Global Epidemiologic Study of Preexisting Immunity to AAV in Adults With Severe Hemophilia
Hemophilia AHemophilia BAssess the seroprevalence of neutralizing antibodies (NAb) to AAV in adults with severe hemophilia A (coagulation factor VIII [FVIII] <1%) or moderately severe to severe hemophilia B (coagulation factor IX [FIX] ≤2%).
A Study Following People With Haemophilia A and B, With or Without Inhibitors, When on Usual Treatment...
Haemophilia AHaemophilia A With Inhibitors2 moreThis study will collect data on bleeds and data related to quality of life in people with severe congenital (a disease existing from birth) haemophilia A and B, with or without inhibitors. The aim for the study is to look at the number of bleeds when on usual treatment for haemophilia. Participants will be asked to keep an electronic diary to track the number of bleeds and the treatment of their bleeds. Participants will be asked to wear an activity tracker on their wrist to capture their level of activity every day for up to 12 weeks. While taking part in this study, participants will keep getting their usual treatment as given to them by their doctor. All study visits at the clinic are done in the same way as the participants are used to. In the time between the participants' visits to the clinic, the study staff at the clinic may call or email the participant. The study will last for about 2½ years.
Treatment Patterns And Outcomes In Patients Treated With Benefix Or Refacto/Refacto AF - A Swedish...
Hemophilia AHemophilia BThe overall aim of the study is to describe demographic and clinical characteristics, treatment patterns and outcomes, in the populations of hemophilia patients treated with BeneFIX and ReFacto/ReFacto AF in Sweden
Project to Update the Study of Congenital Haemophilia in Spain
Congenital Bleeding DisorderHaemophilia A1 moreThis study is conducted in Europe. The aim of the study is to update the knowledge about the prevalence and severity of congenital haemophilia (A and B) in Spain.
Impact of Pain on Functional Impairment and Quality of Life in Adults With Hemophilia
Congenital Bleeding DisorderHaemophilia A3 moreThis study is conducted in the United States of America (USA). The aim of the study is to assess the impact of pain on functional impairment and quality of life in adult persons with hemophilia (PWH) with and without inhibitors with joint bleeding.
Study Evaluating Liver Transplantation in Haemophilia Patients in Spain
HemophiliaHemophilia BThis study will analyze survival and outcome of patients with severe haemophilia who undergo liver transplantation in Spain
To Quantify the Range of Main Psychosocial Factors Affecting Patients and Caregivers in Their Daily...
Congenital Bleeding DisorderHaemophilia A4 moreThis survey is conducted in South America. The purpose is to identify the key psychosocial issues affecting patients with haemophilia.
Prospective, Non-interventional Study to Evaluate Immunogenicity of AryoSeven
Hemophilia A With InhibitorHemophilia B With Inhibitor2 moreThis study evaluates the immunogenicity of the biosimilar rFVIIa (AryoSeven) in subjects receiving AryoSeven in real-life clinical practice.
AAV Gene Therapy Screening/Observational Protocol (ECLIPSE)
Hemophilia BBlood Coagulation Disorders3 moreFreeline is developing adeno-associated virus (AAV) vector based gene therapies for a number of diseases and is actively advancing a programme in Haemophilia B (HB). This study aims to collect prospective data to characterise bleeding events and Factor IX (FIX) concentrate consumption in HB patients that can be used as baseline for participants who elect to participate in a subsequent Freeline gene therapy study. The study will also screen participants for antibodies to a novel AAV vector to assess their suitability for inclusion in a Freeline gene therapy treatment study.
Gait Evaluation in Haemophiliac Patients
Hemophilia AHemophilia BJoint damage secondary to recurrent haemarthroses and chronic synovitis represents the commonest clinical manifestation of haemophilia. Incapacitating pain, loss of joint stability and mobility, axial deviation, deterioration and decreased function are the most frequent complains in patients with severe haemophilic arthropathy In this context, the purpose of our study is to examine and analyse specific changes in gait in patients suffering from haemophilic arthropathy and determine the impact on health-related quality of life in terms of conceptual components of pain and stiffness, psychosocial stress, disability, and activity limitations as recommended by the International Classification of Function (ICF) recently published by the World Health Organization.