A MULTICENTER, OPEN-LABEL, PHASE III CLINICAL TRIAL TO EVALUATE THE EFFICACY, SAFETY, AND PHARMACOKINETICS OF SUBCUTANEOUS ADMINISTRATION OF EMICIZUMAB IN HEMOPHILIA A PEDIATRIC PATIENTS WITH INHIBITORS

CHUGAI PHARMACEUTICAL CO., LTD.0 个研究点目标入组 88 人开始时间: 2016年6月14日最近更新: 2023年10月17日

适应症

试验速览

Emicizumab prophylaxis achieved substantial efficacy and was well tolerated in children with hemophilia A with FVIII inhibitors.

•1) Aged younger than 12 years old or 12~17 years old who weigh < 40 kg at the time of informed consent
•2) Body weight > 3 kg at the time of informed consent
•3) Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (i.e.,>= 5 Bethesda Units)
•4) Requires treatment with bypassing agents

•1) Inherited or acquired bleeding disorder other than hemophilia A
•2) Ongoing (or plan to receive during the study) immune tolerance induction therapy(ITI) or prophylaxis with FVIII. Patients awaiting initiation of ITI will be eligible. Patients in whom ITI has failed will be eligible with a 72-hour washout period prior to the
•first emicizumab administration.
•3) Previous or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which antithrombotic treatment is not currently ongoing) or signs of thromboembolic disease

发起方