- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00557908
The Von Willebrand Disease (VWD) International Prophylaxis Study (VIP)
The VWD International Prophylaxis (VIP) Study
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Actual)
Contacts and Locations
Study Locations
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Malmö, Sweden, SE-20502
- Skane University Hospital
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North Carolina
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Chapel Hill, North Carolina, United States, 27517
- Rho, Inc.
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Wisconsin
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Milwaukee, Wisconsin, United States, 53201-2178
- BloodCenter of Wisconsin
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Genders Eligible for Study
Sampling Method
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.
Description
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.
Study Plan
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Other
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
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VWF/FVIII product infusions
One to three infusions of factor replacement as needed to control bleeding.
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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 |
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
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von Willebrand Disease associated bleeding frequency
Time Frame: 1 year
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1 year
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
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Optimal treatment regimens for joint bleeding, GI bleeding, epistaxis, and menorrhagia
Time Frame: 1 year
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1 year
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Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Erik Berntorp, MD, PhD, Skåne University Hospital, Malmö, Sweden
- Principal Investigator: Thomas Abshire, MD, Versiti
Publications and helpful links
General Publications
- 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.
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- VWD PN
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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