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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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • Number of infusions per bleeding event [ Time Frame: 1 day ] [ Designated as safety issue: No ]
  • Number of infusions per month [ Time Frame: 1 month ] [ Designated as safety issue: No ]
  • Number of infusions per year [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • Dose (IU/kg) per bleeding event [ Time Frame: 1 day ] [ Designated as safety issue: No ]
  • Dose (IU/kg) per month [ Time Frame: 1 month ] [ Designated as safety issue: No ]
  • Dose (IU/kg) per year [ Time Frame: 1 year ] [ Designated as safety issue: No ]
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 ] [ Designated as safety issue: Yes ]
  • Severity of AEs per subject [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
  • Severity of AEs per infusion [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
  • Relatedness of AEs per subject [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
  • Relatedness of AEs per infusion [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
  • Development of FVIII inhibitors [ Time Frame: Samples taken at screening visit, on day 2, on months 1 and 3, and at final visit ] [ Designated as safety issue: Yes ]
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
Endpoint Classification: Safety/Efficacy Study
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.
Male
up to 12 Years
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
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