View clinical trials related to Hemophilia B.
Filter by:Nonacog alfa is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia B. The current single country, multi-centric, open label, non-randomized clinical trial is a post-approval study to fulfill the Central Drugs Standard Control Organization (CDSCO) request for supplementary information relating to the use of nonacog alfa in Indian subjects with hemophilia B.
Freeline 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.
GT2019001 is a Phase 1, open- label, non- randomized, uncontrolled, single dose pilot study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of BBM-H901 in hemophilia B subjects with ≤2IU/dl residual FIX levels. BBM-H901 is an adeno-associated viral (AAV) vector designed to drive expression of the human factor IX (hFIX) transgene and raise circulating levels of endogenous FIX.
The pivotal clinical data support prolonged dosing intervals of up to 14 days for routine prophylaxis in hemophilia B patients. The majority of adult and pediatric patients using Idelvion for routine prophylaxis had a median annualized spontaneous bleeding rate (AsBR) of 0.00. The majority (99%) of bleeding episodes were managed successfully with one or two infusions, and 94% of bleeds were controlled with only one infusion regardless of the cause or location. The rationale for this study is to collect the effectiveness and safety data of severe hemophilia B patients treated with Idelvion following institutional standard of care. It is the aim of this study to extend the results of the clinical trial program to a broader hemophilia B population, and to compare with current alternative factor IX treatments in Taiwan.
Hemophilia A and B are bleeding disorders caused by deficiency of factor VIII and IX, respectively. The deficiency of one of these coagulation factors is due to a mutation on the X chromosome. Accordingly replacement of the deficient factor is currently the main treatment for these disorders. The most disappointing complication of replacement therapy in hemophilia is the development of inhibitors. Unlike haemophilia , inhibitor development in patients with V Willebrand's Disease (VWD) is a rare complication of treatment. Studies on inhibitors whether on hemophilia or VWD are limited in our region. This study aims to 1. To estimate the frequency of factor inhibitors in hemophilia and VWD patients in our region. 2. To investigate modifiable risk factors associated with development of inhibitors in both diseases. 3. To correlate the level of inhibitor with the clinical presentation of the patients. 4. To assess influence of factor inhibitors on quality of life in patients who developed factor inhibitors in both diseases.
This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B with inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use. Participants who usually only take medicine to treat bleeds (on-demand) will be placed in one of two groups. In one group, participants will get study medicine from the start of the study. In the other group, participants will continue with their normal medicine and get study medicine after 6 months. Which treatment the participant gets is decided by chance. Participants who usually take medicine to prevent bleeds (prophylaxis treatment) or who are already being treated with concizumab (study medicine) will receive the study medicine from the start of the study. Participants will get 1 injection with the study medicine every day under the skin. This participants will have to do themselves and can be done at home. The study doctor will hand out the medicine in the form of a pen-injector. The pen-injector will contain the study medicine. The study will last for about six years. The length of time the participants will be in the study depends on when they agreed to take part or when the medicine is available for purchase in their country (12 November 2025 at the latest). Participants will have to come to the clinic for up to 41 times. The time between visits will be approximately 4 weeks for the first 6 to 12 months, depending on the group participants are in and approximately 8 weeks for the rest of the study. Participants will be asked to record information into an electronic diary during the study and may also be asked to wear an activity tracker.
This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B without inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use. Participants who usually only take medicine to treat bleeds (on-demand) will be placed in one of two groups. In one group participants will get study medicine from the start of the study. In the other group participants will continue with their normal medicine and get study medicine after 6 months. Which treatment the participant gets is decided by chance. Participants who usually take medicine to prevent bleeds (prophylaxis treatment) or who are already being treated with concizumab (study medicine) will receive the study medicine from the start of the study. Participants will have to inject themselves with the study medicine 1 time every day under the skin. This can be done at home. The study doctor will hand out the medicine in the form of a pen-injector. The pen-injector will contain the study medicine. The study will last for up to 6.5 years. The length of time the participant will be in the study depends on when they agreed to take part or when the medicine is available for purchase in their country (21 April 2026 at the latest). Participants will have to come to the clinic for up to 40 times. The time between visits will be approximately 4 weeks for the first 6 to 12 months depending on the group participants are in, and approximately 8 weeks for the rest of the study. If the participant attends extra visits due to the prescription medicine not being available for purchase in their country, these will be 14 weeks apart. Participants will be asked to record information in an electronic diary during the study and may also be asked to wear an activity tracker.
The purpose of this study is to investigate the safety and activity in the body of a new drug called SerpinPC. The study will be split into 7 parts: Part 1a will be conducted in healthy male volunteers in the UK (up to 15) and Parts 1b, 2, 3, 4, 5 and 6 will be conducted in haemophilia A & B patients in Moldova and Georgia. Part 1a of the study will look at how safe the drug is when given as single doses to healthy volunteers at different strengths and via 2 different routes of administration (through a vein or via an injection under the skin). Parts 1b, 2, 3, 4, 5 and 6 of the study will look at the safety of the drug when given as an injection under the skin to patients with severe haemophilia A or B. The study will also investigate how the levels of the drug in the blood change over a period of time and how the drug acts in the body by taking blood samples. These blood samples will measure the concentration of the drug in the blood and measure certain aspects of the blood to determine how the drug affects them. The study sponsor (ApcinteX) is developing this drug for the treatment of haemophilia A and haemophilia B, which are 2 types of rare blood disorders which affect the body's ability to form blood clots. Patients who have haemophilia A and B do not have certain clotting factors in their blood which means that they experience difficulty in stopping bleeding after injury and can be prone to extended periods of bleeding. Current treatments for haemophilia involves injections which replace the missing factors in the blood. However these treatments are short term and therefore patients require regular treatments in order to manage the condition. Therefore, there is a need to develop more effective treatments which provide longer term benefits. The aim of SerpinPC is to prevent bleeding rather than to have to treat bleeds to minimise pain and damage after they have occurred.
This multi-center, open label Phase 1 study will evaluate the pharmacokinetics, pharmacodynamics, and safety of a single IV dose of MarzAA followed by ascending single SC doses of MarzAA in adult subjects with moderate or severe Hemophilia A or B, with or without an inhibitor.
Phase 2b, single-center, open-label study designed to evaluate the pharmacokinetics, pharmacodynamics, efficacy and safety of subcutaneous (SC) prophylaxis treatment regimens with CB2679d in 6 adult, male subjects with severe congenital hemophilia B.