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
  Purpose

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.


Condition Intervention Phase
Hemophilia A
Biological: Biostate
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase III, Open-Label, Multicentre Study to Evaluate Efficacy, Pharmacokinetics, and Safety of Biostate® in Paediatric Subjects With Haemophilia A

Resource links provided by NLM:


Further study details as provided by CSL Behring:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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 ]

Enrollment: 35
Study Start Date: August 2010
Study Completion Date: July 2014
Arms Assigned Interventions
Experimental: Biostate 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.

  Eligibility

Ages Eligible for Study:   up to 12 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01229007

Locations
Belarus
Study site
Gomel, Belarus, 246040
Study site
Minsk, Belarus, 223040
Georgia
Study Site
Tbilisi, Georgia, 0179
Guatemala
Study Site
Guatemala, Guatemala, 01010
Lebanon
Study Site
Beirut, Lebanon
Mexico
Study site
Monterrey, Mexico, 64000
Ukraine
Study Site
Dnipropetrovsk, Ukraine
Study Site
Lviv, Ukraine
Sponsors and Collaborators
CSL Behring
Parexel
Investigators
Study Director: Program Director Clinical R&D CSL Behring
  More Information

Additional Information:
No publications provided

Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT01229007     History of Changes
Other Study ID Numbers: CSLCT-BIO-08-53, 2009-015112-18, 1495
Study First Received: October 1, 2010
Last Updated: July 8, 2014
Health Authority: Belarus: Ministry of Health
Guatemala: Ministry of Public Health and Social Assistance
Mexico: Ministry of Health
Georgia: Ministry of Health Labour and Social Affairs of Georgia
Bulgaria: Bulgarian Drug Agency

Keywords provided by CSL Behring:
Hemophilia A
Children

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on October 01, 2014