View clinical trials related to Hemophilia A.
Filter by:This trial is conducted globally. The aim of this trial is evaluating the pharmacokinetics (the exposure of the trial drug in the body) of NovoEight® (turoctocog alfa) in relation to BMI (body mass index) in subjects with haemophilia A.
This trial is conducted in China. The aim of this trial is to evaluate the clinical efficacy of turoctocog alfa in treatment of bleeding episodes in Chinese patients with severe haemophilia A (FVIII≤1%).
Severe haemophilia A and B (SHA, SHB) are inherited bleeding disorders affecting male patients and are characterised by low levels of circulating clotting factors VIII and IX respectively. Clinically low levels present with multiple recurrent bleeds into joints and muscle from the first couple of years of life. In addition patients may present with spontaneous and potentially fatal bleeding into any organ. The mainstay of treatment is replacement with the missing factor in the form of intravenous injections of factor VIII and IX. Clotting factors can be given to treat a bleed or can be given to prevent a bleed, and the latter is termed prophylaxis. Regular prophylaxis is the current standard of care and aims to decrease spontaneous bleeding events and resulting joint damage, and this requires patients to self-infuse factor into their veins two to four times week. Patient's compliance with prescribed regimen and recommendations has a significant influence on outcomes. Advances in biomolecular and protein engineering have extended the duration of the effect of clotting factor VIII and IX through multiple mechanisms. This extension of the duration of the effect presents the clinician and patients with opportunities to tailor the treatment to their particular needs, circumstances and body other characteristics. It has been suggested that decreasing the frequency of infusions will improve adherence and thus contribute to improved outcomes. In rare disorders, it is an accepted fact that post-marketing studies are crucial to understand the generalisability of the efficacy and safety outcomes and identify any new safety and efficacy concerns in relation to specific population group. The investigators propose the development of a registry for systematic collection of information with the dual aim of analysing the relationship between patient and treatment characteristics, and outcomes, and simultaneously identify areas for practice development that can improve the overall quality of life experienced by the haemophilia patient community.
Efficacy, Safety and Pharmacokinetics Study of a rFVIII in Chinese subjects with Hemophilia A.To assess efficacy and safety of rFVIII administered as treatment and as on-demand therapy in adult and adolescent (12-65 years) patients with severe or moderately severe Hemophilia A. To determine the pharmacokinetic (PK) parameters of rFVIII.
Primary: To characterize the safety of RIXUBIS when used under normal clinical care in South Korea. Secondary: To describe hemostatic effectiveness in subjects receiving RIXUBIS under normal clinical care in South Korea.
In this trial safety and efficacy of SCT800 (B-domain deleted recombinant factor VIII) is being evaluated in 50 subjects, 12 to 65 years of age, with moderate to severe Hemophilia A. These subjects will receive open label treatment with SCT800 for approximately 6 months for on-demand treatment.
Investigating single dose pharmacokinetics and safety of turoctocog alfa pegol from the pivotal process and turoctocog alfa pegol from the commercial process in patients with severe haemophilia A
A Phase 1, randomized, single-blind, placebo-controlled, single dose, dose-escalation study to assess the safety, pharmacokinetic and pharmacodynamic profile of subcutaneous administration of a long-acting recombinant factor VIIa (MOD-5014) in healthy adult males.
The German Pediatric Hemophilia Research Database will collect data on the prophylactic and therapeutic use of factor concentrates, complications, outcome measures (joint scores, QoL) and living circumstances in newly diagnosed children with hemophilia.
Hemophilia A is a rare X chromosome-linked recessive bleeding disorder that concerns one individual in 5000. In its severe form, hemophilia A is a life-threatening, crippling hemorrhagic disease. The treatment of bleeding episodes in hemophilia A patients involves the administration of exogenous human FVIII to restore normal hemostasis. The main complication of the substitutive treatment of hemophilia A is the development, in 15 to 30% of the cases, of anti-FVIII antibodies (FVIII inhibitors) that neutralize the pro-coagulant activity of therapeutically administered FVIII. In 2003, the average annual cost of care for a patient with hemophilia A was evaluated to be equal to 63,000 euros (2), which, in France (6000 patients), represents an annual budget of 378 million euros. In order to reduce the cost of treatment and to bypass this complication, different therapeutic strategies (new products or adjunctive therapeutic options) have been explored, including platelet infusion, tranexamic acid, amino caproic acid, molecules that block tissue factor pathway inhibitor, combination of phospholipid -Factor Xa- Factor XIII and antibodies directed to the Tissue Factor Inhibitor Pathway (TFPI). Recently, Soluble thrombomodulin (Solulin) have been developed. This molecule may be used to partially correct the premature lysis defect in Factor VIII deficient plasma through an activated TAFI - dependent mechanism. With a long half-life (15- to 30-hour) and effective dose range estimated to range from the sub-nanomolar to approximately 40nM, Solulin could potentially be administered on a weekly basis and provide the basis for a factor-sparing regime that would cut costs and make therapy more widely available. However, before proceeding to advanced trials, safety concerns stemming from the anticoagulant properties of Solulin must be addressed. The development of Solulin mutants lacking protein C activation capacity would make this concern redundant. At the same time, such mutant molecules are likely to possess an effective dose range. Our project is to compare the behavior of recombinant Solulin and mutants of Solulin lacking protein C activation capacity with respect to their ability to stabilize fibrin clots in whole blood of humans with different coagulation factor deficiencies (hemophilia A, hemophilia B and rare blood coagulation deficiencies (factor X, VII, V).