Trial record 8 of 32 for:    "Von Willebrand disease"

National Study of Moderate and Severe Von Willebrand Disease in the Netherlands (WiN)

This study has been completed.
Sponsor:
Collaborators:
Foundation Haemophilia
CSL Behring
Information provided by:
Erasmus Medical Center
ClinicalTrials.gov Identifier:
NCT00510042
First received: July 31, 2007
Last updated: April 23, 2010
Last verified: April 2010
  Purpose

The objective of this study is to assess the clinical presentation, the treatment and the complications of the disease and treatment in moderate and severe von Willebrand disease. Another goal is to investigate the influence of von Willebrand disease on quality of life.


Condition
Von Willebrand Disease

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Cross-Sectional
Official Title: National Study of Moderate and Severe Von Willebrand Disease in the Netherlands

Resource links provided by NLM:


Further study details as provided by Erasmus Medical Center:

Biospecimen Retention:   Samples With DNA

plasma, DNA


Estimated Enrollment: 1100
Study Start Date: July 2007
Study Completion Date: September 2009
Detailed Description:

The incidence of von Willebrand disease in the Netherlands is unknown, but the occurrence of all types of von Willebrand disease together is estimated at 1:100-1:200 individuals. Most patients have mild von Willebrand disease, mostly type 1. For the more severe forms of von Willebrand disease the incidence in unclear, but the expectation is, that there are at least 500-1500 patients. In the Netherlands, an unique situation exists for the treatment of patients with hemophilia and related coagulation disorders. In 2000 a hemophilia management policy was set up by the Ministry of Health, which stated that the care for these patients should be concentrated in 13 Hemophilia Treatment Centers (HTC). The representatives of the attending centers, the hemophilia specialists, are organized in the NVHB, the Dutch society of Hemophilia treaters. In the hemophilia management policy is stated that all patients with a coagulation disorder dependent of replacement products must be treated in a HTC or under responsibility of a HTC. These patients are seen in a HTC at least once a year.

Because all moderate and severe von Willebrand patients in the Netherlands are known in HTC, it is possible to register and study this population. This is imperative to the research of von Willebrand disease. Because the moderate and severe forms of von Willebrand disease are rare, it is impossible for an individual center to perform research of moderate severe and severe von Willebrand disease. Therefore a national approach is necessary.

For optimal care of patients with the moderate and severe form of von Willebrand disease a better understanding of symptoms, diagnostics, treatment and complications of treatment is necessary. The present study aims to register and investigate all patients in the Netherlands with moderate and severe von Willebrand disease to gain understanding of the clinical presentation, the treatment and the complications of treatment in moderate and severe von Willebrand disease. Another goal is to investigate the influence of von Willebrand disease on quality of life.

To answer these questions a questionnaire is developed, which will be sent to the study population. In addition, blood will be drawn for von Willebrand factor measurement and plasma and DNA will be stored. The relationship between laboratory parameters, (including von Willebrand factor, FVIII and prothrombotic coagulation disorders) and the clinical phenotype in patients with moderate and severe von Willebrand disease will be studied. We will assess the effect of the laboratory parameters on both the severity of bleeding tendency, and the possible protecting effect on the risk of arterial thrombosis. In the future mutation analysis of the VWF gene will be performed in patients with moderate and severe von Willebrand disease in the Netherlands.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Patients with moderate and devere von Willebrand disease, known in a hemophilia treatment center

Criteria

Inclusion Criteria:

  1. haemorrhagic symptoms or a family history of von Willebrand disease
  2. vWF antigen ≤ 30%, lowest measurement counts and/or vWF activity (vWF:RCo of vWF:CB) ≤ 30%, lowest measurement counts and/or FVIII:C ≤ 40 %, lowest measurement counts
  3. being known in a hemophilia treatment center or if only the diagnose is made in a hemophilia treatment center this must be done after 1987

Exclusion Criteria:

  1. Hemophilia A
  2. Carriership of hemophilia A
  3. No informed consent
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00510042

Locations
Netherlands
Academic Medical Center Amsterdam
Amsterdam, Netherlands
VU University Medical Center
Amsterdam, Netherlands
Amphia Hospital
Breda, Netherlands
Haga Hospital
Den Haag, Netherlands
Maxima Medical Center
Eindhoven, Netherlands
University Medical Center Groningen
Groningen, Netherlands
Kennemer Hospital
Haarlem, Netherlands
Leiden University Medical Center
Leiden, Netherlands
Academic Hospital Maastricht
Maastricht, Netherlands
University Medical Center St. Radboud
Nijmegen, Netherlands
Erasmus University Medical Center
Rotterdam, Netherlands, 3000 CA
University Medical Center Utrecht Van Creveldkliniek
Utrecht, Netherlands
Sponsors and Collaborators
Erasmus Medical Center
Foundation Haemophilia
CSL Behring
Investigators
Study Director: Eva M de Wee, MD Erasmus Medical Center
Principal Investigator: Frank WG Leebeek, MD, PhD Erasmus Medical Center
Study Chair: Karin Fijn van Draat, MD, PhD Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Study Chair: Jeroen CJ Eikenboom, MD, PhD Leiden University Medical Center
Study Chair: Arja de Goede-Bolder, MD Erasmus Medical Center
Study Chair: Eveline P Mauser-Bunschoten, MD, PhD Van Creveldkliniek, University Medical Center Utrecht
Study Chair: Karina Meijer, MD, PhD University Medical Center Goningen
Study Chair: Britta Laros-van Gorkom, MD, PhD University Medical Center St. Radboud Nijmegen
Study Chair: Johanna G van der Bom, PhD Leiden University Medical Center
Study Chair: Manon A Degenaar-Dujardin, BA Organization for Hemophilia Patients
  More Information

Publications:
Responsible Party: F.W.G. Leebeek, MD PhD, Erasmus University Medical Center
ClinicalTrials.gov Identifier: NCT00510042     History of Changes
Other Study ID Numbers: MEC-2007-063
Study First Received: July 31, 2007
Last Updated: April 23, 2010
Health Authority: Netherlands: Medical Ethics Review Committee (METC)

Keywords provided by Erasmus Medical Center:
von Willebrand disease
von Willebrand factor
national study
bleeding disorder
quality of life

Additional relevant MeSH terms:
Von Willebrand Diseases
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Blood Platelet Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on August 25, 2014