Status:
COMPLETED
Study of Biostate® in Children With Hemophilia A
Lead Sponsor:
CSL Behring
Collaborating Sponsors:
Parexel
Conditions:
Hemophilia A
Eligibility:
MALE
Up to 12 years
Phase:
PHASE3
Brief Summary
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 haem...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
August 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2014
Estimated Enrollment :
35 Patients enrolled
Trial Details
Trial ID
NCT01229007
Start Date
August 1 2010
End Date
July 1 2014
Last Update
August 24 2017
Active Locations (8)
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1
Study site
Homyel, Belarus, 246040
2
Study site
Minsk, Belarus, 223040
3
Study Site
Tbilisi, Georgia, 0179
4
Study Site
Guatemala City, Guatemala, 01010