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

This study has been completed.
Sponsor:
Collaborator:
Parexel
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT00941616
First received: July 15, 2009
Last updated: April 4, 2013
Last verified: April 2013
  Purpose

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.


Condition Intervention Phase
Von Willebrand Disease
Biological: Biostate®
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy and Safety of Biostate® in Subjects With Von Willebrand Disease.

Resource links provided by NLM:


Further study details as provided by CSL Behring:

Primary Outcome Measures:
  • Haemostatic efficacy at time of non-surgical bleeding (NSB) event [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: No ]
  • Haemostatic efficacy overall [ Time Frame: Monthly (prophylactic therapy) or once every 3 months (for on-demand use) ] [ Designated as safety issue: No ]
  • Number of treatments with blood product transfusions required to resolve any bleeding event [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • Assessment of blood loss during any surgical procedure [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: No ]
  • Number of spontaneous or traumatic NSB events [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: No ]
  • Pharmacokinetic parameters for vWF and FVIII (PK arm only) [ Time Frame: Up to 72 hours following infusions on Day 1 and approximately Day 180 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Development of FVIII inhibitors [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: Yes ]
  • Development of vWF inhibitors [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: Yes ]

Enrollment: 22
Study Start Date: June 2009
Study Completion Date: February 2012
Arms Assigned Interventions
Experimental: PK
Includes subjects participating in the pharmacokinetic component of the study.
Biological: Biostate®
80 IU vWF/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180
Other Name: Human Coagulation Factor VIII / von Willebrand Factor
Experimental: Prophylaxis
Includes subjects receiving 12 months of prophylactic therapy.
Biological: Biostate®
Frequency and dose will be determined by the Investigator based on the subjects clinical condition, previous VWF concentrate requirements, response to therapy, weight and reason for usage.
Other Name: Human Coagulation Factor VIII / von Willebrand Factor
Experimental: On-demand
Includes subjects receiving 12 months of on-demand treatment.
Biological: Biostate®
Frequency and dose will be determined by the Investigator based on the subjects clinical condition, previous VWF concentrate requirements, response to therapy, weight and reason for usage.
Other Name: Human Coagulation Factor VIII / von Willebrand Factor
Experimental: Cross-over to prophylaxis
Includes subjects completing 12 months of on-demand treatment (the "On-demand" arm) who cross-over to prophylactic therapy for an additional 12-month period.
Biological: Biostate®
Frequency and dose will be determined by the Investigator based on the subjects clinical condition, previous VWF concentrate requirements, response to therapy, weight and reason for usage.
Other Name: Human Coagulation Factor VIII / von Willebrand Factor

  Eligibility

Ages Eligible for Study:   12 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  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: NCT00941616

Locations
Bulgaria
Study Site
Sofia, Bulgaria
Poland
Study Site
Warsaw, Poland
Study Site
Wroclaw, Poland
Russian Federation
Study Site
Barnaul, Russian Federation
Ukraine
Study Site
Lviv, Ukraine, 79044
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: NCT00941616     History of Changes
Other Study ID Numbers: CSLCT-BIO-08-54, 1481, 2008-004922-18
Study First Received: July 15, 2009
Last Updated: April 4, 2013
Health Authority: Russia: Ministry of Health of the Russian Federation
Bulgaria: Bulgarian Drug Agency
Brazil: National Health Surveillance Agency
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Ukraine: State Pharmacological Center - Ministry of Health

Keywords provided by CSL Behring:
Von Willebrand Disease

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

ClinicalTrials.gov processed this record on October 23, 2014