A prospective, multicenter study to evaluate the safety of emicizumab under and after immune tolerance induction in patients with congenital hemophilia A with FVIII inhibitors

Matsushita Tadashi0 个研究点目标入组 15 人开始时间: 2020年8月3日最近更新: 2023年11月20日

适应症

试验速览

暂无简介。

•Patients must meet all of the following criteria for study entry:
•1) Informed Consent Form signed by the patient or the patient's legally authorized representative. Pediatric patients who are capable: Informed Assent Form signed by the patient
•2) Patient or caregiver: Willing and able to comply with all study procedures (including filling out questionnaires on bleeds/drugs used)
•3) Diagnosed with congenital hemophilia A and meets either of the following criteria:
•a. Will start ITI after study enrollment and has positive FVIII inhibitor titer ( >= 0.6 BU/mL) as evidenced by most recent titer results within 8 weeks before enrollment
•b. Is already undergoing ITI at study enrollment and has not yet met partial success as evidenced by most recent titer results within 8 weeks before enrollment

•Patients who meet any of the following criteria will be excluded from study entry:
•1) Inherited or acquired bleeding disorder other than congenital hemophilia A
•2) Previous (within the last 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 high risk for thromboembolic diseases(e.g. protein S deficiency)
•3) At high risk for thrombotic microangiopathy (TMA) in the opinion of the investigator based on previous or familial history of TMA (e.g., thrombotic thrombocytopenic purpura [TTP], atypical hemolytic uremic syndrome [aHUS])
•4) Participating or planning to participate in other intervention trials
•5) Otherwise unsuitable for study participation in the opinion of the investigator

发起方

Matsushita Tadashi