Active clinical trials for "Hemophilia A"

The purpose of this study is to validate the newly developed Hemophilia Functional Ability Scoring Tool (Hemo-FAST), which is a fast and simple scoring in haemophilia able to assess patient-reported functional mobility.

The aims of this trials are: to collect published and unpublished individual classic pharmacokinetic data (individual patient data from independent investigators and pharmaceutical companies) to make available population pharmacokinetic models for the concentrates derived from the data collected to develop a web based application intended to use the above models to calculate pharmacokinetic parameters for individual patients, and to test the system functionality via simulation of the use of the prototype by use of faked test data.

OPTIMS is a non interventional validation study of the calculator developed by Bayer for clinician's use in the prophylactic treatment by factor VIII of patients with severe or moderate Haemophilia A with a severe clinical profile. The study takes place during a single visit, at the time of patient enrollment in the study

The goal of this pilot study is to understand the socialization of adult men with hemophilia living in the United States and their quality of life in order to improve comprehensive care for persons with congenital bleeding disorders. Studies in Europe suggest that despite medical, surgical, and biotechnology advances in care for persons with the congenital bleeding disorders hemophilia A and B, men with hemophilia have earlier work disability and health-related lower quality of life than men of the same age who do not have hemophilia in the general population. Congenital bleeding disorders are known to have medical and psychosocial impact not only in school but also in other activities, e.g. participation in sports beginning at a young age. The psychosocial impact of living with a congenital bleeding disorder has been studied and described in childhood. The support relationships in childhood include parents and primary family of origin and these supports are generally considered in pediatric comprehensive care models. Support relationships in adulthood have not been well described or studied. The role of spouse and significant others (SSO) of PWCBD in health care is of interest for the delivery of adult comprehensive care as well as to understand their contribution to the health-related quality of life of PWCBD. Additionally, this study seeks to learn of the impact of congenital bleeding disorders for the SSO. The study uses self-reported medical and social information questionnaires, health-related quality of life surveys, and confidential interview. Results of this study may guide how comprehensive care and support are provided to adult persons with congenital bleeding disorders by hemophilia treatment programs. This study focuses on PWCBD with hemophilia A or B as a model for the experience of persons with other congenital bleeding disorders.

The purpose of this study is to test the external validity of the systemic ultrasound protocol for data acquisition and interpretation, in order to diagnose soft tissue and osteochondral abnormalities in hemophilic children.

The relevance of the study will be in establishing whether Health Related Quality of Life (HRQoL) measures obtained repeatedly during hemophilia care are sensitive to significant changes in health or personal circumstances related to the transition from youth to adulthood. This may support the future use of HRQoL measures as part of routine care in order to identify important changes that are not detectible by other clinical means.

This international study will identify genetic factors that may influence the development of inhibitory antibodies in patients with hemophilia A after treatment with factor VIII. Bleeding episodes in patients with inhibitors are often more difficult to treat. Previous research indicates that genetic factors play a role in the development of inhibitors. A better understanding of the influence of genes in this treatment complication may be helpful in predicting, treating or preventing inhibitors. People in families in which one or more members have severe factor VIII deficiency and one or more have a history of an inhibitor may be eligible for this study. Participants fill out a form with questions about the person's relationship to other family members taking part in the study. Those with hemophilia provide a brief medical history, including hemophilia-related information, inhibitor history and the presence of other conditions such as hepatitis C and HIV. All participants have a blood sample taken for laboratory and research tests.

Retrospective, multicenter study in patients with hemophilia B. This study will be placed at 20 to 50 hemophilia treatment centers in North America and the EU, and is designed to estimate the frequency of Class II and III allergic reactions in association with the administration of any FIX concentrate. Following informed consent and eligibility, historical patient information including demographics, allergy history, hemophilia history, frequency and severity of allergic reaction(s), number of exposure days with products causing allergic reactions, exposure days to blood products including FIX concentrates, treatment for allergic reaction(s), outcome of event(s), any other adverse event(s) occuring within 48 hours of onset of allergic reaction(s), rechallenge and outcome, switch to another FIX product (if applicable) and outcome, current status of patient, and results of any special studies (eg, skin or RAST testing, alloantibody analyses, ect) will be collected.

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.

To determine the risk factors associated with inhibitor formation in hemophilia A and to study the mechanism of tolerance in the murine hemophilia A model.