Study of Biostate® in Children With Hemophilia A

CSL Behring

Status and phase

Completed

Phase 3

Conditions

Hemophilia A

Treatments

Biological: Biostate

Study type

Interventional

Funder types

Industry

Identifiers

NCT01229007

CSLCT-BIO-08-53

1495 (Other Identifier)

2009-015112-18 (EudraCT Number)

Details and patient eligibility

About

The objective of this study is to assess the efficacy and safety of a Von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, and to investigate the pharmacokinetics of Biostate in children with haemophilia A.

Enrollment

35 patients

Sex

Male

Ages

Under 12 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Male subjects between 0 and <12 years of age.
Diagnosed with severe haemophilia A (FVIII:C <1%), and pre-treated for a minimum of 20 to 50 exposure days.
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.
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.

Exclusion criteria

For all subjects at Day 1: Are actively bleeding.
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.
Have a known history of, or who are suspected of having FVIII inhibitors.
Have received aspirin or other non-steroidal anti-inflammatory drugs (NSAIDs) within 7 days of administration of the IMP.
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.
Are human immunodeficiency virus [HIV]-1/-2 antibody positive with a viral load of >200/µL.
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.
Suffering from von Willebrand disease (VWD) with von Willebrand factor: ristocetin cofactor (VWF:RCo) level <50 IU/dL at Screening.
Have a known or suspected hypersensitivity or previous evidence of severe side effects to a plasma-derived FVIII product or to human albumin.
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.
Unwillingness and/or inability to comply with the study requirements.