Pharmacokinetics of Single Bolus Dose of NovoSeven® in Paediatric and Adult Patients With Haemophilia...
Congenital Bleeding DisorderHaemophilia A1 moreThis trial is conducted in Europe. The aim of this trial is to determine the pharmacokinetics of activated recombinant human factor VII (NovoSeven®) in haemophiliac patients in a non-bleeding state.
Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in...
Hemophilia AThe primary objective of the study is to evaluate the long-term safety of recombinant human Factor VIII Fc fusion protein (rFVIIIFc) in participants with hemophilia A. The secondary objective of the study is to evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in participants with hemophilia A.
Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric...
Hemophilia BThe primary objective of the study is to evaluate the safety of Recombinant Human Coagulation Factor IX Fc Fusion Protein (rFIXFc) in previously treated pediatric subjects with hemophilia B. Secondary objectives of this study in this study population are as follows: to evaluate the efficacy of rFIXFc for prevention and treatment of bleeding episodes; to evaluate and assess the pharmacokinetics (PK) of rFIXFc; to evaluate rFIXFc consumption for prevention and treatment of bleeding episodes
Prophylaxis Versus on Demand Treatment for Children With Hemophilia A
Hemophilia AComparison of the effect of three times a week prophylaxis on all bleeds with on-demand treatment for children with severe Hemophilia A.
A Comparison Study of Bypassing Agent Therapy With and Without Tranexamic Acid in Haemophilia A...
Hereditary Factor VIII Deficiency Disease With InhibitorActivated prothrombin complex concentrate (aPCC) and recombinant activated factor VII (rFVIIa) are the only two drugs that are available to treat bleeds in haemophilia A patients with high titer inhibitors. However, management of bleeds in these patients can be challenging due to variation in response and lack of standardized methods to monitor the effect. We hypothesized that significant increase in whole blood clot stability could be achieved when tranexamic acid was given concomitantly with bypassing-agents while thrombin generation remains unaffected. In this prospective crossover study the effect of aPCC and rFVIIa with and without TXA on clot stability and thrombin generation capacity (ETP) were studied, using thromboelastography (ROTEM) and thrombin generation assay (TGA), respectively. In addition, the risk of thrombosis and disseminated intravascular coagulation (DIC) was assessed.
IMMUNINE Pre-Treatment Study
Hemophilia BThe primary objective of this study is to prospectively document the exposure to IMMUNINE and to monitor FIX inhibitors over a period of approximately 20 to 50 exposure days while receiving prophylactic treatment in up to 50 previously treated patients (PTPs) aged 12-64 years and approximately 20 pediatric PTPs up to 11 years of age with severe (FIX level < 1%) or moderately severe (FIX level <= 2%) hemophilia B who are planned to enter BAX326 study 250901, provided all eligibility criteria are met. In addition, this study will evaluate the efficacy, safety, immunogenicity, thrombogenicity, and health-related quality of life (HR QoL) of these subjects.
Safety and Efficacy of Turoctocog Alfa (N8) in Prevention and On-demand Treatment of Bleeding Episodes...
Congenital Bleeding DisorderHaemophilia AThis trial is conducted in Asia, Europe, Japan, Oceania, North America and South America. The aim of the trial is to investigate the safety and efficacy of turoctocog alfa (N8) in Haemophilia A patients. The trial is an extension to trials NN7008-3543 (start: March 2009, stop: September 2011) and NN7008-3545 (start: May 2010, stop: November 2011) and the pharmacokinetic trials NN7008-3600 (start: November 2010, stop: October 2011), NN7008-3893 (start: June 2011, stop: September 2011) and NN7008-4015 (start: August 2012, stop: March 2013).
A Single Dose Trial Investigating Safety and Local Tolerability of Ascending Doses of Long Acting...
Congenital Bleeding DisorderHaemophilia A2 moreThis trial is conducted in Europe. The aim of this clinical trial is to investigate the safety, local tolerability and pharmacokinetic profile (the determination of the concentration of the administered medication in blood over time) of long acting activated recombinant human factor VII when injected subcutaneously (under the skin).
Impact of Conservative Treatment by Custom-made Orthoses in Patients With Haemophilic Ankle Arthropathy...
Haemophilia AHaemophilia BThe purpose of this study intend to evaluate the effectiveness of functional orthoses (including custom-made insoles and shoes) for preventing and controlling repetitive haemarthrosis in patients suffering of haemophilic ankle arthropathy, as well as the orthoses' impact on foot health-related quality of life.
High Dose of Activated Recombinant Human Factor VII for Treatment of Mild/Moderate Joint Bleeds...
Congenital Bleeding DisorderHaemophilia A With Inhibitors1 moreThis trial is conducted in Europe. The aim of this trial is to evaluate the efficacy and safety of two dose schedules of activated recombinant human factor VII in treatment of joint bleeds in haemophilia patients with inhibitors.