The Von Willebrand Disease (VWD) International Prophylaxis Study

Von Willebrand Disease Clinical Trial

— VIP  

Official title:

The VWD International Prophylaxis (VIP) Study

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00557908
• Other study ID #: VWD PN
• Status: Completed
• Phase: N/A
• First received: November 13, 2007
• Last updated: October 5, 2017
• Start date: June 2007
• Est. completion date: February 2013

Study information

• Verified date: October 2017
• Source: Skane University Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 105
• Est. completion date: February 2013
• Est. primary completion date: February 2013
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Von Willebrand Disease
• Von Willebrand Diseases

Intervention

Drug:
• VWF/FVIII products
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

Locations

Country	Name	City	State
Sweden	Skåne University Hospital	Malmö
United States	Rho, Inc.	Chapel Hill	North Carolina
United States	BloodCenter of Wisconsin	Milwaukee	Wisconsin

Sponsors (3)

Lead Sponsor	Collaborator
Skane University Hospital	Blood Center of Wisconsin, CSL Behring

Countries where clinical trial is conducted

United States,  Sweden, 

References & Publications (4)

Abshire TC, Federici AB, Alvárez MT, Bowen J, Carcao MD, Cox Gill J, Key NS, Kouides PA, Kurnik K, Lail AE, Leebeek FW, Makris M, Mannucci PM, Winikoff R, Berntorp E; VWD PN. Prophylaxis in severe forms of von Willebrand's disease: results from the von Wi — View Citation

Berntorp E, Abshire T; von Willebrand Disease Prophylaxis Network Steering Committee. The von Willebrand disease prophylaxis network: exploring a treatment concept. J Thromb Haemost. 2006 Nov;4(11):2511-2. — View Citation

Holm E, Abshire TC, Bowen J, Álvarez MT, Bolton-Maggs P, Carcao M, Federici AB, Gill JC, Halimeh S, Kempton C, Key NS, Kouides P, Lail A, Landorph A, Leebeek F, Makris M, Mannucci P, Mauser-Bunschoten EP, Nugent D, Valentino LA, Winikoff R, Berntorp E. Ch — View Citation

Makris M, Federici AB, Mannucci PM, Bolton-Maggs PH, Yee TT, Abshire T, Berntorp E. The natural history of occult or angiodysplastic gastrointestinal bleeding in von Willebrand disease. Haemophilia. 2015 May;21(3):338-42. doi: 10.1111/hae.12571. Epub 2014 — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	von Willebrand Disease associated bleeding frequency		1 year
Secondary	Optimal treatment regimens for joint bleeding, GI bleeding, epistaxis, and menorrhagia		1 year