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Study of Biostate® in Children With Hemophilia A

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01229007
First Posted: October 27, 2010
Last Update Posted: August 24, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
Parexel
Information provided by (Responsible Party):
CSL Behring
October 1, 2010
October 27, 2010
August 24, 2017
August 2010
July 2014   (Final data collection date for primary outcome measure)
  • 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: None (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
July 2014   (Final data collection date for primary outcome measure)

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 ( EudraCT Number )
1495 ( Other Identifier: CSL Behring )
Yes
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