The Von Willebrand Disease (VWD) International Prophylaxis Study

Completed

• Conditions: Von Willebrand Disease
• Interventions: Drug: VWF/FVIII products

• Registration Number: NCT00557908
• Lead Sponsor: Skane University Hospital

Brief Summary

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).

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 Timeline

• Study Start: 2007-06
• Study First Submitted: 2007-11-13
• Study First Submitted QC: 2007-11-13
• Study First Posted: 2007-11-14
• Primary Completion: 2013-02
• Study Completion: 2013-02
• Status Verified: 2017-10
• Last Update Submitted: 2017-10-05
• Last Update Posted: 2017-10-06

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 105

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.

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
von Willebrand Disease associated bleeding frequency	1 year

Secondary Outcome Measures

Name	Time	Method
Optimal treatment regimens for joint bleeding, GI bleeding, epistaxis, and menorrhagia	1 year

Trial Locations

Locations (3)

Skåne University Hospital; 🇸🇪 Malmö, Sweden

Rho, Inc.; 🇺🇸 Chapel Hill, North Carolina, United States

BloodCenter of Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States