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Phase III: Emicizumab in Type 3 VWD (WILL-EMI)

Clinical Trial Overview

A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease (WILL-EMI)

Objective

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).

VWD = von Willebrand disease; NIS = non-interventional study; SOC = Standard of care

Primary Endpoints

Annualized Bleed Rate (ABR) for Treated Bleeds in the Randomized Arms [Time Frame: From Baseline to at least 24 weeks]

Secondary Endpoints

ABR for All Bleeds in the Randomized Arms [Time Frame: From Baseline to at least 24 weeks]
ABR for Treated Spontaneous Bleeds in the Randomized Arms [Time Frame: From Baseline to at least 24 weeks]
ABR for Treated Joint Bleeds in the Randomized Arms [Time Frame: From Baseline to at least 24 weeks]
Intra-Participant Comparison of the ABR for Treated Bleeds with Prophylactic Emicizumab Versus Prophylactic SOC from the Preceeding Non-Interventional Study (NIS) WP45335 [Time Frame: From Baseline to at least 24 weeks]
Intra-Participant Comparison of the ABR for All Bleeds with Prophylactic Emicizumab Versus Prophylactic SOC from the Preceeding NIS WP45335 [Time Frame: From Baseline to at least 24 weeks]
Intra-Participant Comparison of the ABR for Treated Spontaneous Bleeds with Prophylactic Emicizumab Versus Prophylactic SOC from the Preceeding NIS WP45335 [Time Frame: From Baseline to at least 24 weeks]
Intra-Participant Comparison of the ABR for Treated Joint Bleeds with Prophylactic Emicizumab Versus Prophylactic SOC from the Preceeding NIS WP45335 [Time Frame: From Baseline to at least 24 weeks]
Incidence and Severity of Adverse Events, with Severity Determined According to the World Health Organization (WHO) Toxicity Grading Scale [Time Frame: From first dose of study treatment until 24 weeks after final dose of study treatment (up to 3 years, 11 months)]
Incidence and Severity of Thromboembolic Events [Time Frame: From first dose of study treatment until 24 weeks after final dose of study treatment (up to 3 years, 11 months)]
Incidence and Severity of Thrombotic Microangiopathy Events [Time Frame: From first dose of study treatment until 24 weeks after final dose of study treatment (up to 3 years, 11 months)]
Incidence and Severity of Injection-Site Reactions [Time Frame: From first dose of study treatment until 24 weeks after final dose of study treatment (up to 3 years, 11 months)]
Incidence of Adverse Events Leading to Drug Discontinuation [Time Frame: From first dose of study treatment until 24 weeks after final dose of study treatment (up to 3 years, 11 months)]
Incidence of Severe Hypersensitivity, Anaphylaxis, or Anaphylactoid Reactions [Time Frame: From first dose of study treatment until 24 weeks after final dose of study treatment (up to 3 years, 11 months)]
Incidence of Clinical Laboratory Abnormalities [Time Frame: From first dose of study treatment until 24 weeks after final dose of study treatment (up to 3 years, 11 months)]
Trough Plasma Concentration of Emicizumab at Prespecified Timepoints During the Treatment Period [Time Frame: Predose and at prespecified timepoints from first dose of emicizumab until study completion (up to 3 years, 11 months)]
Percentage of Participants with Anti-Drug Antibodies (ADAs) to Emicizumab at Baseline and with ADAs to Emicizumab During the Treatment Period [Time Frame: Baseline and at prespecified timepoints from first dose of emicizumab until study completion (up to 3 years, 11 months)]
Change from Baseline in Respiratory Rate Over Time [Time Frame: Baseline, Weeks 1, 2, 25, and every 12 weeks thereafter (weeks after switch to emicizumab for Arm B only) until study completion (up to 3 years, 11 months)]
Change from Baseline in Pulse Rate Over Time [Time Frame: Baseline, Weeks 1, 2, 25, and every 12 weeks thereafter (weeks after switch to emicizumab for Arm B only) until study completion (up to 3 years, 11 months)]
Change from Baseline in Systolic Blood Pressure Over Time [Time Frame: Baseline, Weeks 1, 2, 25, and every 12 weeks thereafter (weeks after switch to emicizumab for Arm B only) until study completion (up to 3 years, 11 months)]
Change from Baseline in Diastolic Blood Pressure Over Time [Time Frame: Baseline, Weeks 1, 2, 25, and every 12 weeks thereafter (weeks after switch to emicizumab for Arm B only) until study completion (up to 3 years, 11 months)]
Change from Baseline in Body Temperature Over Time [Time Frame: Baseline, Weeks 1, 2, 25, and every 12 weeks thereafter (weeks after switch to emicizumab for Arm B only) until study completion (up to 3 years, 11 months)]
Change from Baseline in Electrocardiogram (ECG) Parameters Over Time: QT, QTcF, RR, PR, and QRS Intervals [Time Frame: Baseline and study completion (up to 3 years, 11 months)]
Change from Baseline in Heart Rate Over Time, as Measured by Electrocardiogram (ECG) [Time Frame: Baseline and study completion (up to 3 years, 11 months)]
Change from Baseline in the PROMIS-29 Questionnaire Pain Interference Domain Score Over Time [Time Frame: Baseline and at prespecified timepoints until study completion (up to 3 years, 11 months)]
PROMIS-29 stands for Patient-Reported Outcomes Measurement Information System-29
Change from Baseline in the PROMIS-29 Questionnaire Fatigue Domain Score Over Time [Time Frame: Baseline and at prespecified timepoints until study completion (up to 3 years, 11 months)]
PROMIS-29 stands for Patient-Reported Outcomes Measurement Information System-29

VWD = von Willebrand disease
ABR = Annualized Bleed Rate
WHO = World Health Organization
SOC = Standard of care
PROMIS-29 = Patient-Reported Outcomes Measurement Information System-29
NIS = non-interventional study

Emicizumab

Other names: RG6013, ACE910
Modality: Bispecific monoclonal antibody

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Clinical Trial Overview

Inclusion and Exclusion Criteria

Enrollment and Resources

View on ClinicalTrials.gov

Inclusion and Exclusion Criteria

Key Inclusion Criteria

Key Exclusion Criteria

Arms A and B: Male and female participants Age ≥1 month at the time of signing Informed Consent/Assent Form
Arm C: Male and female participants Age ≥2 years at the time of signing Informed Consent/Assent Form
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:

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:

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

VWD = von Willebrand disease; SOC = Standard of care

Enrollment & Resources

Enrollment

For more information on eligibility criteria, view the study or reach out to our team.

Web

ClinicalTrials.gov
Identifier: NCT06998524

Phone

1-888-662-6728
(US and Canada)

Email

[email protected]

Clinical Trial Site Locations

Clinical Trial Site Locations

For more information on recruitment status, or where the study is being conducted

Visit ClinicalTrials.gov

Information is consistent with ClinicalTrials.gov as of December 12, 2025. Products under investigation have not been approved for use outside of the clinical trial setting. This information is presented only for the purposes of providing an overview of clinical trials and should not be construed as a recommendation for use of any product for unapproved purposes.

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ABR
Annualized Bleed Rate
NIS
non-interventional study
PROMIS-29
Patient-Reported Outcomes Measurement Information System-29
SOC
Standard of care
VWD
von Willebrand disease
WHO
World Health Organization