The Von Willebrand Disease (VWD) International Prophylaxis Study
NCT ID: NCT00557908
Last Updated: 2017-10-06
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
105 participants
OBSERVATIONAL
2007-06-30
2013-02-28
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
OTHER
Study Groups
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VWF/FVIII product infusions
One to three infusions of factor replacement as needed to control bleeding.
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
Interventions
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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
Eligibility Criteria
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Inclusion Criteria
* ≤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.
ALL
No
Sponsors
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Versiti
OTHER
CSL Behring
INDUSTRY
Skane University Hospital
OTHER
Responsible Party
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Sharyne M. Donfield, Ph.D.
Principal Research Scientist
Principal Investigators
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Erik Berntorp, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Skåne University Hospital, Malmö, Sweden
Thomas Abshire, MD
Role: PRINCIPAL_INVESTIGATOR
Versiti
Locations
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Rho, Inc.
Chapel Hill, North Carolina, United States
BloodCenter of Wisconsin
Milwaukee, Wisconsin, United States
Skåne University Hospital
Malmo, , Sweden
Countries
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References
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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.
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.
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.
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.
Other Identifiers
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VWD PN
Identifier Type: -
Identifier Source: org_study_id