Active clinical trials for "Hemophilia A"

This low interventional study aims to describe if and how the haemophilia treatment management decisions are impacted by a systematic joint examination (ultrasound, functional, physical) in patients with haemophilia A in France.

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.

Severe hemophilia is a rare and chronic disease characterized by spontaneous bleedings from early childhood, which may lead to various complications especially in joints. The diagnosis of this disease, but also its long term care have an impact on the relatives of the affected persons, including the siblings who bear daily the cognitive, emotional and social impacts of the disease. Studies conducted in the framework of pediatric chronic diseases showed that siblings of affected children presented a higher prevalence of physical and psychological troubles (emotional distress, behavioral disorders, etc.) than siblings who were not concerned by a disease. Few studies have been conducted in the framework of severe hemophilia, and to our knowledge, no study addresses this issue in France.

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.

Hemophilia A (HA) is a rare X-linked bleeding disorder caused by a deficiency in factor VIII (FVIII) affecting 1/5,000 males1. Carriers of HA are females carrying the pathogenic variant responsible for the familial HA at a heterozygous status. About 30% of HA carriers have low FVIII levels and can therefore have abnormal bleeding symptoms2,3. Such as males with moderate/mild HA, bleeding can be treated or prevented with either FVIII concentrates or desmopressin4,5. This drug acts as a vasopressin type 2-receptor (V2R) agonist that causes endothelial cells to rapidly secrete von Willebrand factor (VWF) and FVIII from Weibel-Palade bodies into the bloodstream6,7. However, the mechanism of action of post-DDAVP FVIII increase remains poorly understood in hemophilia A. One advantage of DDAVP is that it increases the level of endogenous FVIII, thus avoiding the need for potentially immunogenic exogenous FVIII. It is also cheaper than FVIII concentrates. Finally, it is more widely available in pharmacies in all hospitals with emergency rooms and surgical facilities. The FVIII response profile to DDAVP in carriers appears quite similar to that seen in men with mild/moderate HA8-11. A post-DDAVP increase in the FVIII level of 2-4 fold the basal level is usually observed. This FVIII response presents an important inter-individual variation making it necessary to carry out a therapeutic test before its use for the anti-hemorrhagic treatment. The basal FVIII level logically conditions the intensity of the post-DDAVP FVIII peak. However, other factors influencing the post-DDAVP FVIII response are very likely. Unfortunately, few series describing the FVIII response to DDAVP in HA carriers have been reported to date and they included too small numbers of patients to precisely analyze the factors of variation in the post-DDAVP FVIII pharmacokinetics (PK). Candy et al did not find any difference depending on the severity of the pathogenic variants for HA or on the age11. However, this study was carried out in a cohort including only 17 patients, therefore too small for a reliable statistical analysis. The GIDEHAC study (Genetic Influence of Desmopressin Efficacy in Hemophilia A Carriers) is a French study with the following objectives: the description of the post-DDAVP FVIII PK in a large retrospective cohort of HA carriers, the research of patients-related factors influencing this FVIII PK, and the building of predictive population- and Bayesian-based models.

Background. The recurrence of hemarthrosis in patients with congenital coagulopathies favors the development of a progressive, degenerative and intra-articular lesion (hemophilic arthropathy) that mainly affects the knees, ankles and elbows. Pain is one of the main clinical manifestations of hemophilic arthropathy. As a consequence of the COVID-19 pandemic, the Government of Spain, among others, established a total confinement for two months, in order to avoid contagion of the population Objective. To assess the effect of COVID-19 confinement on bleeding frequency, pain perception, and range of motion in patients with hemophilic arthropathy. Study design. Prospective observational study. Method. 27 patients with hemophilia A and B will be included in this study. Patients will be recruited from the Spanish Hemophilia Federation (Fishemo) specialized center for hemophilia patients. The dependent variables will be: the frequency of bleeding (through a self-report), the perception of pain (measured with the visual analog scale and a pressure algometer), the joint state (with the Hemophilia Joint Health Score), and the range of joint movement (measured with a goniometer). Two evaluations will be carried out: pre-treatment (carried out in the month of February, as a periodic evaluation) and post-treatment (at the end of the period of confinement in Spain). Expected results. The aim is to observe the sequelae caused by confinement and a sedentary lifestyle in patients with hemophilic arthropathy, through changes in joint status, pain and range of motion.

The main aim of this study is to learn about changes in the lowest blood levels of Factor VIII in men and boys when upgraded from standard prophylaxis with Advate to individualized prophylaxis with Advate. No study medicines will be provided to participants in this study. The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study. Participants will need to visit the study doctor 3 times in total during the study. During these visits, study data will be collected by the study doctor.

Haemophilia A (HA) is a rare constitutional haemorrhagic disease whose drug management is based on the use of chronic lifelong replacement therapy. The occurrence of an inhibitor is a dreaded complication that impacts conventional management, consisting in using factor VIII (FVIII)-based replacement therapy, most often for prophylaxis. Although effective, these treatments can only be administered intravenously, leading to accessibility constraints and significant mental burden for patients and their relatives. Before June 15, 2021 in France, the emicizumab (HEMLIBRA®) was available only in hospital pharmacies for the prevention or reduction of bleedings. The introduction of the dual dispensing circuit in hospital or community pharmacies, left to patient's choice, is effective from this date. These changes have important organizational consequences for patients and health professionals alongside the pathway of care. Therefore, the effectiveness of this new organization requires to be evaluated with a national French study, called PASO DOBLE DEMI. The aims of this study are twofold : I. To evaluate the direct impact of the training programs provided to the new placeholders of the dispensing circuit ; the community pharmacists, II. To evaluate satisfaction of patients or their relatives regarding the emicizumab treatment whether they chose dispensing in the community pharmacy, or kept the dispensing at the hospital pharmacy. The methodology was based on the 4-level of the Kirkpatrick's evaluation model; 1) the immediate reaction of community pharmacists following the trainings (Reaction), 2) their knowledge acquisition (Learning), 3) their professional practice (Behavior) and 4 ) the patients' satisfaction related to their treatment whether dispensing in hospital or in community pharmacies (Results). The PASO DOBLE DEMI I was based on the first three levels of the evaluation model.

The purpose of this survey is to understand the following items in the actual clinical use of ADYNOVATE in patients: Unexpected adverse drug reactions Occurrence of adverse drug reactions in the actual clinical use Factors that may affect safety and efficacy Occurrence of Factor VIII inhibitor development in patients with coagulation factor VIII deficiency (hereinafter hemophilia A) Safety and efficacy for hemophilia A patients who received routine prophylactic therapy and on-demand therapy

The purpose of the study is to evaluate the effectiveness of Elocta compared to conventional factor products in the prophylactic treatment of patients with haemophilia A over a 24-month prospective period. Data will also be collected for a 12 month retrospective period.