Surveillance of Tolerability And Treatment Efficacy of Wilate® in von Willebrands disease
| ISRCTN | ISRCTN98642570 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN98642570 |
| Secondary identifying numbers | WIL-25 |
- Submission date
- 13/09/2012
- Registration date
- 05/12/2012
- Last edited
- 09/05/2017
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Haematological Disorders
Plain English summary of protocol
Background and study aims
Patients with von Willebrand disease (a bleeding disorder that affects your blood's ability to clot) may require substitution with a coagulation factor concentrate like Wilate. In this study, data about the treatment with Wilate will be collected for safety surveillance and efficacy evaluations.
Who can participate?
All patients with von Willebrands disease in need for replacement therapy whos doctor had first decided to use Wilate and then to participate in the study can be included.
What does the study involve?
The treatment with Wilate will be the same as prescribed by the doctor without the study no additional interventions are required. Careful documentation of therapy by the doctor or in case of home treatment by the patient is necessary.
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 von Willebrand disease under routine conditions. There are no risks arising from participation.
Where is the study run from?
The study will take place in several haemophilia centres in Germany.
When is the study starting and how long is it expected to run for?
June 2012 to May 2020
Who is funding the study?
Octapharma GmbH (Germany)
Who is the main contact?
Dr Susanne Seeger
susanne.seeger@octapharma.de
Contact information
Scientific
Elisabeth-Selbert-Str. 11
Langenfeld
40764
Germany
| Phone | +49 (0)2131 917131 |
|---|---|
| susanne.seeger@octapharma.de |
Study information
| Study design | Observational open prospective multi-centre study |
|---|---|
| Primary study design | Observational |
| Secondary study design | Other |
| Study setting(s) | Hospital |
| Study type | Treatment |
| Participant information sheet | Not available in web format, please use the contact details to request a patient information sheet |
| Scientific title | Surveillance of Tolerability And Treatment Efficacy of Wilate® in von Willebrands disease: an observational study |
| Study acronym | Wilate-STATE |
| Study objectives | Wilate 500/1000 is safe and efficacious for treatment of von Willebrand's disease in clinical practice. |
| Ethics approval(s) | Ethics Committee of Medical Faculty of Johann Wolfgang Goethe University Frankfurt/Main Germany, November 2012 |
| Health condition(s) or problem(s) studied | von Willebrand's disease |
| Intervention | For this non-interventional study, details of the patient history and the current treatment with Wilate will carefully be recorded. This included information on of injections, duration and intensity of bleeding episodes and information regarding possible surgical interventions. The efficacy will be assessed according to a 4-point verbal rating scale. Suspected adverse drug reactions (side effects) will be documented. The observation time depends on the clinical needs of the patient, e.g. from a few days in case Wilate is required only to cover a surgery or until study end in case of prophylactic treatment. |
| Intervention type | Drug |
| Pharmaceutical study type(s) | |
| Phase | Not Applicable |
| Drug / device / biological / vaccine name(s) | Wilate® |
| Primary outcome measure | Rate of adverse drug reactions. Laboratory parameters relevant to safety when documented |
| Secondary outcome measures | 1. Percentage of efficacy rating "excellent" or "good" in bleeding episodes and surgeries 2. Bleeding frequency in prophylactically treated patients and - if available - course of laboratory parameters indicating anemia. Comparison of efficacy results with precursor study with Wilate 450/900 when feasible |
| Overall study start date | 28/06/2012 |
| Completion date | 31/05/2020 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Sex | Both |
| Target number of participants | 120 |
| Key inclusion criteria | 1. The patient suffers from hereditary or acquired von Willebrand's disease and is in need for replacement of coagulation concentrate containing von Willebrand factor (VWF) 2. When bleeding history is positive, other causes were excluded |
| Key exclusion criteria | 1. The patient could actually be treated with DDAVP (Desmopressin Acetate) 2. The patient has a von Willebrand factor activity over 70 % and there are no findings manifesting the von Willebrand (VWD) diagnosis like 2.1. Bleeding pattern compliant with VWD 2.2. Positive family history 2.3. Mutation analysis 2.4. Multimer pattern |
| Date of first enrolment | 28/07/2012 |
| Date of final enrolment | 30/04/2020 |
Locations
Countries of recruitment
- Germany
Study participating centre
40764
Germany
Sponsor information
Industry
Elisabeth-Selbert-Str. 11
Langenfeld
40764
Germany
| Phone | +49 (0)2173 917100 |
|---|---|
| VWS@octapharma.de | |
| Website | http://www.octapharma-biopharmaceuticals.com |
"ROR" |
https://ror.org/002k5fe57 |
Funders
Funder type
Industry
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 |
Editorial Notes
04/05/2017: The overall trial end date was changed from 30/05/2017 to 31/05/2020.
