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Pharmacokinetics, Safety and Efficacy of Recombinant Von Willebrand Factor (rVWF) in the Treatment of Bleeding Episodes in Von Willebrand Disease (VWD)

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Baxter Innovations GmbH
Information provided by (Responsible Party):
Baxter Healthcare Corporation
ClinicalTrials.gov Identifier:
NCT01410227
First received: August 4, 2011
Last updated: February 20, 2013
Last verified: February 2013
History of Changes
  Purpose

The purpose of this Phase 3 study is to assess the pharmacokinetics of rVWF:rFVIII and rVWF, and to assess the safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding events in subjects with severe hereditary von Willebrand disease (VWD).


Condition Intervention Phase
Von Willebrand Disease
 Biological: Recombinant von Willebrand factor (rVWF) with or without rFVIII (Advate)
 Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 3 Clinical Study to Determine the Pharmacokinetics, Safety and Efficacy of Recombinant Von Willebrand Factor : Recombinant Factor VIII (rVWF:rFVIII) and rVWF in the Treatment of Bleeding Episodes in Subjects Diagnosed With Von Willebrand Disease

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Baxter Healthcare Corporation:

Primary Outcome Measures:
  • Number of subjects with treatment success for treated bleeding episodes [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    Treatment success will be defined as a mean efficacy rating score of < 2.5 for a subject´s bleeding episodes treated with the investigational product while in a treatment period. Scores used to assess the extent of control of the bleeding episodes: Excellent = 1, Good = 2, Moderate = 3, None = 4.


Estimated Enrollment: 36
Study Start Date: September 2011
Estimated Study Completion Date: March 2014
Estimated Primary Completion Date: March 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: PK 80 Arm (minimum of 22 subjects with severe VWD)
PK assessment (80 IU/kg rVWF) + 12-month treatment period
 Biological: Recombinant von Willebrand factor (rVWF) with or without rFVIII (Advate)
Intravenous administration
Experimental: PK 50 Arm (14 subjects with type 3 VWD)
Two single-blinded PK assessments (50 IU/kg rVWF + rFVIII/placebo) + 12-month treatment period
 Biological: Recombinant von Willebrand factor (rVWF) with or without rFVIII (Advate)
Intravenous administration
Experimental: PK 50 Only Arm (minimum of 7 subjects with type 3 VWD)
PK assessment (50 IU/kg rVWF) only, no treatment of bleeding episodes
 Biological: Recombinant von Willebrand factor (rVWF) with or without rFVIII (Advate)
Intravenous administration
Experimental: Treatment Only (up to 7 subjects independent of VWD subtype)
Treatment of bleeding episodes for a total of 12 months
 Biological: Recombinant von Willebrand factor (rVWF) with or without rFVIII (Advate)
Intravenous administration

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Selected Inclusion Criteria:

  • The subject has been diagnosed with type 3 von Willebrand disease (VWD:Ag <= 3 IU/dl) or severe non-type 3 VWD (VWF:RCo < 20 IU/dL) or type 2N VWD (FVIII:C <10% and historically documented genetics)
  • The subject, who participates for the treatment of bleeding episodes, has had a minimum of 6 documented bleeds (medical history) requiring VWF coagulation factor replacement therapy during the previous 3 years prior to enrollment
  • The subject is at least 18 and not older than 65 years of age at enrollment

Selected Exclusion Criteria:

  • The subject has been diagnosed with pseudo VWD or another hereditary or acquired coagulation disorder other than VWD (e.g., qualitative and quantitative platelet disorders or elevated PT/international normalized ratio [INR] > 1.4)
  • The subject has a documented history of a VWF:RCo half-life of < 6 hours
  • The subject has a history or presence of a VWF inhibitor at screening
  • The subject has a history or presence of a factor VIII (FVIII) inhibitor with a titer >= 0.4 BU (by Nijmegen assay) or >= 0.6 BU (by Bethesda assay)
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01410227

  Show 52 Study Locations
Sponsors and Collaborators
Baxter Healthcare Corporation
Baxter Innovations GmbH
Investigators
Study Director: Benny Sorensen, MD Baxter Healthcare Corporation
  More Information

No publications provided

Responsible Party: Baxter Healthcare Corporation
ClinicalTrials.gov Identifier: NCT01410227     History of Changes
Other Study ID Numbers: 071001, 2010-024108-84
Study First Received: August 4, 2011
Last Updated: February 20, 2013
Health Authority: Australia: Department of Health and Ageing Therapeutic Goods Administration
Austria: Agency for Health and Food Safety
Belgium: Federal Agency for Medicinal Products and Health Products
Bulgaria: Bulgarian Drug Agency
Canada: Health Canada
Germany: Paul-Ehrlich-Institut
India: Drugs Controller General of India
Italy: The Italian Medicines Agency
Japan: Pharmaceuticals and Medical Devices Agency
Netherlands: Medicines Evaluation Board (MEB)
Spain: Agencia Española de Medicamentos y Productos Sanitarios
Sweden: Medical Products Agency
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United States: Food and Drug Administration

Additional relevant MeSH terms:
Hemorrhage
Von Willebrand Diseases
Pathologic Processes
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 May 19, 2013