View clinical trials related to Hemophilia A.
Filter by:This study prospectively investigates the safety, FVIII immunogenicity, and hemostatic efficacy of prophylactic HEMLIBRA® given with a concomitant low dose recombinant factor VIII (rFVIII) known as NUWIQ®, in HA infants and children <3 years old who have had little to no previous exposure to FVIII. In addition, the study investigates the safety and efficacy of a novel FVIII ITI regimen in children <21 with existing low and high titer inhibitors (LTI and HTI).
This is a non-interventional, multicenter, observational, international study in male persons with haemophilia A who have developed inhibitors to any replacement coagulation factor VIII (FVIII) product. The purpose of the study is to capture different approaches in the management of persons with haemophilia A and FVIII inhibitors, document current immune tolerance induction approaches, and evaluate the efficacy and safety of immune tolerance induction, including the combination of FVIII and emicizumab. Patients will be assigned to 1 of 3 groups based on the treatments they receive, and may switch to another group if their treatment is changed. Participants will be followed after a maximum observational period of 5 years.
This study is being conducted to compare how the body distributes and excretes the drugs Jivi (BAY 94-9027) and Adynovi. Jivi is a recently approved blood clotting Factor VIII (FVIII) medication for the treatment of hemophilia A (bleeding disorder resulting from a lack of FVIII). Both drugs are FVIII products which have been manufactured via recombinant technology and have an extended half-live, i.e. they will stay longer in the body than other FVIII products. Therefore these products act longer in the body which reduces the frequency of drug injections. To compare the two drugs, a cross-over design was chosen, i.e. each patient will receive both products one after another. Patients participating in this study will receive one dose of Jivi and one dose of Adynovi. Both drugs are injected into a vein. Observation will last for about 10 weeks, and blood samples will be taken from the participants to measure the blood levels of FVIII. Generic name of Jivi is Damoctocog-alfa-pegol, generic name of Adynovi is Rurioctocog alfa pegol.
The purpose of this study is to investigate the safety of a test drug to treat hemophilia in adult men.
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.
Primary Objective: - To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to <12 years of age) with severe hemophilia A or B Secondary Objectives: - To characterize the safety and tolerability - To determine fitusiran plasma concentrations at selected time points
This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor IX into patients with hemophilia B, to evaluate the safety and efficacy of infusion of lentiviral gene modified autologous stem cells in patients.
A chart review study of patients with haemophilia A with inhibitors treated with rFVIIIFc (Elocta®) for immune tolerance induction.
This study is a multi-center, open-label, extension trial to evaluate the safety, efficacy of SCT800 in long term prophylaxis and on-demand treatment in patients with severe hemophilia A who have been previously treated with coagulation factor VIII(FVIII) . 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, on-demand treatment is given when bleeding episodes occur. The study period is 120 consecutive weeks.
This study is a multicenter phase III uncontrolled open-label 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) . 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 Visit 1 and prophylaxis with SCT800 shall continue for 24 consecutive weeks.