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Tests treatment safety and results for Von Willebrand Disease, Type 3
Official title A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease
ClinicalTrials.gov ID: NCT06998524
What this study is testing
What is Emicizumab?
Emicizumab is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for von willebrand disease, type 3.
Also referred to as Hemlibra, RO5534262.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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).
- Phase 3: a large, late-stage study
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 and older
You may be able to join if
- 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 for Arms A and B:
- Age ≥1 month at the time of signing Informed Consent/Assent Form
You likely can't join if
- 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
See the full eligibility 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 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 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
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Sacramento, California, United States
- Gainesville, Florida, United States
- Minneapolis, Minnesota, United States
- St Louis, Missouri, United States
- Richmond, Virginia, United States
- Leuven, Belgium
- Toronto, Ontario, Canada
- Montreal, Quebec, Canada
- Medellín, Colombia
- Lille, France
- Paris, France
- Bonn, Germany
- Duisburg, Germany
- Frankfurt/M., Germany
- Rome, Lazio, Italy
- Milan, Lombardy, Italy
- Florence, Tuscany, Italy
- Fukuoka, Japan
- Nagoya, Japan
- Rotterdam, Netherlands
+ 7 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 month and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Sacramento, California, United States; Gainesville, Florida, United States; Minneapolis, Minnesota, United States; St Louis, Missouri, United States; Richmond, Virginia, United States; Leuven, Belgium and 21 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.