A MULTICENTER, OPEN-LABEL, PHASE III CLINICAL TRIAL TO EVALUATE THE EFFICACY, SAFETY, AND PHARMACOKINETICS OF EMICIZUMAB GIVEN EVERY 2 WEEKS AND 4 WEEKS IN HEMOPHILIA A PEDIATRIC PATIENTS AGED LESS THAN 12 YEARS WITHOUT FVIII INHIBITORS

CHUGAI PHARMACEUTICAL CO., LTD0 个研究点目标入组 13 人开始时间: 2017年9月12日最近更新: 2023年10月17日

适应症

试验速览

Emicizumab administered Q2W or Q4W was efficacious and safe in paediatric patients with severe haemophilia A without inhibitors.

•1) Children < 12 years of age at the time of informed consent
•2) Body weight > 3 kg at the time of informed consent
•3) Diagnosis of severe congenital hemophilia A (i.e., endogenous FVIII activity level < 1%)
•4) A negative test for FVIII inhibitors (i.e., < 0.6 BU/mL) in 8 weeks prior to enrollment
•5) For patients < 2 years of age: Documentation of bleeds and treatment with coagulation factor in the last 12 weeks prior to enrollment
•For patients >= 2 years of age: Documentation of bleeds and treatment with coagulation factor in the last 24 weeks prior to enrollment

•1) Inherited or acquired bleeding disorder other than hemophilia A
•2) Ongoing immune tolerance induction (ITI) therapy
•3) Previous (in the past 12 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease
•4) Other diseases (i.e., certain autoimmune disease [e.g., systemic lupus erythematosus, antiphospholipid antibody syndrome], cardiovascular disease [e.g., fallot's tetralogy, tricuspid atresia]) that may increase risk of bleeding or thrombosis
•5) Patients who are at high risk for thrombotic microangiopathy (TMA) (e.g., have a previous medical or family history of TMA [e.g., thrombotic thrombocytopenic purpura, atypical hemolytic uremic syndrome]), in the investigator's judgment

发起方

CHUGAI PHARMACEUTICAL CO., LTD