Brief Title
Study of Biostate® in Children With Hemophilia A
Official Title
A Phase III, Open-Label, Multicentre Study to Evaluate Efficacy, Pharmacokinetics, and Safety of Biostate® in Paediatric Subjects With Haemophilia A
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 haemophilia A.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Subjective assessment of Haemostatic efficacy
Secondary Outcome
Frequency of adverse events (AEs)
Condition
Hemophilia A
Intervention
Biostate
Study Arms / Comparison Groups
Biostate
Description:
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
35
Start Date
August 2010
Completion Date
July 2014
Primary Completion Date
July 2014
Eligibility Criteria
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.
Gender
Male
Ages
N/A - 12 Years
Accepts Healthy Volunteers
No
Contacts
Program Director Clinical R&D, ,
Location Countries
Belarus
Location Countries
Belarus
Administrative Informations
NCT ID
NCT01229007
Organization ID
CSLCT-BIO-08-53
Secondary IDs
2009-015112-18
Responsible Party
Sponsor
Study Sponsor
CSL Behring
Collaborators
Parexel
Study Sponsor
Program Director Clinical R&D, Study Director, CSL Behring
Verification Date
July 2014