The Hemophilia Ultrasound Project
Hemophilia AHemophilia BTo evaluate the prevalence of subclinical arthropathy in children with severe hemophilia undergoing a prophylaxis regimen and without evidence of target joints, using a validated ultrasound scoring method.
Prediction of the Efficacy of Activated Recombinant Human Factor VII in Adult Congenital Haemophilia...
Congenital Bleeding DisorderHaemophilia A With Inhibitors1 moreThis trial is conducted in Europe and the United States of America (USA). The aim of this trial is to evaluate the basal and spiked TEG® (Thromboelastography) or ROTEM® (Thromboelastometry) profiles of frequently bleeding haemophilia subjects with inhibitors in a non-bleeding state.
Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With Hemophilia B...
Hemophilia BTo evaluate the safety (acute adverse effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding and control of hemorrhaging during prophylaxis of IB1001 in subjects with hemophilia B.
A Study to Evaluate the Real-world Usage and Effectiveness of Elocta and Alprolix in Patients With...
Haemophilia AHaemophilia BElocta (rFVIIIFc) and Alprolix (rFIXFc) are recombinant extended half-life coagulation factor products. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Elocta and Alprolix in the prophylactic treatment of haemophilia A and B.
An Institutional Pilot Study to Investigate Physical Activity Patterns in Boys With Hemophilia
Hemophilia AHemophilia B2 moreThis pilot project was developed to investigate subjective and objective data related to the patterns of physical activity participation among hemophilia patients (FVIII or FIX) between the ages of 5 and 18 years. Physical activity participation among different levels of disease severity will be compared using accelerometers to calculate the amount of time spent in moderate to vigorous physical activity (MVPA) and the number of bouts of MVPA; additionally, data will be collected from two validated questionnaires (PedHAL and 3 day physical activity recall). Current literature that relates level of physical activity to disease severity in the pediatric hemophilia population is limited. The information gained about the type and quantity of physical activity participation in children with varying levels of hemophilia will assist in the development of education and interventions to promote good physical activity and potentially examine the role of tailored prophylaxis.
Study of Prophylaxis, ACtivity and Effectiveness (SPACE) in Hemophilia Patients Currently Treated...
Hemophilia AHemophilia BThis 6-month prospective outcomes study addresses the association between timing of infusion, patient activity levels, and bleeding episodes through patient-reported measurements.
BeneFIX Drug Use Results Survey [All-Case Surveillance]
Hemophilia BThe survey is intended to investigate the following matters, etc. under the actual use status after marketing in all patients who are administered this drug for a certain period of time after the launch. Occurrence status of adverse events Factors that may influence the safety Efficacy In addition, the following occurrence statuses will be investigated as priority items of the survey: Incidence rate of inhibitor, reduction in drug, efficacy, Allergic reaction, and Thrombosis.
A Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia...
Haemophilia BAlprolix (rFIXFc) is a recombinant extended half-life coagulation factor product. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Alprolix in the on-demand and prophylactic treatment of haemophilia B.
Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe...
Hemophilia BA long-term follow-up study to evaluate the safety, tolerability, and efficacy of DTX101 in adult males with moderate/severe to severe hemophilia B.
Pharmacogenetic Testing of Saliva Samples From Patients With Five or More Exposure Days to rFVIIa...
Congenital Bleeding DisorderHaemophilia A With Inhibitors1 moreThis study is conducted globally. This study describes pharmacogenetic testing of saliva samples from patients who participated in the NN1731-3562 trial (adept™2) (NCT01392547). The objective is to determine the HLA (human leukocyte antigen) type and polymorphisms in the FVII gene in patients previously exposed to rFVIIa analogue.