Active clinical trials for "Hemophilia A"

This NIS aims to assess the patient-reported outcomes (PROs) in enrolled subjects

The KAPPa project has the aim to create an international database in which information about clinical features, therapeutic management, burden of illness and costs of severe and moderate haemophilia A patients from different countries and sites is collected. The aim of this project is to analyse the influence of such different characteristics on medical, psychosocial and economic outcomes in patients over the long-term.

To provide emergency access to OBI-1 for patients with Acquired Hemophilia and a serious bleeding episode who do not have access to an active clinical study site associated with the OBI-1-301 study.

The compassionate use programme will give participants concizumab for free, even though it is not yet approved by health authorities. This is because participants need this medicine to treat their haemophilia properly. The programme will check that participants are safe and that the medicine works for them. The programme may last for years. Participants will take one injection under their skin every day. Participants will have 4-5 visits with the study doctor for the first half year. After that they will have 1 visit every half year. At all clinic visits participants will have blood samples taken. Participants will fill in a diary between the visits. A patient is considered to have completed the programme when any of the following criteria occurred first: 1) when the patient is included in a clinical trial with concizumab or 2) up to 6 months after concizumab is commercially available in the patient's country and approved for the patient (The time span of 6 months should provide ample time for the patient to obtain concizumab commercially) or 3) the sponsor decides to discontinue concizumab clinical development for the patient's population.

This open-label, multicenter expanded access program (EAP) is designed to provide emicizumab to eligible participants with hemophilia A with factor VIII (FVIII) inhibitors before it is commercially available in the United States for the indication of hemophilia A with FVIII inhibitors. Discontinuation may occur earlier if participant or physician decides to discontinue treatment or the sponsor discontinues emicizumab clinical development.

This is an investigator-initiated, industry-funded, multi-centre, international study that will be carried out prospectively at hemophilia treatment centres across Canada, the Czech Republic and Australia with SickKids as the coordinating site. The study will use a central laboratory not directly affiliated with any of the participating sites. Enrollment target is 50 participants, both adult and pediatric with severe hemophilia A receiving Advate, who will each complete a 2-point and 6-point pharmacokinetic (PK) sampling. The main aim is to compare the results of a 2 sample PK using clinically practical time points and myPKFiT™ (a web-based, population PK Bayesian tool) to a 6 sample population PK to determine whether the results obtained are in good agreement.

Research project for validation of a questionnaire on compliance to treatment in patients with hemophilia. It is intended to validate the American VERITAS-PRO scale according to the international methodology validation questionnaires: reverse translation, according to intersubject patients, psychometric validation and reliability analysis with large sample of patients.

Multidisciplinary evaluation of patients with hemophilia Spain, El Salvador and Bolivia. It aims to analyze and evaluate the skeletal muscle of patients affected with hemophilia Spain, El Salvador and Bolivia; describe deficit mobility and muscular strength, depending on the degree of injury, age, presence of inhibitors and the development of physical activity; identify the relationship between the functional deficit by clinical assessment performed with the use of validated measurement scales, the assessment of the degree of injury to specific scales of hemophilia; detect biomechanical alterations of the lower limbs and their impact on gait in patients with hemophilia; and evaluate the factors that influence the perception of illness and quality of life of patients with hemophilia.

Analyze and evaluate the skeletal muscle of patients affected with hemophilia in the Region of Murcia. Describe the periarticular muscular deficit of the lower limbs, depending on the degree of injury: acute and chronic, or subacute. Identify the range of motion of joints with higher prevalence of hemophilic arthropathy. Detect the biomechanical changes of the lower limbs and their impact on gait in patients with hemophilia. Assess the factors that influence the perception of illness and quality of life of patients with hemophilia.

Psychosocial factors and adherence to treatment in patients with hemophilia. A multicenter study. Multicenter cross sectional study of patients with hemophilia and their families