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Looking for participantsPhase3

A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease

Sponsor: Hoffmann-La Roche

NCT ID: NCT06998524

View on ClinicalTrials.gov ↗

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).

Von Willebrand Disease, Type 3

How this trial compares with your answers

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What we know so far

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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

positive or negative

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
Inclusion Criteria: * 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, including titer if available) * Adequate hematologic, hepatic, and renal function * For participants of childbearing potential: agreement to remain abstinent or adhere to the contraception requirements Additional Inclusion Criteria for Arms A and B: * Age ≥1 month at the time of signing Informed Consent/Assent Form * Documented previous use of on-demand therapy with intermittent (less than once a week) on-demand SOC therapy for VWD * Having ≥2 treated bleeds (except menstrual bleeds) with factor concentrate within 24 weeks prior to enrollment Additional Inclusion Criteria for Arm C: * Age ≥2 years at the time of signing Informed Consent/Assent Form * Documented and confirmed previous use of SOC prophylactic therapy for VWD (1-3 times weekly, as per prescribed dose) as described in the eligibility of Study WP45335 * Have completed all study requirements as defined in the WP45335 protocol for at least 24 weeks 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

Recruiting
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

Recruiting
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

Recruiting
Hospital R&D contact (matched)

Elizabeth Mainwaring

R&D.applications@mft.nhs.uk0161 276 3340

How to Get in Touch

Reference Study ID Number: WP45338 https://forpatients.roche.com/

Sponsor contact

CONTACT

888-662-6728 (U.S. Only) global-roche-genentech-trials@gene.com

Fastest response: use the inquiry form. No email attachments. https://www.gene.com/contact-us/submit-medical-inquiry

Sponsor contact

CONTACT

Data sourced from ClinicalTrials.gov · Last verified: 2026-10