An Open Study to Investigate the Safety and Efficacy of Replenine®-VF in Severe Haemophilia B Patients...
Haemophilia BTo compare the first and second recovery assessments on Replenine®-VF and to evaluate recovery of different batches if patients changed batches during the study. To evaluate Replenine®-VF in terms of long-term clinical efficacy, tolerance and safety
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.
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
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.
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.
Study of Next-Generation Recombinant Factor IX Variant in Adult Subjects With Hemophilia B
Hemophilia BPhase 2b, single-center, open-label study designed to evaluate the pharmacokinetics, pharmacodynamics, efficacy and safety of subcutaneous (SC) prophylaxis treatment regimens with CB2679d in 6 adult, male subjects with severe congenital hemophilia B.
Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients...
Hemophilia BPhase 3/4, single arm, open-label study to evaluate PK, safety, and efficacy of APVO101 prophylaxis in severe or moderately severe hemophilia B subjects < 12 years of age.
Alterations in Coagulation Factor Levels in Patients With End Stage Liver Disease
Liver CirrhosisHemophilia5 moreThe goal of this observational study is to learn about the changes in coagulation factor VIII and IX levels in patients undergoing liver transplantation to help guide future management of coagulation factor replacement in patients with hemophilia and liver disease. The question we aim to answer is: should the recommendations for factor replacement in patients with hereditary bleeding disorders be altered in the setting of end stage liver cirrhosis? Participants will be asked to provide two blood samples, one at the beginning of their liver transplant, and one after their liver transplant.