View clinical trials related to Hemophilia A.
Filter by:The aim of this study is to evaluate the prevalence of synovitis in adult patients with haemophilia A.
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 goal of this multicentre, prospective, open-label, cross-over clinical study is to determine whether individualized PK-guided dosing of emicizumab is non-inferior to conventional dosing of emicizumab in the prevention of bleeding in congenital haemophilia A patients.
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 this study is to evaluate the efficacy of SPK-8011 in preventing bleed episodes compared with FVIII prophylaxis in participants with hemophilia A without FVIII inhibitors on routine FVIII prophylaxis.
This study will assess the safety and efficacy of multiple-dose of STSP-0601 for the treatment of bleeding episodes in hemophilia A or B patients with inhibitor.
Background. The main physical sequela of patients with hemophilia is the development of a progressive, degenerative intra-articular lesion, known as hemophilic arthropathy). This sequela is manifested by chronic pain, limited range of motion, axial abnormalities, and periarticular muscle atrophy. Objective. To assess the safety and effectiveness of an intervention through blood flow restriction, regarding the frequency of bleeding and the improvement in muscle activation and strength, range of motion, stability, joint pain, joint status and the perception of quality of life in patients with hemophilic ankle arthropathy. Study design. Randomized, multicenter, single-blind clinical study. Method. 32 patients with hemophilia A and B will be recruited in this study. Patients will be recruited in 4 regions of Spain. The dependent variables will be: bleeding frequency (self-registration), pain (measured with the visual analog scale and pressure algometer), quality of life (SF-36 scale), joint status (Hemophilia Joint Health Score scale), strength (dynamometer) and muscle activation (surface electromyograph), range of motion (goniometer) and stability (The Single Leg Stance Test). Three evaluations will be carried out: pre-treatment, post-treatment and after a follow-up period of 4 weeks. Expected results. Observe the safety of blood flow restriction in hemophilia patients. To analyze the efficacy of blood flow restriction in improving muscle strength and activation, range of motion, chronic pain, stabilit, and the perception of quality of life in patients with hemophilic ankle arthropathy.
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in Japanese patients with severe hemophilia A.
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.