View clinical trials related to Hemophilia A.
Filter by:This study will collect information on side effects and how well Refixia/REBINYN works during long-term treatment (prophylaxis) in males with haemophilia B. While taking part in this study, participants will receive the same treatment as given to them by their study doctor. All visits at the clinic are done in the same way as the participants are used to. During visits at the clinic, participants might be asked for some relevant tests if considered useful by their study doctor. During the visits, the participants study doctor might ask if the participants had any side effects since their last study visit. The participants will be asked to note down the number of bleeds and the treatment of their bleeds as well as their regular prophylaxis. During the visits to the clinic, the participants will be asked to answer some questionnaires about their quality of life and their ability to be physically active. The participant's participation in the study will last for 4-9 years, depending on when they join the study. Participants are free to leave the study at any time and for any reason. This will not affect their current and future medical care.
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.
SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with severe hemophilia A and no measurable inhibitor against FVIII.
The purpose of this prospective study is to determine the efficacy and safety of Rituximab plus Bortezomib in patients with newly diagnosed acquired hemophilia A.
International, post-authorisation non-interventional study to evaluate real-life effectiveness, safety and utilisation patterns of Octapharma's FVIII concentrates Nuwiq, Octanate, and Wilate in previously untreated and minimally treated severe haemophilia A patients in routine clinical practice.
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.
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and families who screen positive, and 3) evaluate the Early Check program. The Early Check screening will lead to earlier identification of newborns with rare health conditions in addition to providing important data on the implementation of this model program. Early diagnosis may result in health and development benefits for the newborns. Infants who have newborn screening in North Carolina will be eligible to participate, equating to over 120,000 eligible infants a year. Over 95% of participants are expected to screen negative. Newborns who screen positive and their parents are invited to additional research activities and services. Parents can enroll eligible newborns on the Early Check electronic Research Portal. Screening tests are conducted on residual blood from existing newborn screening dried blood spots. Confirmatory testing is provided free-of-charge for infants who screen positive, and carrier testing is provided to mothers of infants with fragile X. Affected newborns have a physical and developmental evaluation. Their parents have genetic counseling and are invited to participate in surveys and interviews. Ongoing evaluation of the program includes additional parent interviews.
Severe haemophilia B (HB) is a bleeding disorder where a protein made by the body to help make blood clot is either partly or completely missing. This protein is called a clotting factor; with severe HB, levels of clotting Factor IX (nine; FIX) are very low and affected individuals can suffer life threatening bleeding episodes. HB mainly affects boys and men (approximately one in every 30,000 males). Current treatment for HB involves intravenous infusions of FIX as regular treatment (prophylaxis) or 'on demand' treatment. On demand treatment is highly effective at stopping bleeding but cannot fully reverse long-term damage that follows after a bleed. Regular treatment can prevent bleeding; however it is invasive for patients and also expensive. This clinical study aims to investigate the long-term safety and durability of FIX activity in participants who have been dosed with a new gene therapy product (FLT180a) in earlier clinical studies. Following administration, FLT180a results in production of FIX in the participants' liver cells which is then released into the blood stream. The aim is to have the participants' own body produce levels of FIX that allow for clotting to occur as normal as would be seen in a non-HB individual. This would remove the need for prophylaxis or on demand treatment following just a single administration of FLT180a. Up to 50 participants who have already been administered with FLT180a in the EU and US will take part in this study. Participants will be followed up in this trial until they have reached 15 years after being dosed. Participants will undergo procedures including physical examinations, join assessments, blood tests and liver ultrasounds. Participants will also need to complete a diary to document occurrence of bleeding episodes and record the amount of Factor IX concentrate they receive. Patient reports outcomes including measures of Quality of Life, disability and physical activity will also be collected.
There are large inter-individual differences in the bleeding pattern of patients with moderate or mild hemophilia. The major determinant of bleeding phenotype is the level of coagulant factor VIII or IX. In hemophilia A, studies addressing the association between factor VIII level and the clinical bleeding pattern yield conflicting results. In hemophilia B such studies have not yet been performed. The primary aim of this project is to analyze the association between factor VIII and factor IX levels and the bleeding phenotype. The secondary aim is to analyze potential differences in phenotype between hemophilia A and B. The project is a multicentre observational cohort study. We will include 500 patients with moderate or mild hemophilia A (FVIII 0.02-0.35 IU/mL) and 500 patients with moderate or mild hemophilia B (FIX 0.02-0.35 IU/mL) who are 12 to 55 years old. The main cohort study consists of clinical data collection, one blood sample and an online questionnaire for patients. Data will be collected on the nature and duration of all bleeding episodes, disease and treatment characteristics, physical activity level and musculoskeletal status. One blood withdrawal will be performed for centralized laboratory assays for FVIII or FIX levels (both one-stage and chromogenic assays) and genetic analysis for the most prevalent prothrombotic mutations. The online questionnaire for patients focuses on bleeds experienced in the past. A subset of 200 patients aged 24 years or older (100 with moderate or mild hemophilia A and 100 with moderate or mild hemophilia B) will be investigated in more detail by longitudinal data collection including analysis of physical joint status, MRI imaging of joints and biomarkers for joint damage. This longitudinal observation will consist of two time points that lie two years apart, allowing us to identify any changes that occur over the observed time period with respect to joint status.