At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Emicizumab (drug), von Willebrand Factor (VWF) Concentrates (drug), Factor VIII (FVIII) Concentrates (drug), von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates (drug)
- How long the study runs
- Study runs about 46 months (dates as stated)
- About the drug or intervention
- Emicizumab — drug: Participants will receive emicizumab 3 milligrams per kilogram (mg/kg) subcutaneous (SC) injections every week (QW) for the first 4 weeks as loading doses, followed by maintenance doses of emicizumab 3 mg/kg SC once every 2 weeks (Q2W). · von Willebrand Factor (VWF) Concentrates — drug: Used according to local labeling or local treatment guidelines. · Factor VIII (FVIII) Concentrates — drug: Used according to local labeling or local treatment guidelines. · von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates — drug: Used according to local labeling or local treatment guidelines. · Bypassing Agents — drug: Used according to local labeling or local treatment guidelines.
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 1 Month and over
- Who
- All
- Number of participants
- 75
- Started
- 2025-06-27
- Last checked
- 2026-10
Plain English Summary
What is this study?
- • Testing a new treatment for von willebrand disease, type 3
- • Phase3 - 75 participants
- • This is a Phase III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab prophylaxis in participants aged 1 month and above, who have been diagnosed with Type 3 von Willebrand disease (VWD)
Who can take part?
- • Ages 1 Month 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 is a Phase III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab prophylaxis in participants aged 1 month and above, who have been diagnosed with Type 3 von Willebrand disease (VWD). Participants on prior standard of care (SOC) on-demand therapy will be assessed via a randomized comparison (Arm A - emicizumab prophylaxis and Arm B - continuation of SOC on-demand therapy), while participants on prior SOC prophylactic therapy (Arm C - emicizumab prophylaxis) will be assessed via intra-participant analysis with data obtained from the preceding non-interventional study (NIS), WP45335 (NCT06883240).
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: 1 Month and over
- Who can join: All genders
Biomarkers mentioned
What the study is looking for
- ✓Confirmed diagnosis of Type 3 von Willebrand disease (VWD), based on medical records
- ✓Preexisting medical record verifying the status of von Willebrand factor (VWF) inhibitor (positive or negative,...
- ✓Adequate blood, liver, and kidney function
- ✓For participants of childbearing potential: agreement to remain abstinent or adhere to the contraception requirements
- ✓Additional Inclusion Criteria for Arms A and B:
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
How to Get in Touch
Reference Study ID Number: WP45338 https://forpatients.roche.com/
Sponsor contactCONTACT
Fastest response: use the inquiry form. No email attachments. https://www.gene.com/contact-us/submit-medical-inquiry
Sponsor contactCONTACT
