Skip to main content
UK clinical trials - updated daily from ClinicalTrials.gov
TrialConnect
← Back to Search
ACTIVE NOT RECRUITINGN/A

An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment

Sponsor: Hoffmann-La Roche

NCT ID: NCT06883240

View on ClinicalTrials.gov ↗

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.

Von Willebrand Disease, Type 3

How this trial compares with your answers

Answer 2 more questions to improve match

What we know so far

Condition· Matched your search
Age· Tell us your age for better matching
Gender· Tell us your sex for better matching

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
Inclusion Criteria: * Confirmed diagnosis of Type 3 von Willebrand disease (VWD), based on medical records * Adequate hematologic, hepatic, and renal function * Documented and confirmed previous use of SOC prophylactic therapy for VWD (1-3 times weekly, as per prescribed dose) and anticipation to remain on the same regimen during the study * For participants of childbearing potential: agreement to remain abstinent or adhere to the contraception requirements Exclusion Criteria: * 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 * History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection * Use of systemic immunomodulators (e.g., interferon) at enrollment or planned use during the study, with the exception of anti-retroviral therapy

Where Is This Study? (3 UK sites)

St Thomas' Hospital

London SE1 7EH, United Kingdom

Hospital R&D contact (matched)

Main Email: gstt.research.rbhh@nhs.net

gstt.research.rbhh@nhs.netn/a

Great Ormond Street Hospital

London WC1N 3JH, United Kingdom

Hospital R&D contact (matched)

Main Email: Research.Governance@gosh.nhs.uk

Research.Governance@gosh.nhs.uk0207 905 2700

Manchester Royal Infirmary

Manchester M13 9WL, United Kingdom

Hospital R&D contact (matched)

Elizabeth Mainwaring

R&D.applications@mft.nhs.uk0161 276 3340
Data sourced from ClinicalTrials.gov · Last verified: 2026-08