View clinical trials related to Hemophilia B.
Filter by: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.
This 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.
The Hemophilia Growth and Development Study (HGDS) nearly 25 years ago showed haemophilia and HIV impacted brain development, thinking, and behaviour in children and adolescents. The eTHINK study is designed to understand whether advances in hemophilia treatment have removed any impact of hemophilia. If there still is an impact of hemophilia, the eTHINK study will help to identify which children or adolescents are still at risk, and how to screen other children. Participants and their child will participate in a comprehensive assessment of child's brain development, thinking and behavior through completing tests and questionnaires. There is only one study visit of 1 to 1.5 hours with no follow-up required
Alprolix (rFIXFc) is a recombinant extended half-life coagulation factor product. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Alprolix in the on-demand and prophylactic treatment of haemophilia B.
The purpose of this study is to characterize the safety and describe the effectiveness of RIXUBIS in routine clinical practice.
Children with hemophilia are exposed to intravenous (IV) procedures that cause pain/anxiety and distraction methods can be employed to improve the patient experience. Three-dimensional virtual reality (VR) environments can reduce distress related to procedures. To be utilized in a clinical setting, however, these devices must address infection control concerns and fit pediatric patients. Additionally, for use in hemophilia care, environments should encourage a subject to keep their hands/arms still to facilitate procedures. Nursing orchestration of the VR environment via an iPad dashboard has the potential to increase feasibility and acceptance by patients, families and providers of the VR experience without disrupting routine clinical care.
This is an open-label, single-dose, single-arm, multi-center trial, with a screening, a treatment + post-treatment follow-up phase, and a long-term follow-up phase. The IMP AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a liver-specific promoter. The IMP is identified as AAV5-hFIXco-Padua (AMT- 061). The pharmaceutical form of AMT-061 is a solution for intravenous infusion. The administered dose of AMT-061 will be 2 x 10^13 gc/kg.
The primary objective of this study is to assess the pharmacokinetics in patients with severe hemophilia. The secondary objective is to assess the pharmacodynamics of BAY1093884 based on tissue factor pathway inhibitor activity
An online web-based cross-sectional survey in which participants will complete a distinct questionnaire for patients with hemophilia and obesity (PwHO) and/or their carers/spouses/partners, or a distinct questionnaire for healthcare professionals. Each questionnaire consists of more than 100 items including pre-coded (multiple choice) closed-ended questions, yes/no questions and rating scales. It is estimated that this one-off survey will take approximately 45-60 minutes to complete. As a cross-sectional study, there will be no treatment of patients.
Primary Objective: -To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by the frequency of bleeding episodes. Secondary Objectives: - To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by: - The frequency of spontaneous bleeding episodes. - The frequency of joint bleeding episodes. - Health-related quality of life (HRQOL) in participants >=17 years of age. - To determine the frequency of bleeding episodes during the onset period. - To determine the safety and tolerability of fitusiran.