View clinical trials related to Hemophilia A.
Filter by:This study is to generate qualitative data to evaluate the impact of frequency of FVIII infusions on patients' satisfaction with treatment and their quality of life.
Prospective, multinational, non-interventional post-authorisation study to collect additional clinical data and to ensure consistency in the long-term between the outcome from pre-authorisation clinical studies (in 135 previously treated paediatric and adult patients) and routine clinical practice. Besides aspects such as general product safety and efficacy, there will be a focus on immunogenicity, particularly on inhibitor development. The diagnosis of FVIII inhibitor will be based on clinical observations and confirmed by FVIII inhibitor testing in the laboratory.
The purpose of this study is to obtain additional data on the safety and efficacy of Wilate in PTPs with hemophilia A with at least 150 previous exposure days (EDs) to a FVIII concentrate who undergo prophylactic treatment with Wilate for 6 months and at least 50 EDs, thus supplementing the existing database to obtain approval of Wilate for the indication hemophilia A in the USA.
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%).
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.
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.
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).
The purpose of this study is to evaluate the relative effectiveness of two psychological interventions, cognitive-behavioral therapy (CBT) and hypnosis (Hyp), in preventing and managing pain, regulating emotional state and improving quality of life in Portuguese PWH.