Collection of data on the use of a VWF/FVIII concentrate (wilate®) in all clinical applications in von Willebrand disease
| ISRCTN | ISRCTN11568655 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN11568655 |
| Secondary identifying numbers | WIL-34 |
- Submission date
- 19/01/2022
- Registration date
- 09/02/2022
- Last edited
- 27/05/2025
- Recruitment status
- No longer recruiting
- Overall study status
- Ongoing
- Condition category
- Haematological Disorders
Plain English summary of protocol
Background and study aims
Von Willebrand disease (VWD) is a blood disorder in which the blood does not clot properly due to low levels of the clotting protein von Willebrand factor (VWF). Medicines such as wilate can be used to replace the missing factor. The aim of this study is to determinate the effectiveness and tolerability of wilate in the prophylaxis (prevention) and treatment of bleeding as well as in surgical interventions under everyday clinical conditions.
Who can participate?
Patients with VWD requiring treatment with VWF-containing product (wilate)
What does the study involve?
The routine treatment of VWD patients will be documented, including all treatments with Wilate, any bleeding episodes, surgical interventions and optional data on quality of life and joint health.
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 VWD under routine conditions. There are no risks arising from participation.
Where is the study run from?
Gerinnungszentrum Rhein-Ruhr (GZRR) (Germany)
When is the study starting and how long is it expected to run for?
January 2020 to May 2025
Who is funding the study?
Octapharma GmbH (Germany)
Who is the main contact?
Dr Susanne Seeger
susanne.seeger@octapharma.com
Contact information
Scientific
Elisabeth-Selbert-Str. 11
Langenfeld
40764
Germany
| Phone | +49 (0)2173917131 |
|---|---|
| susanne.seeger@octapharma.com |
Study information
| Study design | Open prospective multi-centre national non-interventional study |
|---|---|
| Primary study design | Observational |
| Secondary study design | Cohort study |
| Study setting(s) | Other |
| Study type | Treatment |
| Participant information sheet | Not available in web format, please use the contact details to request a patient information sheet |
| Scientific title | Non-interventional study to capture data on the use of a VWF/FVIII concentrate (wilate®) in all clinical settings of von Willebrand disease including health-related quality of life |
| Study acronym | wilate-NEWS |
| Study objectives | Wilate is efficacious and safe for the treatment of von Willebrand's disease in clinical practice. |
| Ethics approval(s) | Approved 28/09/2020, Ethics Committee of Medical Association of North Rhine (Ärztekammer Nordrhein, Tersteegenstraße 9, 40474 Düsseldorf, Germany; +49 (0)211 4302 2272; ethik@aekno.de), ref: 2020260 |
| Health condition(s) or problem(s) studied | Von Willebrand disease (VWD) |
| Intervention | wilate-NEWS is an non-interventional study for which the routine treatment of VWD patients on prophylaxis or on-demand treatment is documented. All treatments occurring with wilate, any bleeding episodes occurring or surgical procedures are carefully documented. Additionally, a joint score (HJHS), quality of life questionnaire (SF-36) and/or a pictorial blood assessment chart (PBAC) can be documented on an optional basis. The observation period of each patient is not fixed. Follow-up is planned at regular intervals (every 6 months) after the inclusion of the patients. |
| Intervention type | Drug |
| Pharmaceutical study type(s) | |
| Phase | Not Applicable |
| Drug / device / biological / vaccine name(s) | wilate® |
| Primary outcome measure | Percentage of haemostatic efficacy ratings of "excellent" and "good" for bleeding treatments and surgical interventions, as well as the annual bleeding rate in prophylactically treated patients, measured at the end of the study. According to the study protocol, efficacy is rated by the treating physicians for every bleeding event treated with Wilate and surgery respectively treated with Wilate on a 4-point Likert scale (excellent, good, moderate, none). |
| Secondary outcome measures | 1. Safety assessed using the number of patients experiencing at least one adverse drug reaction (ADR) measured at the end of the study 2. Calculation of the VWF dose/kg body weight per injection and calculation of the duration of use in the different treatment options (prophylaxis, bleeding treatment, surgical intervention), depending on the VWD type or severity of bleeding or intervention, at the end of the study 3. Health-related quality of life measured optionally half-yearly using SF-36 during treatment with Wilate, including a comparison between types of VWD and between female and male patients - as far as case numbers allow 4. Comparison of joint status determined optionally half-yearly using the Haemophilia Joint Health Score (HJHS) and annualised bleeding rate, taking into account the treatment mode as well as the VWD type (as far as the case numbers allow), at the end of the study 5. PBAC (Pictorial Blood Assessment Chart) scores in relation to treatment regimen and dosages at the end of the study |
| Overall study start date | 02/01/2020 |
| Completion date | 31/05/2030 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | All |
| Sex | Both |
| Target number of participants | 120 |
| Key inclusion criteria | 1. The patient has congenital or acquired Von Willebrand disease (VWD) and requires injections with VWF-containing concentrate (wilate) 2. With a positive bleeding history, other possible causes have been excluded |
| Key exclusion criteria | 1. Patients with known contraindications as specified in the Summary of Product Characteristics (SPC) 2. The patient could actually be treated with DDAVP (Desmopressin Acetate) 3. The patient has a von Willebrand factor activity over 70% and there are no findings manifesting the VWD diagnosis |
| Date of first enrolment | 01/06/2020 |
| Date of final enrolment | 31/05/2025 |
Locations
Countries of recruitment
- Germany
Study participating centre
47051
Germany
Sponsor information
Industry
Elisabeth-Selbert-Str. 11
Langenfeld
40764
Germany
| Phone | +49 (0)2173917 0 |
|---|---|
| info.de@octapharma.com | |
| Website | https://www.octapharma.com/ |
Funders
Funder type
Other
No information available
Results and Publications
| Intention to publish date | 31/05/2026 |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Data sharing statement to be made available at a later date |
| Publication and dissemination plan | Planned publication as posters on congresses and in a peer-reviewed journal. Within 1 year after completion of the data collection, Octapharma GmbH submits a final report to the federal authority (Paul Ehrlich Institute) which is also made available to the public via an internet portal. |
| IPD sharing plan | The data-sharing plans for the current study are unknown and will be made available at a later date. |
Editorial Notes
27/05/2025: The overall end date was changed from 31/05/2025 to 31/05/2030.
02/02/2022: Trial's existence confirmed by the Ethics Committee of Medical Association of North Rhine.
