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

October 5, 2017 updated by: Sharyne M. Donfield, Ph.D., Skane University Hospital

The VWD International Prophylaxis (VIP) Study

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).

Study Overview

Status

Completed

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Actual)

105

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

      • Malmö, Sweden, SE-20502
        • Skane University Hospital
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27517
        • Rho, Inc.
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53201-2178
        • BloodCenter of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

The VWD population includes those with Type 1 if </=20% RCo and/or </=20% FVIII; and DDAVP non-responsive; Type 2 if DDAVP non-responsive, or Type 2B; and Type 3; who meet bleeding indication criteria having defined patterns of gastrointestinal bleeding, joint bleeding, epistaxis, or menorrhagia.

Individuals already on prophylaxis for VWD, for any indication, and individuals who were on a regimen of prophylaxis for at least six months that was discontinued because it was no longer required, or those with a history of GI bleeding due either to proven angiodysplasia or unexplained by other factors.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Other

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
VWF/FVIII product infusions
One to three infusions of factor replacement as needed to control bleeding.

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
von Willebrand Disease associated bleeding frequency
Time Frame: 1 year
1 year

Secondary Outcome Measures

Outcome Measure
Time Frame
Optimal treatment regimens for joint bleeding, GI bleeding, epistaxis, and menorrhagia
Time Frame: 1 year
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Collaborators

Investigators

  • Principal Investigator: Erik Berntorp, MD, PhD, Skåne University Hospital, Malmö, Sweden
  • Principal Investigator: Thomas Abshire, MD, Versiti

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2007

Primary Completion (Actual)

February 1, 2013

Study Completion (Actual)

February 1, 2013

Study Registration Dates

First Submitted

November 13, 2007

First Submitted That Met QC Criteria

November 13, 2007

First Posted (Estimate)

November 14, 2007

Study Record Updates

Last Update Posted (Actual)

October 6, 2017

Last Update Submitted That Met QC Criteria

October 5, 2017

Last Verified

October 1, 2017

More Information

Terms related to this study

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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