CLINICAL STUDY TO INVESTIGATE THE EFFICACY, PHARMACOKINETICS, IMMUNOGENICITY AND SAFETY OF WILATE IN SEVERE VON WILLEBRAND DISEASE PATIENTS UNDER THE AGE OF 6 YEARS

Octapharma Pharmazeutika Produktionsges.m.b.H.0 个研究点目标入组 12 人开始时间: 2022年7月12日最近更新: 2023年9月18日

适应症

试验速览

暂无简介。

•Inclusion Criteria:
•1. Patients aged <6 years at the time of screening
•2. Type 3 (at least four patients), severe type 2 (except 2N) or severe type 1 VWD (any of which with VWF:RCo <20%) according to medical history, requiring substitution therapy with a VWF-containing product
•3. Minimum body weight 11.0 kg at the time of screening
•4. Voluntarily given, fully informed written and signed consent obtained before any study-related procedures are conducted (obtained from the patient’s parent(s)/ legal guardian(s))
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 12
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•Exclusion Criteria:
•1. History, or current suspicion of VWF or FVIII inhibitors
•2. Injection of DDAVP or VWF-containing product within 72 hours prior to inclusion
•3. Medical history of a thromboembolic event
•4. Platelet count <100,000/µL at screening (except for VWD type 2B)
•5. Patients receiving, or scheduled to receive, immunosuppressant drugs (other than antiretroviral chemotherapy), such as prednisone (equivalent to >10 mg/day), or similar drugs
•6. Treatment with any investigational medicinal product (IMP) in another interventional clinical study currently or within four weeks before enrolment
•7. Other coagulation disorders or bleeding disorders
•8. Known hypersensitivity to any of the components of the study drug

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