Surveillance of results of long-term prophylactic treatment of von Willebrand disease with Wilate®
ISRCTN | ISRCTN25330204 |
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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
Scientific
Oberlaaerstrasse 235
Vienna
1100
Austria
Phone | +43 (0)1 61032 1208 |
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martina.jansen@octapharma.com |
Study information
Study design | Prospective, open-labelled, international multi-centre post-marketing surveillance |
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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 |
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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
1100
Austria
Sponsor information
Octapharma AG (Switzerland)
Industry
Industry
Seidenstrasse 2
Lachen
8853
Switzerland
Phone | +41 (0)55 4512140 |
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robert.kuhelj@octapharma.ch | |
Website | http://www.octapharma.com |
https://ror.org/002k5fe57 |
Funders
Funder type
Industry
Octapharma AG (Switzerland)
No information available
Results and Publications
Intention to publish date | |
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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 |