WP45335 - Type 3 Von Willebrand Disease
Research type
Research Study
Full title
A MULTICENTER NON-INTERVENTIONAL STUDY EVALUATING BLEEDS AND HEALTH-RELATED QUALITY OF LIFE IN PATIENTS WITH TYPE 3 VON WILLEBRAND DISEASE ON PROPHYLACTIC STANDARD OF CARE TREATMENT
IRAS ID
351734
Contact name
Head EU/ International Regulatory Affairs
Contact email
Sponsor organisation
F. Hoffmann-La Roche Ltd
Duration of Study in the UK
1 years, 5 months, 30 days
Research summary
The purpose of this observational (non- interventional) study is to see how the usual medicines patients take to prevent bleeding caused by Type 3 von Willebrand Disease (VWD) work in real life. Also, to gather more information about the safety and how well patients feel with the medicines.
The study will enroll participants with Type 3 VWD, aged 2 years and older. Participants will be followed up for at least a 24 week observation period on their standard of care (SOC) medication taken to prevent bleeds (prophylactic medication).
There are many factors that can only be studied in routine medical practice, particularly if many patients participate. Examples include little known side effects of the medication may be detected or the influence of dose changes or other medications being taken along with prophylactic SOC medicines can be observed. This study may help to understand better how to use prophylactic SOC therapy and may optimize the treatment of patients with Type 3 VWD.
The study will last approximately 76 weeks.
40 patients will be enrolled globally and approximately 8 patients will be enrolled from 3 UK hospitals
Research Summary; Version Number 1.0 dated 29 November 2024
REC name
London - Fulham Research Ethics Committee
REC reference
25/PR/0091
Date of REC Opinion
25 Feb 2025
REC opinion
Further Information Favourable Opinion