Study of Biostate® in Children With Hemophilia A

This study has been completed.
Sponsor:
Collaborator:
Parexel
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT01229007
First received: October 1, 2010
Last updated: July 8, 2014
Last verified: July 2014

October 1, 2010
July 8, 2014
August 2010
Not Provided
  • Subjective assessment of Haemostatic efficacy [ Time Frame: Over minimum of 50 exposure days ]
  • Incremental recovery of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ]
  • Half-life of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ]
  • Area under the concentration curve (AUC) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ]
  • Mean residence time (MRT) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ]
  • Volume of distribution at steady state (Vss) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ]
  • Maximum Plasma Concentration (Cmax) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ]
  • Minimum Plasma Concentration (Cmin) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ]
  • Time the maximum concentration occurs (tmax) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ]
  • Total clearance of the drug from the body (CL=dose/AUC) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ]
  • Number of infusions per bleeding event [ Time Frame: 1 day ]
  • Number of infusions per month [ Time Frame: 1 month ]
  • Number of infusions per year [ Time Frame: 1 year ]
  • Dose (IU/kg) per bleeding event [ Time Frame: 1 day ]
  • Dose (IU/kg) per month [ Time Frame: 1 month ]
  • Dose (IU/kg) per year [ Time Frame: 1 year ]
Same as current
Complete list of historical versions of study NCT01229007 on ClinicalTrials.gov Archive Site
  • Frequency of adverse events (AEs) [ Time Frame: 6 months ]
  • Severity of AEs per subject [ Time Frame: 6 months ]
  • Severity of AEs per infusion [ Time Frame: 6 months ]
  • Relatedness of AEs per subject [ Time Frame: 6 months ]
  • Relatedness of AEs per infusion [ Time Frame: 6 months ]
  • Development of FVIII inhibitors [ Time Frame: Samples taken at screening visit, on day 2, on months 1 and 3, and at final visit ]
Same as current
Not Provided
Not Provided
 
Study of Biostate® in Children With Hemophilia A
A Phase III, Open-Label, Multicentre Study to Evaluate Efficacy, Pharmacokinetics, and Safety of Biostate® in Paediatric Subjects With Haemophilia A
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.
Not Provided
Interventional
Phase 3
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Hemophilia A
Biological: Biostate
1 dose of 50 IU FVIII/kg body weight of Biostate administered intravenously on Day 1 in the PK component, followed by the Efficacy component for continuation of Biostate therapy, as required for a minimum of 50 exposure days.
Experimental: Biostate
Intervention: Biological: Biostate
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
35
July 2014
Not Provided

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.
Sexes Eligible for Study: Male
up to 12 Years   (Child)
No
Contact information is only displayed when the study is recruiting subjects
Belarus,   Georgia,   Guatemala,   Lebanon,   Mexico,   Ukraine
 
 
NCT01229007
CSLCT-BIO-08-53, 2009-015112-18, 1495
Yes
Not Provided
Not Provided
Not Provided
CSL Behring
CSL Behring
Parexel
Study Director: Program Director Clinical R&D CSL Behring
CSL Behring
July 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP