Desmopressin in the management of von Willebrand disease; Biological versus clinical efficacy.
- Conditions
- von Willebrand disease (VWD) is an inherited bleeding disorder, characterised mainly by mucosal bleedings, which may be life-threatening, and joint bleeds in severe VWD cases. VWD is caused by a lack of von Willebrand factor (VWF) and coagulation factor VIII (FVIII). Treatment of VWD aims at normalizing the VWF activity in plasma, which can be achieved by stimulating the endogenous release of VWF with desmopressin (DDAVP, 1-desamino-8-D arginine vasopressin) or by infusion of a VWF concentrate.
- Registration Number
- EUCTR2005-004496-38-DK
- Lead Sponsor
- Rigshospitalet, Copenhagen
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 150
Well-characterized VWD patients with all type 1 and 2 (including 2B if treatment with DDAVP is allowed in any centre), with the widest range of age (from young children to senior adults). Due to the increased number of blood sampling, kinetic studies will be performed in patients older than 12 only.
Diagnosis of type 1 and 2 VWD should be correctly performed by the participating centre following recommendations provided by the ISTH-SSC on VWF. If available, molecular diagnosis (mutations) should also be reported to confirm classification in a specific VWD type. In a few patients, to be considered undefined because of a difficult diagnosis, plasma samples should be kept and sent, upon request by the Centre, to a more experienced laboratory. All these difficult diagnosis will be confirmed by the Steering Committee by using clinical and laboratory data.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
Patients should be excluded from the study in the following cases: a) Acquired von Willebrand Syndrome; b) Additional congenital and acquired defects of platelet function (ex, Acquired Storage Pool defect + VWD); c) use of anti-inflammatory drugs that may affect platelet function within the previous 10 days; d) previous history of seizures in the family; e) recent serious cardiovascular episodes (Acute myocardial infarction and stroke) in the VWD patient.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method