NCT00557908

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).

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
105

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Jun 2007

Longer than P75 for all trials

Geographic Reach
2 countries

3 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2007

Completed
6 months until next milestone

First Submitted

Initial submission to the registry

November 13, 2007

Completed
1 day until next milestone

First Posted

Study publicly available on registry

November 14, 2007

Completed
5.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2013

Completed
Last Updated

October 6, 2017

Status Verified

October 1, 2017

Enrollment Period

5.7 years

First QC Date

November 13, 2007

Last Update Submit

October 5, 2017

Conditions

Von Willebrand Disease

Keywords

Von Willebrand DiseaseVWDprophylaxis

Outcome Measures

Primary Outcomes (1)

  • von Willebrand Disease associated bleeding frequency

    1 year

Secondary Outcomes (1)

  • Optimal treatment regimens for joint bleeding, GI bleeding, epistaxis, and menorrhagia

    1 year

Study Arms (1)

VWF/FVIII product infusions

One to three infusions of factor replacement as needed to control bleeding.

Drug: VWF/FVIII products

Interventions

VWF/FVIII productsDRUG

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 product infusions

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-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.

You may qualify if:

  • 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.
  • +1 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Rho, Inc.

Chapel Hill, North Carolina, 27517, United States

Location

BloodCenter of Wisconsin

Milwaukee, Wisconsin, 53201-2178, United States

Location

Skåne University Hospital

Malmo, SE-20502, Sweden

Location

Related Publications (4)

  • Holm E, Abshire TC, Bowen J, Alvarez 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. Changes in bleeding patterns in von Willebrand disease after institution of long-term replacement therapy: results from the von Willebrand Disease Prophylaxis Network. Blood Coagul Fibrinolysis. 2015 Jun;26(4):383-8. doi: 10.1097/MBC.0000000000000257.

    PMID: 25688461RESULT

  • Makris M, Federici AB, Mannucci PM, Bolton-Maggs PHB, Yee TT, Abshire T, Berntorp E. The natural history of occult or angiodysplastic gastrointestinal bleeding in von Willebrand disease. Haemophilia. 2015 May;21(3):338-342. doi: 10.1111/hae.12571. Epub 2014 Nov 7.

    PMID: 25381842RESULT

  • Abshire TC, Federici AB, Alvarez 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 Willebrand Disease Prophylaxis Network (VWD PN). Haemophilia. 2013 Jan;19(1):76-81. doi: 10.1111/j.1365-2516.2012.02916.x. Epub 2012 Jul 23.

    PMID: 22823000RESULT

  • 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. doi: 10.1111/j.1538-7836.2006.02179.x. No abstract available.

    PMID: 17059476RESULT

MeSH Terms

Conditions

von Willebrand Diseases

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersBlood Platelet DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Erik Berntorp, MD, PhD

    Skåne University Hospital, Malmö, Sweden

    PRINCIPAL INVESTIGATOR

  • Thomas Abshire, MD

    Versiti Blood Health

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
OTHER
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Research Scientist

Study Record Dates

First Submitted

November 13, 2007

First Posted

November 14, 2007

Study Start

June 1, 2007

Primary Completion

February 1, 2013

Study Completion

February 1, 2013

Last Updated

October 6, 2017

Record last verified: 2017-10

Data Sharing

IPD Sharing
Will not share

Locations

SE(1)US(2)