The Von Willebrand Disease (VWD) International Prophylaxis Study - Full Text View

Sponsor:	Skane University Hospital
Collaborators:	Blood Center of Wisconsin CSL Behring
Information provided by:	Skane University Hospital
ClinicalTrials.gov Identifier:	NCT00557908

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:

Genetics Home Reference related topics: hemophilia MYH9-related disorder von Willebrand disease Help Me Understand Genetics

U.S. FDA Resources

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 ]

Estimated Enrollment:	200
Study Start Date:	June 2007
Estimated Study Completion Date:	December 2012
Estimated Primary Completion Date:	December 2012 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
3 Dose levels one, two and three.	Drug: VWF/FVIII products (see below) Participants enrolled in the prospective phase of the study will undergo an escalation of treatment from one to three dose levels of VWD product. All subjects enrolled will begin treatment on the level one dose and remain on this dose 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.

Arms

Assigned Interventions

3

Dose levels one, two and three.

Drug: VWF/FVIII products (see below)

Participants enrolled in the prospective phase of the study will undergo an escalation of treatment from one to three dose levels of VWD product. All subjects enrolled will begin treatment on the level one dose and remain on this dose 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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00557908

Contacts

Contact: Erik Berntorp, MD, PhD	46-40-332-392	erik.berntorp@med.lu.se
Contact: Thomas Abshire, MD	414-937-6334	thomas.abshire@bcw.edu

Locations

United States, North Carolina
Rho, Inc.	Recruiting
Chapel Hill, North Carolina, United States, 27517
Contact: Sharyne Donfield, PhD 919-595-6225 sdonfield@rhoworld.com
Contact: Joel Bowen, BS 919-595-6470 jbowen@rhoworld.com
Principal Investigator: Sharyne Donfield, PhD
United States, Wisconsin
BloodCenter of Wisconsin	Recruiting
Milwaukee, Wisconsin, United States, 53201-2178
Contact: Thomas Abshire, MD 414-937-6334 thomas.abshire@bcw.edu
Principal Investigator: Thomas Abshire, MD
Sweden
Skåne University Hospital	Recruiting
Malmö, Sweden, SE-20502
Contact: Erik Berntorp, MD 46-40-332-392 erik.berntorp@med.lu.se
Principal Investigator: Erik Berntorp, MD

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

No publications provided

Responsible Party:	Professor Erik Berntorp, Malmö University Hospital
ClinicalTrials.gov Identifier:	NCT00557908 History of Changes
Other Study ID Numbers:	VWD PN
Study First Received:	November 13, 2007
Last Updated:	July 20, 2011
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, Inherited
Blood Coagulation Disorders
Hematologic Diseases

Coagulation Protein Disorders
Blood Platelet Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on February 09, 2012