A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease
Recruiting
Phase 3Interventional Study
Von Willebrand Disease, Type 3
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
Ready to participate?
Review the details below, then apply to join this clinical trial.
At a Glance
Age
1 Month and older
Sex
Any
Trial phase
Phase 3
Study type
Interventional
Purpose
Treatment
Participants needed
75 (estimated)
Sponsor
Hoffmann-La Roche · Industry
Who this trial is looking for
This trial is looking for people aged 1 month and older who have been diagnosed with Type 3 von Willebrand disease. Participants will receive either emicizumab treatment or continue their current therapy, depending on their previous treatment history.
Are You a Good Fit for This Trial?
You may be able to join if
I have been diagnosed with Type 3 von Willebrand disease
I have a medical record showing my von Willebrand factor inhibitor status
I have adequate blood, liver, and kidney function
I will follow the birth control requirements if I can get pregnant
I am at least 1 month old
I have used on-demand therapy for my condition recently
I have had at least 2 treated bleeds in the last 24 weeks
You may not be able to join if
I have another bleeding disorder besides Type 3 von Willebrand disease
I have had gastrointestinal bleeding in the last 18 months
I have a history of bleeding in the brain
I have been treated for blood clots or have signs of blood clots
I have conditions that increase my bleeding or blood clot risk
I have had severe allergic reactions to similar treatments
I am using certain immune-modulating medications
Summarized in plain language from this trial's official eligibility criteria.
The full criteria are further down this page — only the research team can
confirm whether you qualify.
Think this trial could be right for you?
Answer three quick questions written from this study's own rules to see if you may qualify. No account needed to find out.
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 - emicizuma…
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).
Trial Locations
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City
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Status
Eligibility 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 funct…
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
Contacts
Reference Study ID Number: WP45338 https://forpatients.roche.com/
Fastest response: use the inquiry form. No email attachments. https://www.gene.com/contact-us/submit-medical-inquiry
CONTACT
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