This clinical study investigates the effectiveness and safety of Biostate in children with Haemophilia A, a blood clotting disorder. An up to 3 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.

• Conditions: Haemophilia A; MedDRA version: 15.0Level: LLTClassification code 10018937Term: Haemophilia ASystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2009-015112-18-BG
• Lead Sponsor: CSL Behring GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-10-26
• Last Update Posted: 2 March 2015

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 24

Inclusion Criteria

1. Male subjects between 0 and <12 years of age.
2. Have been diagnosed with severe haemophilia A (FVIII:C <1%), and pre-treated for a minimum of 20 to 50 exposure days.
3. 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 enrolment. Children < 1 year of age may be included in the study without evidence of vaccination against hepatitis A.
4. 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: 24
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. For all subjects at Day 1:Are actively bleeding.
2. Have received an infusion of any FVIII product, cryoprecipitate, whole blood, plasma or desmopressin acetate in the 4 days prior to their dosing within the PK component.
3. Have a known history of, or who are suspected of having FVIII inhibitors.
4. Have received aspirin or other non-steroidal anti-inflammatory drugs (NSAIDs) within 7 days of administration of the IMP.
5. Have an impaired liver function ie, bilirubin >1.5 x upper limit of normal (ULN) and/or aspartate/alanine aminotransferase (AST/ALT) >2.5 x ULN (referring to limits of the laboratory that performs the determination) at Screening.
6. Are human immunodeficiency virus [HIV]-1/-2 antibody positive with a viral load of >200/µL.
7. Suffer from an acute or chronic medical condition, other than haemophilia A, which may, in the opinion of the Investigator, affect the conduct of the study.
8. Suffering from von Willebrand disease (VWD) with von Willebrand factor: ristocetin cofactor (VWF:RCo) level <50 IU/dL at Screening.
9. Have a known or suspected hypersensitivity or previous evidence of severe side effects to a plasma-derived FVIII product or to human albumin.
10. 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.
11. Unwillingness and/or inability to comply with the study requirements.

Study & Design

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