View clinical trials related to Hemophilia A.
Filter by:In this study, researchers will observe and study the data from participants with hemophilia A who receive damoctocog alfa pegol as prescribed by their doctors. Participants will not receive any advice or changes to their healthcare during the study. Hemophilia A is a genetic bleeding disorder. It is caused by the lack of a protein called clotting factor 8 (FVIII) that helps blood to clot properly. Lack of FVIII can result in excessive blood loss or bleeding inside the body after being injured or having surgery. The study drug, damoctocog alfa pegol, can be used to prevent or treat bleeding episodes by replacing missing FVIII in the body of people with hemophilia A. It is already approved for people with hemophilia A who are at least 12 years old and have previously used other hemophilia A treatments. Through this study, researchers want to learn more about its safety in a real-world setting. The participants will receive damoctocog alfa pegol as prescribed by their doctors during routine practice according to the approved product information. The main purpose of this study is to learn more about how safe damoctocog alfa pegol is in Korean participants with hemophilia A who previously used other hemophilia A treatments. To do this, researchers will collect information about any medical problems participants have during their treatment. Data will be collected from December 2023 to March 2026 and cover a period of about 8 months for each participant. Data will come from participants' health records and information collected during their routine clinic visits. In this study, only available data from routine care will be collected. No visits or tests are required as part of this study.
Hemophilia A is an X linked disorder characterized by a deficiency in Factor VIII. The clinical hallmark of this disease is increased tendency to spontaneous bleeding with hemarthrosis accounting for 90% of the hemorrhages. In addition to development of hemophilic arthropathy, the emergence of alloantibodies that inhibit the coagulant activity of FVIII remains the most feared complication related to the treatment of hemophilia A. 30% of patients with hemophilia A develop these inhibitors, making treatment with standard replacement therapy ineffective. Up until the approval of emicizumab, bypassing agents like activated prothrombin complex concentrate (aPCC) and activated recombinant activated factor VII (rFVIIa) were the only approved therapies for the treatment hemophilia A with inhibitors. The response to bypassing therapy is often unpredictable, variable and difficult to monitor. Emicizumab is a first generation bispecific antibody mimicking the activity of FVIIIa in tenase complex. Early in the HAVEN 1 clinical trial with emicizumab (1), cases of thrombotic microangiopathy (TMA) and thrombotic events were reported when on average a cumulative amount of >100 U/kg/24 hours of aPCC was administered for 24 hours or more to patients receiving emicizumab, resulting in a protocol adjustment for the HAVEN 1 trial and subsequent trials to recommend using the lowest doses of bypassing agents expected to achieve hemostasis, and avoiding the combination of emicizumab and aPCC if possible. So far the only proposed strategies for treating events of breakthrough bleeds in patients on emicizumab prophylaxis include rFVIIa, FVIII in patients with a low titer of inhibitors, and lower doses of aPCC, knowing that emicizumab provides an existing level of thrombin generation. (2) While the exact mechanism leading to the development of thrombotic complications (TMA and VTE) remains poorly understood, many speculated on the accumulation of FIX and FX, the substrates of emicizumab, with multiple doses of aPCC (3) Mim8 is a novel, next-generation FVIIIa mimetic designed for the subcutaneous prophylactic treatment of patients with HA with and without inhibitors. Mim8 is a fully human, bispecific antibody that mimics FVIIIa function by bridging FIXa and FX on the phospholipid surface of activated platelets, enhancing the proteolytic activity of FIXa, and thus facilitating effective FX activation. Data from studies using in vitro HA-like human blood, as well as in vivo HA mouse models, indicate that Mim8 is ~15-fold more potent than a sequence identical analogue (SIA) of the FVIII mimetic emicizumab (4). Mim8 nonclinical safety program in cynomolgus monkeys showed that subcutaneous administration of up to 3 mg/kg/week (several fold greater than expected clinical exposure) for 26 weeks resulted in relevant pharmacodynamic effects, observed in thrombin generation and APTT, with no signs of thrombi or excessive coagulation activation. So far, Mim8 procoagulant activity was evaluated in platelet poor plasma samples only (5,6) This in vitro study aims to evaluate TGA to monitor Mim8. We hypothesized that TG profiles (ETP and peak thrombin) may be different with different triggers used. We recently modified TGA to better detect haemostatic activity of emicizumab, by using a combined trigger (low TF+low FIXa). Differently from emicizumab, Mim8 stimulates the proteolytic activity of FIXa in the range of 15,000-fold. TGA test conditions may be therefore different for Mim8 and emicizumab.
The goal of this multicenter, two-stage, open-label study is to investigate the safety, immunogenicity, and efficacy of ANB-010 in subjects with hemophilia A. The study will have a dose-escalation design with elements of phase I/II seamless adaptive design.
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.
- To outcome between low dose Emicizumab and low dose prophylaxis with FVIII concentration - To study pharmacokinetic, side effect of low dose Emicizumab
This is an exploratory, single group, Phase 1, 1-arm study to assess treatment with fitusiran prophylaxis after switching from emicizumab prophylaxis. This study aims to evaluate the safety and tolerability of switching to fitusiran after a transition period from the last dose of emicizumab. The study will be conducted in male participants with severe hemophilia A, with or without inhibitors, aged ≥18 years, who were previously receiving emicizumab prophylaxis. Study details include: - The study duration will be up to approximately 28 months: - There will be an approximately 2-month screening period. - There will be a transition period before fitusiran treatment starts (pre-fitusiran treatment period) - The fitusiran treatment duration will be up to 18-months (fitusiran treatment period) - The antithrombin (AT) follow-up (FU) period will be up to 6 months after the last dose of fitusiran (during which the AT activity level will be monitored at approximately monthly intervals following the final fitusiran dose until AT activity levels return to at least 60%). - The study site visits are scheduled at monthly/ every 2 months intervals of 28 days (4 weeks) / 56 days (8 weeks), respectively, during the fitusiran treatment period.
This is a phase 2 trial of foscenvivint in liver cirrhosis patients caused by HIV/HCV co-infection with hemophilia to evaluate the efficacy, safety and pharmacokinetics.
To evaluate the prophylactic efficacy of recombinant human coagulation factor Ⅷ-Fc fusion protein (FRSW117) for injection in patients with severe hemophilia A. To evaluate the safety of recombinant human coagulation factor Ⅷ-Fc fusion protein (FRSW117) for injection in patients with severe hemophilia A. Secondary purpose: To evaluate the efficacy of recombinant human coagulation factor Ⅷ-Fc fusion protein for injection (FRSW117) in hemostasis and surgical hemostasis in patients with severe hemophilia A. To evaluate the pharmacokinetic (PK) characteristics of recombinant human coagulation factor Ⅷ-Fc fusion protein (FRSW117) for injection in treated patients with severe hemophilia A. To evaluate the immunogenicity of recombinant human coagulation factor Ⅷ-Fc fusion protein (FRSW117) for injection in treated patients with severe hemophilia A.
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 a multi-center, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H803 injection in heavy Hemophilia A subjects. BBM-H803 is an adeno-associated viral (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII transgene and raises circulating levels of endogenous FVIII.