Condition category
Haematological Disorders
Date applied
22/08/2008
Date assigned
01/09/2008
Last edited
04/09/2008
Prospective/Retrospective
Prospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

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

http://www.octapharma.com

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

Publication citations

Additional files

Editorial Notes