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Von Willebrand Disease in the Netherlands (WiN-Pro)

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ClinicalTrials.gov Identifier: NCT03521583
Recruitment Status : Not yet recruiting
First Posted : May 10, 2018
Last Update Posted : May 23, 2018
Sponsor:
Collaborators:
Stichting Haemophilia (Dutch Haemophilia Foundation)
CSL Behring
Shire
Information provided by (Responsible Party):
Frank W.G. Leebeek, MD, PhD, Erasmus Medical Center

Tracking Information
First Submitted Date April 30, 2018
First Posted Date May 10, 2018
Last Update Posted Date May 23, 2018
Estimated Study Start Date October 2018
Estimated Primary Completion Date January 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: April 30, 2018)
Bleeding rate [ Time Frame: 2 years ]
Number of bleedings in an individual divided by the follow-up duration
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT03521583 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Von Willebrand Disease in the Netherlands
Official Title Von Willebrand Disease in the Netherlands - Prospective Study (WiN-Pro)
Brief Summary The primary aim of this study is to prospectively investigate the current bleeding tendency of children and adults with VWD.
Detailed Description

Von Willebrand disease (VWD) is the most common inherited bleeding disorder, and is characterized by a defective platelet adhesion and aggregation. VWD is caused by a reduced (type 1), an abnormal function (type 2) or a complete absence (type 3) of von Willebrand factor (VWF).

In recent years, large retrospective cohort studies have provided valuable insights on the clinical presentation, bleeding phenotype, quality of life, diagnostics, genetics and treatment of patients with VWD. One of these large studies is the von Willebrand in the Netherlands (WiN) study, which is a nationwide cross sectional study of moderate and severe von Willebrand disease patients, that was initiated in 2007. Over 800 VWD patients were included in the WiN study, which was about 80% of all known VWD patients in the Netherlands. Although the WiN study and large retrospective studies in other countries provided important insights in understanding VWD, some significant challenges remain and large prospective studies are lacking to provide answers.

All large retrospective cohort studies have assessed the bleeding phenotype of patients with VWD using bleeding scores or retrospective questionnaires. Bleeding scores calculate the sum of all bleeding episodes during lifetime. Therefore, they provide useful information on the bleeding tendency during lifetime. However, bleeding scores do not provide information on the change of bleeding tendency. If a patient had a period in his or her lifetime in which he or she had many bleeding episodes, then the bleeding score is high. Though, the patient could have had those bleeds 30 years ago and did not have a bleeding episode since then. Therefore, bleeding scores do not provide information on the current bleeding phenotype of VWD patients. Furthermore, previous studies provided limited information on the frequency of mild bleedings, like gum bleeding or epistaxis, that occur in daily life but do not require therapy. Nevertheless, these bleeding episodes can cause a major impairment in quality of life. This is especially important in children, because school-going children with VWD have a lower quality of life and have a different bleeding tendency, characterized by more cutaneous bleeding (81%), oropharyngeal bleeding (64%) and epistaxis (56%).

Therefore, the primary aim of this study is to prospectively investigate the current bleeding tendency of children and adults with VWD.

Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Von Willebrand disease patients known in a Hemophilia Treatment Center in the Netherlands
Condition Von Willebrand Disease
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Not yet recruiting
Estimated Enrollment
 (submitted: April 30, 2018)
1100
Original Estimated Enrollment Same as current
Estimated Study Completion Date January 2021
Estimated Primary Completion Date January 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Historically lowest VWF:Ag and/or VWF:RCo and/or VWF:CB ≤ 0.30 IU/mL and/or FVIII:C ≤ 0.40 IU/mL
  • Treatment at a Hemophilia treatment center in the Netherlands
  • All types of VWD
  • All ages

Exclusion Criteria:

- Other known bleeding disorders present.

Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Frank WG Leebeek, MD, PhD +31107031369 f.leebeek@erasmusmc.nl
Listed Location Countries Netherlands
Removed Location Countries  
 
Administrative Information
NCT Number NCT03521583
Other Study ID Numbers NL62238.078.18
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Frank W.G. Leebeek, MD, PhD, Erasmus Medical Center
Study Sponsor Erasmus Medical Center
Collaborators
  • Stichting Haemophilia (Dutch Haemophilia Foundation)
  • CSL Behring
  • Shire
Investigators
Principal Investigator: Frank WG Leebeek, MD, PhD Erasmus Medical Center
Study Director: Ferdows Atiq, MD Erasmus Medical Center
Study Chair: Marjon H Cnossen, MD, PhD Erasmus Medical Center
Study Chair: Karin Fijnvandraat, MD, PhD Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Study Chair: Jeroen Eikenboom, MD, PhD Leiden University Medical Center
Study Chair: Johanna G van der Bom, PhD Leiden University Medical Center
Study Chair: Britta AP Laros-van Gorkom, MD, PhD Radboud University
Study Chair: Karina Meijer, MD, PhD University Medical Center Groningen
Study Chair: Karin PM van Galen, MD, PhD UMC Utrecht
Study Chair: Joke de Meris Netherlands Hemophilia Society
PRS Account Erasmus Medical Center
Verification Date May 2018