At a glance
- What the study gets you
- Health checks and monitoring — no treatment given
- Type of study
- Observational (no treatment given)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Von Willebrand Factor Concentrates (drug), Von Willebrand Factor Concentrates and Factor VIII Concentrates (drug), Factor VIII Concentrates (drug), Recombinant Activated Factor VII (drug)
- How long the study runs
- Study runs about 19 months (dates as stated)
- About the drug or intervention
- Von Willebrand Factor Concentrates — drug: Used according to local labeling or local treatment guidelines. · Von Willebrand Factor Concentrates and Factor VIII Concentrates — drug: Used according to local labeling or local treatment guidelines. · Factor VIII Concentrates — drug: Used according to local labeling or local treatment guidelines. · Recombinant Activated Factor VII — drug: Used according to local labeling or local treatment guidelines. · Activated Prothrombin Complex Concentrate — drug: Used according to local labeling or local treatment guidelines.
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Observing health over time
- Ages
- 2 Years and over
- Who
- All
- Number of participants
- 44
- Started
- 2025-04-29
- Last checked
- 2026-08
Plain English Summary
What is this study?
- • Testing a new treatment for von willebrand disease, type 3
- • Clinical study - 44 participants
- • This non-interventional study (NIS) is designed to collect information on the effectiveness and safety of treatment received in routine clinical care, as well as measure the health-related quality of life (HRQoL) of participants with Type 3 von Willebrand disease (VWD) receiving prophylactic therapy per local standard of care (SOC) over an observation period of at least 24 weeks
Who can take part?
- • Ages 2 Years and over
- • Diagnosed with von willebrand disease, type 3
Where?
- • London - St Thomas' Hospital
- • London - Great Ormond Street Hospital
- • Manchester - Manchester Royal Infirmary
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
This non-interventional study (NIS) is designed to collect information on the effectiveness and safety of treatment received in routine clinical care, as well as measure the health-related quality of life (HRQoL) of participants with Type 3 von Willebrand disease (VWD) receiving prophylactic therapy per local standard of care (SOC) over an observation period of at least 24 weeks.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 2 Years and over
- Who can join: All genders
What the study is looking for
- ✓Confirmed diagnosis of Type 3 von Willebrand disease (VWD), based on medical records
- ✓Adequate blood, liver, and kidney function
- ✓Documented and confirmed previous use of SOC prophylactic therapy for VWD (1-3 times weekly, as per prescribed dose)...
- ✓For participants of childbearing potential: agreement to remain abstinent or adhere to the contraception requirements
Who cannot take part
- ✗Inherited or acquired bleeding disorder other than Congenital Type 3 VWD
- ✗History of gastrointestinal bleeding within 18 months prior to enrollment, or any previous diagnosis of angiodysplasia
- ✗History of intracranial hemorrhage
- ✗Previous or current treatment for thromboembolic disease or signs of thromboembolic disease
- ✗Other conditions (e.g., certain autoimmune diseases) that may increase risk of bleeding or thrombosis
See the full criteria
Where Is This Study? (3 UK sites)
St Thomas' Hospital
London SE1 7EH, United Kingdom
Great Ormond Street Hospital
London WC1N 3JH, United Kingdom
Main Email: Research.Governance@gosh.nhs.uk
Research.Governance@gosh.nhs.uk0207 905 2700Manchester Royal Infirmary
Manchester M13 9WL, United Kingdom
