This clinical study investigates the effectiveness and safety of Biostate in children with von Willebrand's disease (VWD), a blood clotting disorder. A two day investigation of the pharmacokinetics (metabolism of Biostate by the child's body) is a part of the investigation – 0.5, 4, 8, 12, 24 and 48h after administration.

Phase 1

• Conditions: Von Willebrand Disease; MedDRA version: 14.0Level: PTClassification code 10047715Term: Von Willebrand's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2009-017753-34-BG
• Lead Sponsor: CSL Behring GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-10-13
• Last Update Posted: 2 October 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

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

Exclusion Criteria

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified