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Completed

NCT Number: NCT01213446

Study of Biostate® in Children With Von Willebrand Disease

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.

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Key information

Conditions

Von Willebrand Disease Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Blood Platelet Disorders Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders von Willebrand Diseases

Age range

Up to 12 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Study site, Homyel, Belarus

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Biostate

Biological

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.

Primary outcomes

  1. Haemostatic efficacy

    Time frame: From Day 1 until final study visit

  2. 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

  3. 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

  4. 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

  5. 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

  6. 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

  7. AUC of FVIII

    Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1

  8. 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

  9. 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

  10. 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

  11. 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

  12. 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

  13. 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

  14. 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

  15. 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

  16. 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

  17. 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

  18. 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

  19. 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

Secondary outcomes

  1. Frequency of adverse events (AEs) per infusion

    Time frame: 13 months

  2. Severity of AEs per infusion

    Time frame: 13 months

  3. Severity of AEs per subject

    Time frame: 13 months

  4. Relatedness of AEs per infusion

    Time frame: 13 months

  5. Relatedness of AEs per subject

    Time frame: 13 months

  6. Development of VWF inhibitors

    Time frame: Sample taken at baseline, then every 3 months up to 12 months

  7. Development of FVIII inhibitors

    Time frame: Sample taken at baseline, then every 3 months up to 12 months

  8. Frequency of adverse events (AEs) per subject

    Time frame: 13 months

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Collaborators

  • Parexel

Registry information

Official study 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

Important dates

Study start
2010
Primary completion
2013
Study completion
2013
First posted
Oct 4, 2010
Registry last updated
Oct 3, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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