Surveillance of results of long-term prophylactic treatment of von Willebrand disease with Wilate®

ISRCTN	ISRCTN25330204
DOI	https://doi.org/10.1186/ISRCTN25330204
Secondary identifying numbers	WIL-18

Submission date: 22/08/2008
Registration date: 01/09/2008
Last edited: 04/09/2008

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Haematological Disorders

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Record updated in last year

Plain English summary of protocol

Not provided at time of registration

Contact information

Ms Martina Jansen
Scientific

Oberlaaerstrasse 235
Vienna
1100
Austria

Phone	+43 (0)1 61032 1208
Email	martina.jansen@octapharma.com

Study information

Study design	Prospective, open-labelled, international multi-centre post-marketing surveillance
Primary study design	Interventional
Secondary study design	Non randomised controlled trial
Study setting(s)	Not specified
Study type	Treatment
Participant information sheet	Not available in web format, please use the contact details below to request patient information material
Scientific title
Study acronym	WILCOME
Study objectives	Long-term prophylactic treatment of von Willebrand disease (VWD) patients in clinical practice.
Ethics approval(s)	Ethics approval received from the Ethics Committee of University Hospital Malmö on the 7th August 2008.
Health condition(s) or problem(s) studied	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
Pharmaceutical study type(s)
Phase	Phase IV
Drug / device / biological / vaccine name(s)	Wilate®
Primary outcome measure	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 study start date	01/09/2008
Completion date	01/01/2013

Eligibility

Participant type(s)	Patient
Age group	Not Specified
Sex	Both
Target number of participants	24
Key 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
Key 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
Date of first enrolment	01/09/2008
Date of final enrolment	01/01/2013

Locations

Countries of recruitment

Austria
Germany
Russian Federation
Sweden

Study participating centre

Oberlaaerstrasse 235

Vienna
1100
Austria

Sponsor information

Octapharma AG (Switzerland)
Industry

Seidenstrasse 2
Lachen
8853
Switzerland

Phone	+41 (0)55 4512140
Email	robert.kuhelj@octapharma.ch
Website	http://www.octapharma.com
"ROR"	https://ror.org/002k5fe57

Funders

Funder type

Industry

Octapharma AG (Switzerland)

No information available

Results and Publications

Intention to publish date
Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
Publication and dissemination plan	Not provided at time of registration
IPD sharing plan