients. All patients included in the study will continue to be followed up until 28 days after SS109 administration.
Randomized, double-blind, single-dose, 5 ways crossover, exploratory clinical trial evaluating four different doses of AryoSeven (eptacog alfa, activated) and NovoSeven on selected pharmacodynamic parameters in patients with hemophilia with inhibitors.
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 multi-center, open label Phase 1 study will evaluate the pharmacokinetics, pharmacodynamics, and safety of a single IV dose of MarzAA followed by ascending single SC doses of MarzAA in adult subjects with moderate or severe Hemophilia A or B, with or without an inhibitor.
A chart review study of patients with haemophilia A with inhibitors treated with rFVIIIFc (Elocta®) for immune tolerance induction.
This is a multi-center cohort study of approximately 250 previously untreated patients (PUPs) with congenital moderate to severe hemophilia A or B in a network of up to 50 US Hemophilia Treatment Centers (HTCs). Participants will be followed as they receive their first 50 exposure days (ED) to clotting factor replacement product, both prospectively and retrospectively. The data collected on evolving treatment practices will define the incidence and risk factors for inhibitor development during the high risk period of first 50 ED and improve the outcomes of this vulnerable population.
Phase 2, multi-center, open-label study designed to evaluate the PK, bioavailability, PD, efficacy and safety of a daily subcutaneous [SC] treatment regimen with MarzAA for bleeding prophylaxis in 12 adult subjects with hemophilia A or B with an inhibitor and history of frequent spontaneous bleeding episodes.
This study evaluates the immunogenicity of the biosimilar rFVIIa (AryoSeven) in subjects receiving AryoSeven in real-life clinical practice.
Several non-genetic and genetic factors that could influence the risk of inhibitor development in hemophilia A have been discussed but not fully explored. The aim of the HIGS is to identify these genetic factors.