View clinical trials related to Hemophilia A.
Filter by:The purpose of this study is to assess the safety and tolerability of MG1113 in the single ascending dose study (IV injection or SC injection) in healthy subjects and hemophiia patients.
Phase 3/4, single arm, open-label study to evaluate PK, safety, and efficacy of APVO101 prophylaxis in severe or moderately severe hemophilia B subjects < 12 years of age.
This is a research study of adults with hemophilia that have limitations in elbow joint motion that is the result of bleeding into the joint. People with hemophilia who have bleeding into their elbow joint may have limitations in elbow joint movement and pain in the joint. This research is being done to determine the effect a strength training program has on the amount of movement people with hemophilia and elbow joint disease have.
Characterizing the impact and treatment of reproductive tract bleeding on women and post-menarchal girls with bleeding disorders. Objectives: This is a cross-sectional observational study of women and girls (WG) with bleeding disorders enrolled in the American Thrombosis and Hemostasis Network (ATHN) dataset. Based on the investigators' study of currently available data of WG with bleeding disorders in the ATHNdataset, the investigators hypothesize that the information currently captured in the core data elements of the ATHNdatasest does not adequately capture data specific to WG with bleeding disorders. Further, the investigators hypothesize that it is feasible for Hemophilia Treatment Centers (HTCs) to include data points specific to WG with bleeding disorder when enrolling participants in the ATHNdataset. This hypothesis will be evaluated through the following specific aims: Specific Aim 1: Characterize reproductive tract bleeding in a cohort of WG with bleeding disorders cared for at US HTCs. Specific Aim 2: Characterize the treatment strategies for and the impact of heavy menstrual bleeding in a cohort of females with bleeding disorders cared for at HTCs. Specific Aim 3: Evaluate the feasibility of adding female specific core data points to the ATHNdataset.
The main aim of this study is to check for side effects and medical problems in participants with hemophilia A who are receiving ADYNOVATE in routine clinical practice. Also, it will be checked how effective ADYNOVATE is at stopping a bleed, at preventing bleeding episodes when used routinely for prevention and at controlling hemophilia-related bleeding during surgeries. Participants with hemophilia A who will be newly prescribed with ADYNOVATE treatment or have already been taking ADYNOVATE treatment according to the physician's decision will be included in this study. Participants will visit the study clinic as a part of their routine clinical practice and will be contacted by phone calls/home visits by home care workers for collection of treatment related data during the 6-month observation period.
This is a Phase I study. This research study is being conducted to find new ways to treat severe hemophilia A. This study is a gene therapy study. Gene therapy is an experimental way to introduce, into a person's cells, specific genetic material. A gene can be delivered/introduced into a cell using a carrier known as a "vector." In this study, a virus (lentivirus), the "vector", is used to introduce or deliver a gene that creates and stores a protein Factor VIII (FVIII) in your platelets. These platelets are made from stem cells (mother cells for your bone marrow) that are removed from your blood by a procedure called apheresis. This research study will take some of the patient's own stem cells, from the apheresis procedure, and genetically modify them using the vector in order to make them produce FVIII in platelets that arise from the stem cells. They will then give the genetically modified stem cells back to the patient so that they can possibly create platelets that produce and store Factor VIII on their own.
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 is a multi-center, open-label, single-arm trial to evaluate the efficacy,safety and pharmacokinetics of SCT800 in regular prophylaxis and perioperative treatment in patients (≥12 years old) with severe hemophilia A who have been previously treated with coagulation factor VIII(FVIII:C) . This study includes two phases: the screening period and prophylaxis period.Prophylaxis with 25 - 50 IU/kg of SCT800 shall be administered once every other day or three times per week starting from V1 and prophylaxis with SCT800 shall continue for 24 consecutive weeks.
Primary Objective: To characterize the long-term safety and tolerability of fitusiran Secondary Objectives: - To characterize the efficacy and long-term efficacy of fitusiran as assessed by the frequency of: - Bleeding episodes - Spontaneous bleeding episodes - Joint bleeding episodes - To characterize the effects of fitusiran on health-related quality of life (HRQOL) measures in participants ≥17 years of age
Primary objective: To assess the pharmacokinetics of Recombinant Human Coagulation Factor VIII-Fc fusion protein for Injection at two dose levels in patients with hemophilia A. Secondary objectives: To assess Safety and Tolerability by monitoring FVIII recovery and adverse events in patients with hemophilia A.