Study identification

PURI

https://redirect.ema.europa.eu/resource/26513

EU PAS number

EUPAS12560

Study ID

26513

Official title and acronym

Surveillance of Safety and Efficacy of wilate® in patients with von Willebrand disease (Wil-20)

DARWIN EU® study

No

Study countries

Argentina
Canada
Colombia
Czechia
Germany
Portugal
Spain
Sweden
United Kingdom
United States
Uruguay

Study description

Primary objective is to document the safety and tolerability of wilate® for prophylaxis and treatment of bleeding in VWD, incl. surgeriesSecondary objective is to document the efficacy of wilate® in the treatment of acute bleeding, in the prophylaxis of VWD and in interventional procedures (e.g. minor/major surgery, dental care, invasive diagn. proced. etc.).Population:VWD patients of any gender, age, or VWD type, previously treated (PTPs) or previously untreated patients (PUPs).Investigational and reference therapy:wilate® - human coagulation factor VIII and human von Willebrand factor (VWF)Design:Open-label, prospective, multicentre, multinational, post-marketing, observational, non-interventional surveillanceEfficacy assessments:Assessment of efficacy of wilate® in prevention and/or treatment of bleeding episodes and in surgical procedures will be based on a 4-point hemostatic efficacy scale as “excellent”, “good” “moderate” or “none”. The frequency of bleeding episodes in total and per bleeding site, days of treatment of bleeding episodes in total and per bleeding site, exposure days and consumption of wilate® per event, per patient and in total will be calculated.Safety/Tolerability assessments:Assessment of safety will be based on recorded Adverse Drug Reactions during the full course of the observation. Assessment of tolerability will be based on a 3 point Verbal Rating Scale.As recomm. assessment, this study will observe development of inhibitors against VWF in response to wilate® treatment (ELISA). Inhibitor assessment should be performed before and after first wilate® application, and then the every 3 months.As recomm. assessment, study will observe the coagulation parameters based on assessment of prothrombin fragment 1 and 2 (F1+2) and D-dimer (DD) by latex enhanced immunoturbimetric test. Thrombogenicity assessment should be performed before first wilate® application, 1 hour, 3 and 24 hours after application and every 3 months

Study status

Finalised
Research institutions and networks

Institutions

Octapharma
First published:
01/02/2024
Institution
Multiple centres: 30 centres are involved in the study

Contact details

Irina Kruzhkova

Primary lead investigator
Study timelines

Date when funding contract was signed

Actual:

Study start date

Actual:

Date of final study report

Planned:
Actual:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

Octapharma
Regulatory

Was the study required by a regulatory body?

Unknown

Is the study required by a Risk Management Plan (RMP)?

EU RMP category 3 (required)