The Von Willebrand Disease (VWD) International Prophylaxis Study (VIP)

This study has been completed.
Sponsor:
Collaborators:
Blood Center of Wisconsin
CSL Behring
Information provided by (Responsible Party):
Sharyne M. Donfield, Ph.D., Skane University Hospital
ClinicalTrials.gov Identifier:
NCT00557908
First received: November 13, 2007
Last updated: March 24, 2014
Last verified: March 2014
  Purpose

The von Willebrand Disease Prophylaxis Network (VWD PN) is an international study group formed with the goal of investigating the role of prophylaxis in clinically severe VWD that is non-responsive to other treatment(s).


Condition Intervention
Von Willebrand Disease
Drug: VWF/FVIII products (see below)

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Prevention
Official Title: The VWD International Prophylaxis (VIP) Study

Resource links provided by NLM:


Further study details as provided by Skane University Hospital:

Primary Outcome Measures:
  • von Willebrand Disease associated bleeding frequency [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Optimal treatment regimens for joint bleeding, GI bleeding, epistaxis, and menorrhagia [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Enrollment: 105
Study Start Date: June 2007
Study Completion Date: February 2013
Primary Completion Date: February 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Infusions per week/menstrual cycle Drug: VWF/FVIII products (see below)

Participants in the prospective phase of the study undergo an escalation of treatment from receipt of one to three levels of VWD product. All subjects enrolled will begin treatment on the level one and remain on this regimen for the duration of follow-up, or until they meet the criteria for escalation to level two or three (if indeed they do meet the criteria.)

Dosing for joint bleeding, epistaxis, and GI bleeding indications: 50 U RCo/kg once per week, 50 U RCo/kg twice per week, or 50 U RCo/kg three times per week.

Dosing for menorrhagia: 50 U RCo/kg on day 1 of menses for 2 cycles, 50 U RCo/kg on days 1 and 2 of menses for 2 cycles, or 50 U RCo/kg on days 1, 2, and 3 of menses

VWF/FVIII products labeled for use in the treatment of VWD will be chosen for use at the discretion of the treating physician. Examples of such products include, but are not limited to, Humate P/Haemate P, Alphanate, Wilate, and Fanhdi.


Detailed Description:

The most common indications for vWD prophylaxis included joint bleeding, epistaxis, gastrointestinal (GI) bleeding, and menorrhagia. Thus, an effort to establish optimal treatment regimens for these indications, through a period of prospective evaluation, is the primary focus of this research. Other goals include a retrospective study of the effect of prophylaxis on bleeding frequency, and a retrospective natural history study of GI bleeding in VWD.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Type 1: eligible for participation if

  • ≤20% RCo and/or ≤20% FVIII; and
  • DDAVP non-responsive, defined as occurrence of bleeding episodes not responding satisfactorily to desmopressin, or deemed non-responsive a priori by the investigator; and
  • Bleeding indication criteria are met

Type 2: eligible for participation if

  • DDAVP non-responsive, defined as occurrence of bleeding episodes not responding satisfactorily to desmopressin, or deemed non-responsive a priori by the investigator; or Type 2B;
  • Bleeding indication criteria are met

Type 3: eligible for participation if

  • Bleeding indication criteria are met

Bleeding Indication Criteria:

  • Joint Bleeding: documentation of at least two apparently spontaneous bleeding episodes in the same joint in the six months prior to enrollment; or three or more apparently spontaneous bleeding episodes in different joints in the six months prior to enrollment.
  • GI Bleeding: history of two or more severe GI bleeding episodes associated with either a drop in hemoglobin of ≥ 2 g/dl or requiring red blood cell transfusion or treatment with VWD concentrate.
  • Failure to identify other causes of bleeding.
  • Menorrhagia: a diagnosis of menorrhagia; prospectively completed Pictorial Blood Assessment Chart score >185 or required treatment with a VWD product for menstrual bleeding on one or more occasions in the year prior to enrollment.
  • Normal cervical cytology (PAP) within the six months prior to enrollment for females ≥ 18 years of age.
  • Epistaxis 1. Three or more bleeding episodes in a six-month period that required treatment with VWD concentrates or red cell transfusions.
  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: NCT00557908

Locations
United States, North Carolina
Rho, Inc.
Chapel Hill, North Carolina, United States, 27517
United States, Wisconsin
BloodCenter of Wisconsin
Milwaukee, Wisconsin, United States, 53201-2178
Sweden
Skåne University Hospital
Malmö, Sweden, SE-20502
Sponsors and Collaborators
Skane University Hospital
Blood Center of Wisconsin
CSL Behring
Investigators
Principal Investigator: Erik Berntorp, MD, PhD Skåne University Hospital, Malmö, Sweden
Principal Investigator: Thomas Abshire, MD Blood Center of Wisconsin
  More Information

Publications:
Responsible Party: Sharyne M. Donfield, Ph.D., Senior Research Scientist, Skane University Hospital
ClinicalTrials.gov Identifier: NCT00557908     History of Changes
Other Study ID Numbers: VWD PN
Study First Received: November 13, 2007
Last Updated: March 24, 2014
Health Authority: Sweden: Medical Products Agency

Keywords provided by Skane University Hospital:
Von Willebrand Disease
VWD
prophylaxis

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

ClinicalTrials.gov processed this record on October 20, 2014