Plain English Summary
Background and study aims
Patients with von Willebrand disease (a bleeding disorder that affects your blood's ability to clot) may require substitution with a coagulation factor concentrate like Wilate. In this study, data about the treatment with Wilate will be collected for safety surveillance and efficacy evaluations.
Who can participate?
All patients with von Willebrands disease in need for replacement therapy whos doctor had first decided to use Wilate and then to participate in the study can be included.
What does the study involve?
The treatment with Wilate will be the same as prescribed by the doctor without the study no additional interventions are required. Careful documentation of therapy by the doctor or in case of home treatment by the patient is necessary.
What are the possible benefits and risks of participating?
All information collected in the course of the treatment with Wilate will add knowledge on the use of Wilate and treatment of the von Willebrand disease under routine conditions.
There are no risks arising from participation.
Where is the study run from?
The study will take place in several haemophilia centres in Germany.
When is the study starting and how long is it expected to run for?
The study starts in June 2012 and is planned for a duration of 5 years.
Who is funding the study?
The study is beeing funded by Octapharma, the company that produces and distributes Wilate.
Who is the main contact?
Ms Jennifer Feddern
+49 (0)2173 917233
Surveillance of Tolerability And Treatment Efficacy of Wilate® in von Willebrands disease: An observational study
Wilate 500/1000 is safe and efficacious for treatment of von Willebrand's disease in clincial practice.
Ethics Committee of Medical Faculty of Johann Wolfgang Goethe University Frankfurt/Main Germany, November 2012
Observational open prospective multi-centre study
Primary study design
Secondary study design
Patient information sheet
Not available in web format, please use the contact details below to request a patient information sheet
von Willebrand's disease
For this non-interventional study, details of the patient history and the current treatment with Wilate will carefully be recorded. This included information on of injections, duration and intensity of bleeding episodes and information regarding possible surgical interventions. The efficacy will be assessed according to a 4-point verbal rating scale. Suspected adverse drug reactions (side effects) will be documented. The observation time depends on the clinical needs of the patient, e.g. from a few days in case Wilate is required only to cover a surgery or until study end in case of prophylactic treatment.
Primary outcome measures
Rate of adverse drug reactions. Laboratory parameters relevant to safety when documented
Secondary outcome measures
1. Percentatge of efficacy rating "excellent" or "good" in bleeding episodes and surgeries
2. Bleeding frequency in prophylactically treated patients and - if available - course of laboratory parameters indicating anemia. Comparison of efficacy results with precursor study with Wilate 450/900 when feasible.
Overall trial start date
Overall trial end date
Participant inclusion criteria
1. The patient suffers from hereditary or acquired von Willebrand's disease and is in need for replacement of coagulation concentrate containing von Willebrand factor (VWF)
2. When bleeding history is positive, other causes were excluded
Target number of participants
Participant exclusion criteria
1. The patient could actually be treated with DDAVP (Desmopressin Acetate)
2. The patient has a von Willebrand factor activity over 70 % and there are no findings manifesting the von Willebrand (VWD) diagnosis like
2.1. Bleeding pattern compliant with VWD
2.2. Positive family history
2.3. Mutation analysis
2.4. Multimer pattern
Recruitment start date
Recruitment end date
Countries of recruitment
Trial participating centre
Octapharma GmbH (Germany)
Funding Body Type
Funding Body Subtype
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
Participant level data
Not provided at time of registration
Results - basic reporting