View clinical trials related to Hemophilia B.
Filter by:The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in healthy participants (Part A) and to evaluate the safety, tolerability, PK, PD, and efficacy of SR604 in participants with severe Hemophilia A or Hemophilia B, with or without inhibitors (Part B).
The purpose of this study is to show that KN057 can prevent bleeds in patients with haemophilia A or B with inhibitors and is safe to use. Successfully screened participants will be randomly assigned to KN057 Prophylaxis (Arm 1) versus No Prophylaxis (Arm 2) at a ratio of 2:1. Participants in KN057 Prophylaxis will receive KN057 prophylaxis for 52 weeks upon enrollment. Participants in No Prophylaxis will first receive on-demand treatment for 26 weeks, then switch to KN057 prophylaxis for 26 weeks.The trial period is 59 weeks, including a 3-week screening period, a 26-week main trial, a 26-week extension period, and a 4-week follow-up period after the last administration.
The purpose of the study is to investigate the safety and effectiveness of Alhemo® in all participants under real-world clinical practice in Japan. Participants will get Alhemo® as prescribed by the study doctor. The study will last for about 2 years.
Haemophilia A and haemophilia B are inherited bleeding disorders resulting from the absence or deficiency of coagulation factors VIII and IX, respectively. The peri-operative period of people with haemophilia is commonly managed with replacement therapy. In phase 3 studies of Elocta® (extended half-life recombinant factor VIII-Fc) and Alprolix® (extended half-life recombinant factor IX-Fc), haemostatic efficacy was demonstrated to be good or excellent, close to the haemostatic efficacy usually seen in people without haemophilia, with maintenance and stability of circulating FVIII and FIX levels compatible with the surgical procedure, while reducing the frequency of infusions and consumption of therapeutic units. In 2019, the National Protocol for Diagnosis and Care in haemophilia recommended 2 methods for managing patients with haemophilia in the peri-operative period, either discontinuous injections of standard or extended half-life factor VIII/IX or a continuous infusion of FVIII/IX. Many countries, including France, have adopted these rFVIII/IXFc therapeuitic products and recommended their use in the surgical management of patients. However, the use of these two products in real life during surgery in haemophilic A and B patients has not been described in detail. It seems therefore relevant to better document their use in order to progressively specify their use during surgeries with varied bleeding risks.
Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma. Its presence in maternal plasma has allowed development of noninvasive prenatal diagnosis for single-gene disorders (SGD-NIPD). This can be performed from 9 weeks of amenorrhea and offers an early, safe and accurate definitive diagnosis without the miscarriage risk associated with invasive procedures. One of the major difficulties is distinguishing fetal genotype in the high background of maternal cfDNA, which leads to several technical and analytical challenges. Besides, unlike noninvasive prenatal testing for aneuploidy, NIPD for monogenic diseases represent a smaller market opportunity, and many cases must be provided on a bespoke, patient- or disease-specific basis. As a result, implementation of SGD-NIPD remained sparse, with most testing being delivered in a research setting. The present project aims to take advantage of the unique French collaborative network to make SGD-NIPD possible for theoretically any monogenic disorder and any family.
This is an open- label, non- randomized, uncontrolled, dose-escalation pilot study to evaluate the safety and efficacy of KL001 injection solution in hemophilia B subjects with ≤2 IU/dl residual FIX levels.
The goal of this multicenter, two-stage, open-label study is to investigate the safety, immunogenicity, and efficacy of ANB-002 in subjects with hemophilia В. The study will have a dose-escalation design with elements of phase I/II seamless adaptive design.
This is an open- label, non- randomized, uncontrolled, dose-escalation pilot study to evaluate the safety and efficacy of KL001 injection solution in hemophilia B subjects with ≤2 IU/dl residual FIX levels.
This is a Phase 1a, double-blind, randomized, placebo- controlled, SAD study to assess safety, tolerability, PK, and PD of TU7710 in warfarin treated healthy male participants.
The goal of this observational study is to learn about the changes in coagulation factor VIII and IX levels in patients undergoing liver transplantation to help guide future management of coagulation factor replacement in patients with hemophilia and liver disease. The question we aim to answer is: should the recommendations for factor replacement in patients with hereditary bleeding disorders be altered in the setting of end stage liver cirrhosis? Participants will be asked to provide two blood samples, one at the beginning of their liver transplant, and one after their liver transplant.