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Completed

NCT Number: NCT00941616

Study of a pd VWF/FVIII Concentrate, Biostate®, in Subjects With Von Willebrand Disease

The aim of this study is to assess the pharmacokinetics (PK), efficacy, and safety of Biostate® in subjects with Von Willebrand Disease (VWD).

Pharmacokinetic Component:

PK parameters will be determined from a subgroup of subjects. Subjects who complete the PK component will subsequently continue in the efficacy component of the study, either continuing on a previously established prophylaxis regimen or continuing to receive on-demand treatment with the occurrence of non-surgical bleeding (NSB) events.

Efficacy Component:

Three treatment arms are defined for the efficacy component of the study. (1) Subjects who are currently being treated on a set prophylaxis regimen with a VWF product at the time of study entry will be enrolled in the "Prophylaxis" arm. (2) Subjects not being treated on a set prophylaxis regimen at the time of study entry who require a VWF product for the treatment of NSB events will be enrolled in the "On-demand" arm and commence using Biostate in the treatment of NSB events. (3) Subjects enrolled in the "On-demand" arm have the possibility to enter the "Cross-over to Prophylaxis" arm to receive an additional 12 months of prophylactic treatment.

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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

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Study Site, Sofia, Bulgaria

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Diagnosed with VWD
  • Desmopressin acetate (DDAVP) treatment is ineffective or contraindicated or not available
  • Evidence of vaccination against hepatitis A and B (or presence of antibodies against hepatitis A and B) within 10 years prior to their first dose of Biostate®
  • Written informed consent given

Exclusion criteria

(for participation in the PK component):

  • Actively bleeding immediately prior to initial PK period
  • Have received DDAVP or a VWF product in the 5 days prior to their first dose of study product
  • Have Type 2B, 2N or 2M VWD

Exclusion criteria

(for all subjects):

  • Requiring a VWF product for a planned surgical procedure at enrolment
  • Have received aspirin or other non-steroidal anti-inflammatory drugs within 7 days prior to their first dose of study product
  • Known history of, or are suspected to have, VWF or FVIII inhibitors
  • Suffering an 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 Biostate®, VWF/FVIII concentrates, or human albumin
  • Impaired liver function at screening
  • Evidence or a history (within the previous 12 months) of abuse of any drug substance, licit or illicit
  • Participation in a clinical study or use of an investigational compound in the 3 months preceding the first day of study drug administration, or plans to enter such a study during the study period.
  • Females who are pregnant, breast-feeding or who have a positive pregnancy test at screening

Treatment and study plan

Biostate®

Biological

80 IU vWF/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180

Other names: Human Coagulation Factor VIII / von Willebrand Factor

Primary outcomes

  1. Haemostatic efficacy at time of non-surgical bleeding (NSB) event

    Time frame: From Day 1 until final study visit

  2. Haemostatic efficacy overall

    Time frame: Monthly (prophylactic therapy) or once every 3 months (for on-demand use)

  3. Number of treatments with blood product transfusions required to resolve any bleeding event

    Time frame: From Day 1 until final study visit

  4. vWF/FVIII concentrate usage (number of infusions, IU/kg per dose, per event, per month and per year)

    Time frame: From Day 1 until final study visit

  5. Assessment of blood loss during any surgical procedure

    Time frame: From Day 1 until final study visit

  6. Number of spontaneous or traumatic NSB events

    Time frame: From Day 1 until final study visit

  7. Pharmacokinetic parameters for vWF and FVIII (PK arm only)

    Time frame: Up to 72 hours following infusions on Day 1 and approximately Day 180

Secondary outcomes

  1. Development of FVIII inhibitors

    Time frame: From Day 1 until final study visit

  2. Development of vWF inhibitors

    Time frame: From Day 1 until final study visit

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Collaborators

  • Parexel

Registry information

Official study title

An Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy and Safety of Biostate® in Subjects With Von Willebrand Disease.

Important dates

Study start
2009
Primary completion
2012
Study completion
2012
First posted
Jul 17, 2009
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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