Von Willebrand DiseaseVIPNCT00557908
The Von Willebrand Disease (VWD) International Prophylaxis Study
An observational study of Von Willebrand Disease, run by Skane University Hospital at 3 sites in 2 countries.
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). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Von Willebrand Disease
- What is being tested?
- VWF/FVIII products
- Type of treatment
- Injection or infusion
- How many people
- 105 participants
- Where
- 3 sitesin 2 countries
- Study dates
- Jun 2007to Feb 2013
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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;
Show all 16Show fewer
- 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.
Who cannot join
None listed.
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
United States2 sites
Rho, Inc.
Chapel Hill, North Carolina, 27517
BloodCenter of Wisconsin
Milwaukee, Wisconsin, 53201-2178
About this study
Official title: The VWD International Prophylaxis (VIP) Study
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).
Read the 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.
- Conditions
- Von Willebrand Disease
- Being tested
- VWF/FVIII products
- Keywords
- Von Willebrand Disease, VWD, prophylaxis
- Medical subject headings
- von Willebrand Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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
| Study group | Role | What they receive |
|---|---|---|
| VWF/FVIII product infusions | One to three infusions of factor replacement as needed to control bleeding. Receives: Drug: VWF/FVIII products |
What is being measured
The primary outcome is the main question the study is designed to answer.
von Willebrand Disease associated bleeding frequency
Measured: 1 year
Secondary outcomes (1)
Optimal treatment regimens for joint bleeding, GI bleeding, epistaxis, and menorrhagia
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Nov 2007 (estimated)
- Study startJun 2007
- Primary completionFeb 2013
- Study completionFeb 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Von Willebrand DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 105 participants (actual)
Sponsor and investigators
- Lead sponsor
- Skane University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Versiti Blood Health, CSL Behring
- Responsible party
- Principal investigator, Sharyne M. Donfield, Ph.D., Skane University Hospital
- Investigators
- Erik Berntorp, MD, PhD, Principal investigator, Skåne University Hospital, Malmö, SwedenThomas Abshire, MD, Principal investigator, Versiti Blood Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00557908
- Sponsor's protocol ID
- VWD PN
- Acronym
- VIP
- Status last verified
- Oct 2017
- First submitted
- 13 Nov 2007
- Last updated
- 6 Oct 2017
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
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.
PubMed 25688461Makris 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.
PubMed 25381842Abshire 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.
PubMed 22823000Berntorp 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.
PubMed 17059476
Study at a glance
From the registry record, last updated by the sponsor on 6 Oct 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The VWD International Prophylaxis (VIP) Study
- ClinicalTrials.gov ID
- NCT00557908
- Acronym / sponsor ID
- VIP / VWD PN
- Conditions
- Von Willebrand Disease
- Keywords
- Von Willebrand Disease, VWD, prophylaxis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 105 participants (actual)
- Lead sponsor
- Skane University HospitalUniversity, hospital or other
- Collaborators
- Versiti Blood Health, CSL Behring
- Study start
- Jun 2007
- Primary completion
- Feb 2013 (actual)
- Study completion
- Feb 2013 (actual)
- First posted
- 14 Nov 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
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.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VWF/FVIII product infusions | – | Drug: VWF/FVIII productsOne to three infusions of factor replacement as needed to control bleeding. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (16 listed)
- 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
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VWF/FVIII productsParticipants 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 | Drug | VWF/FVIII product infusions |
Primary outcome
von Willebrand Disease associated bleeding frequency
Time frame: 1 year
1 secondary outcome
Optimal treatment regimens for joint bleeding, GI bleeding, epistaxis, and menorrhagia
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Oct 2017.
- Results publication: PubMed 25688461Holm 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.
- Results publication: PubMed 25381842Makris 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.
- Results publication: PubMed 22823000Abshire 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.
- Results publication: PubMed 17059476Berntorp 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.
- Individual participant dataSharing planned: No
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Von Willebrand Diseases
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Erik Berntorp, MD, PhD principal investigator
Skåne University Hospital, Malmö, Sweden
Thomas Abshire, MD principal investigator
Versiti Blood Health
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Von Willebrand Disease, run by Skane University Hospital at 3 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Oct 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
von Willebrand Disease associated bleeding frequency (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00557908, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Oct 2017.