Contact information
Type
Scientific
Primary contact
Ms Martina Jansen
ORCID ID
Contact details
Oberlaaerstrasse 235
Vienna
1100
Austria
+43 (0)1 61032 1208
martina.jansen@octapharma.com
Additional identifiers
EudraCT number
ClinicalTrials.gov number
Protocol/serial number
WIL-18
Study information
Scientific title
Acronym
WILCOME
Study hypothesis
Long-term prophylactic treatment of von Willebrand disease (VWD) patients in clinical practice.
Ethics approval
Ethics approval received from the Ethics Committee of University Hospital Malmö on the 7th August 2008.
Study design
Prospective, open-labelled, international multi-centre post-marketing surveillance
Primary study design
Interventional
Secondary study design
Non randomised controlled trial
Trial setting
Not specified
Trial type
Treatment
Patient information sheet
Not available in web format, please use the contact details below to request patient information material
Condition
von Willebrand's disease
Intervention
Treatment details:
The following dosing regimes are only recommendations, based on the Swedish experience with the long-term prophylaxis in VWD:
1. For mucosal, joint bleeds and menorrhagia, the suggested prophylactic dosing is 30 IU Wilate® (corresponding to 30 IU FVIII:C)/kg body weight 2 - 3 times/week
2. For gastrointestinal bleeds, the suggested prophylactic dosing is 40 IU Wilate® (40 IU FVIII:C)/kg body weight 2 - 3 times/week
Wilate® is administered as an intravenuos bolus injection. Dose and dosing schedule are at the full discretion of the treating physician.
Quality of life:
The Quality of Life assessments performed during the surveillance will include:
1. The patient's self-reported health-related quality of life prior to and during prophylaxis with Wilate using the validated generic instruments WHOQOL-BREF (for adults and/or parents/legal guardians) and KINDL (for children), and
2. A disease-specific VWD-QoL questionnaire (for adults, children and their parents/legal guardians)
Quality of life assessments will be measured prior to the start of the prophylaxis, as well as after 6 and 12 months of treatment.
Intervention type
Drug
Phase
Phase IV
Drug names
Wilate®
Primary outcome measures
To assess the bleeding frequency in VWD patients prior to and after introduction of regular prophylactic therapy with the VWF-containing concentrate Wilate®. Outcomes will be measured at baseline, 6 and 12 months after treatment. Please note that the number of days the patients missed school or work, as well as occurred adverse drug reactions are documented when the patient visits his doctor, so outcomes may be measured more frequently than every 6 months.
Secondary outcome measures
1. To describe the joint morbidity prior to and during prophylaxis with Wilate®, using the haemophilia joint health score
2. To monitor absence from school/work prior to and during prophylaxis with Wilate®
3. To evaluate the patient's health-related quality of life prior to and during prophylaxis with Wilate® and the patient's self-reported health status prior to and during prophylaxis with Wilate®, using the validated generic instruments of World Health Organization Quality of Life-BREF (WHOQOL-BREF) (for adults) and the generic children's health-related quality of life (KINDL) (for children), and the disease specific VWD-QoL questionnaire
4. To assess treatment satisfaction and treatment efficacy prior to and during prophylaxis with Wilate®, using a 4-point Verbal Rating Scale (VRS), and the Hemo-SatA treatment questionnaire adapted for VWD (VWD-Sat)
5. To evaluate the tolerability of prophylactic treatment with Wilate®:
5.1. Using a 3-point VRS
5.2. By documenting all possibly related adverse drug reactions by the patients, and
5.3. By assessing and - if applicable - reporting all adverse drug reactions by the treating physician occurring during the treatment period with Wilate®
Outcomes will be measured at baseline, 6 and 12 months after treatment. Please note that the number of days the patients missed school or work, as well as occurred adverse drug reactions are documented when the patient visits his doctor, so outcomes may be measured more frequently than every 6 months.
Overall trial start date
01/09/2008
Overall trial end date
01/01/2013
Reason abandoned
Eligibility
Participant inclusion criteria
1. Male and female patients of any age
2. Suffering from congenital VWD
3. In need of replacement therapy with factor concentrate
4. Patients starting with a prophylactic treatment must have documentation of at least three apparently spontaneous bleeding episodes (any bleeding site and treated with factor concentrate) in the 6 months prior to enrolment
5. Patients switching from a prophylactic treatment with another factor concentrate to prophylaxis with Wilate® should have anamnesis of bleeds with respective documentation in the period of 12 months prior to enrolment
Participant type
Patient
Age group
Not Specified
Gender
Both
Target number of participants
24
Participant exclusion criteria
1. Presence of a bleeding disorder other than VWD
2. History of non-compliance
3. Difficulties in achieving venous access that would prohibit prophylaxis
4. Incapability to follow the requirements of the surveillance, e.g. unable to keep a patient diary
Recruitment start date
01/09/2008
Recruitment end date
01/01/2013
Locations
Countries of recruitment
Germany, Russian Federation, Sweden
Trial participating centre
Oberlaaerstrasse 235
Vienna
1100
Austria
Sponsor information
Organisation
Octapharma AG (Switzerland)
Sponsor details
Seidenstrasse 2
Lachen
8853
Switzerland
+41 (0)55 4512140
robert.kuhelj@octapharma.ch
Sponsor type
Industry
Website
Funders
Funder type
Industry
Funder name
Octapharma AG (Switzerland)
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
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
Publication summary