Efficacy Study of Activated Prothrombin Complex for Prevention of Bleeds in Hemophilia A With Inhibitors...
Hemophilia A With InhibitorsThe objective of this study is to assess whether prophylactic therapy with an activated prothrombin complex concentrate (FEIBA)will result in a significant reduction in the number of bleeds in patients with hemophilia and persistent high responding inhibitors.
Low-dose ITI Strategy for Children in Hemophilia A With High-titer Inhibitor and Poor ITI Risk in...
Hemophilia A With InhibitorThe study start on January 18, 2017. The Severe(FⅧ<1%) and moderate hemophilia A (FⅧ1%~5%)children with high titer inhibitor(historical peak inhibitor titer≥5BU ) combining with poor ITI risk(s) were enrolled. The low-dose ITI was alone or combined with immunosuppression.
Joint Status in Subjects With Severe Hemophilia A in Relation to Different Treatment Regimens
Hemophilia AThe joint status (knees, ankles) of patients suffering from severe Hemophilia A (too little blood clotting factor VIII in blood) is evaluated in a single magnetic resonance imaging session. No study medication is given.
Pharmacokinetic (PK)Research on Chinese Children of Hemophilia
PharmacokineticsThe study start on June 30, 2018. The Severe(F Ⅷ<1%) hemophilia A children without F Ⅷ inhibitor combining were recruited to Test the concentration of the drug in the blood to provide better treatment.
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 to Test the Safety of an Investigational Drug Given Repeatedly to Adult Men With Severe Hemophilia...
HemophiliaThe purpose of this study is to investigate the safety of a test drug to treat hemophilia in adult men.
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.
ATHN 2: Factor Switching Study
HemophiliaThis is a longitudinal, observational study of patients with Hemophilia A or B who are planning to switch to a newly approved coagulation factor replacement product, or who have recently switched factor products. The study will follow each patient for up to 1 year. Patients will be recruited at Hemophilia Treatment Centers (HTC) which are ATHN-affiliates. The primary outcome being studied is the development of inhibitor (i.e., antibodies to factor) at 1 year or 50 exposure days, whichever comes first. The study will be conducted at approximately 30 HTCs, with a planned enrollment of 600 patients.The entire study duration is projected to be approximately 6 years. In addition, optional substudies will be included for some products, as "Product-Specific Modules". These will be questionnaires to collect data for subjects receiving selected Factor products. For example, subjects receiving Kovaltry will be approached to participate in the 'Kovaltry Product-Specific Module'; subjects receiving Adynovate will be approached to participate in the 'Adynovate Product-Specific Module'. Questions will be related to product use, perceptions of product use, and other post-marketing consumer data.
World Bleeding Disorders Registry Pilot Study
Hemophilia A and BThe World Federation of Hemophilia (WFH) is conducting the pilot phase of an international, observational, World Bleeding Disorders Registry (WBDR). This pilot phase will assess the feasibility of conducting an expanded WBDR to more centres with a more comprehensive case report form, around the world.
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.