A Phase III Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy, and Safety of Biostate® in Paediatric Subjects with von Willebrand Disease.

CSL Behring GmbH0 个研究点目标入组 12 人开始时间: 2010年1月15日最近更新: 2014年1月27日

适应症

试验速览

暂无简介。

•1. Male and female subjects between 0 and <12 years of age.
•2. Have been diagnosed with VWD Type 1, 2A, or 3.
•3. Where desmopressin acetate (DDAVP) treatment is ineffective, contraindicated, or not available.
•4. VWF: ristocetin cofactor [RCo] is <20% at Screening (after a minimum of 5 days since last VWF treatment) or the subject has a history of VWF:RCo <10% documented in their medical notes at enrolment. Children < 1 year of age may be included in the study without evidence of vaccination against hepatitis A.
•5. Have evidence of vaccination against hepatitis A and B or presence of antibodies against hepatitis A and B due to either a previous infection or prior immunisation as documented in the medical notes at Screening. Children < 1 year of age may be included in the study without evidence of vaccination against hepatitis A.
•6. The subject and/or legal guardian understand(s) the nature of the study and has/have given written informed consent to participate in the study and is/are willing to comply with the protocol.
•Are the trial subjects under 18? yes
•Number of subjects for this age range:
•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

•1. Are actively bleeding immediately prior to initial PK period.
•2. Have received treatment with DDAVP or a VWF concentrate product for their VWD in the 5 days prior to their first dose of the IMP.
•3. Have received aspirin or other non-steroidal anti-inflammatory drugs (NSAIDs) within 7 days of commencing the PK period.
•4. Have a known history of, or are suspected to have VWF or FVIII inhibitors.
•5. Suffering an acute or chronic medical condition, other than VWD, which may in the opinion of the Investigator, affect the conduct of the study.
•6. Have a known or suspected hypersensitivity or previous evidence of severe side effects to FVIII/VWF concentrates like Biostate.
•7. Have participated in a clinical study or used an investigational compound in another study (eg, a new chemical entity not registered for clinical use) in the 3 months preceding the first day of IMP administration, or are planning to enter such a study during the study period.
•8. Are not willing and/or not able to comply with the study requirements.

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