Study of Biostate® in Children With Von Willebrand Disease

This study has been completed.
Sponsor:
Collaborator:
Parexel
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT01213446
First received: October 1, 2010
Last updated: November 12, 2013
Last verified: November 2013
  Purpose

This is an open-label study to investigate the pharmacokinetics (PK), efficacy, and safety of a von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, in children with Von Willebrand disease (VWD) in whom treatment with a VWF product is required for prophylactic therapy, haemostatic control during surgery, or control of a non-surgical, spontaneous, or traumatic bleeding event.


Condition Intervention Phase
Von Willebrand Disease
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, Multi-centre Study to Assess the Pharmacokinetics, Efficacy, and Safety of Biostate® in Paediatric Subjects With Von Willebrand Disease

Resource links provided by NLM:


Further study details as provided by CSL Behring:

Primary Outcome Measures:
  • Haemostatic efficacy [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: No ]
  • Incremental Recovery of VWF [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose ] [ 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 VWF [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose ] [ 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 VWF [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose ] [ Designated as safety issue: No ]
  • 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 ]
  • Maximum plasma concentration (Cmax) of VWF [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose ] [ 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 VWF [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose ] [ 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 to maximum concentration (tmax) of VWF [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose ] [ Designated as safety issue: No ]
  • Time to maximum concentration (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 ]
  • Mean residence time (MRT) of VWF [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose ] [ 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 ]
  • Clearance (CL) of VWF [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose ] [ Designated as safety issue: No ]
  • Clearance (CL) 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 of steady state (Vss) of VWF [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose ] [ Designated as safety issue: No ]
  • Volume of distribution of 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 ]

Secondary Outcome Measures:
  • Frequency of adverse events (AEs) per infusion [ Time Frame: 13 months ] [ Designated as safety issue: Yes ]
  • Severity of AEs per infusion [ Time Frame: 13 months ] [ Designated as safety issue: Yes ]
  • Severity of AEs per subject [ Time Frame: 13 months ] [ Designated as safety issue: Yes ]
  • Relatedness of AEs per infusion [ Time Frame: 13 months ] [ Designated as safety issue: Yes ]
  • Relatedness of AEs per subject [ Time Frame: 13 months ] [ Designated as safety issue: Yes ]
  • Development of VWF inhibitors [ Time Frame: Sample taken at baseline, then every 3 months up to 12 months ] [ Designated as safety issue: Yes ]
  • Development of FVIII inhibitors [ Time Frame: Sample taken at baseline, then every 3 months up to 12 months ] [ Designated as safety issue: Yes ]
  • Frequency of adverse events (AEs) per subject [ Time Frame: 13 months ] [ Designated as safety issue: Yes ]

Enrollment: 17
Study Start Date: August 2010
Study Completion Date: August 2013
Arms Assigned Interventions
Experimental: Biostate Biological: Biostate

PK component: Single bolus infusion of 80 IU VWF:RCo/kg administered intravenously on Day 1, and approximately Day 180 in Type 3 VWD subjects only.

Efficacy component: Repeated bolus doses over 12 months as required to manage VWD condition.


  Eligibility

Ages Eligible for Study:   up to 12 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male and female subjects between 0 and <12 years of age
  • Diagnosed with VWD Type 1, 2A, or 3
  • Desmopressin acetate (DDAVP) treatment is ineffective, contraindicated, or not available for subject
  • von Willebrand factor: ristocetin cofactor (VWF:RCo) is <20% at screening or the subject has a history of VWF:RCo <10%
  • 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 immunization
  • Written informed consent given

Exclusion Criteria:

  • Active bleeding immediately prior to initial PK period
  • Received treatment with DDAVP or a VWF concentrate product for their VWD in the 5 days prior to their first study treatment
  • Have received aspirin or other non-steroidal anti-inflammatory drugs (NSAIDs) within 7 days of commencing the PK period.
  • Known history or suspicion of having VWF or FVIII inhibitors
  • Acute or chronic medical condition, other than VWD, which may affect the conduct of the study
  • Known or suspected hypersensitivity or previous evidence of severe side effects to other FVIII/VWF concentrates
  • Participation in a clinical study or use of an investigational compound in another study in the 3 months preceding study start
  • 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: NCT01213446

Locations
Belarus
Study site
Homel, Belarus, 246040
Study site
Minsk, Belarus, 223040
Georgia
Study site
Tbilisi, Georgia, 0179
Germany
Study site
Bremen, Germany, 28177
Guatemala
Study site
Guatemala, CP, Guatemala, 01010
Lebanon
Study site
Beirut, Lebanon
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: NCT01213446     History of Changes
Other Study ID Numbers: CSLCT-BIO-08-52, 2009-017753-34, 1494
Study First Received: October 1, 2010
Last Updated: November 12, 2013
Health Authority: Belarus: Ministry of Health
Germany: Paul-Ehrlich-Institut
Guatemala: Ministry of Public Health and Social Assistance
Mexico: Ministry of Health
Georgia: Ministry of Labour Health and Social Affairs of Georgia
Bulgaria: Bulgarian Drug Agency
Ukraine: Ministry of Health

Keywords provided by CSL Behring:
Von Willebrand Disease

Additional relevant MeSH terms:
Von Willebrand Diseases
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Blood Platelet Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on August 20, 2014