Phase 3Recruiting

Comparing 4 approaches for type 3 von willebrand disease

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

A Phase III, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Prophylaxis in Patients With Type 3 Von Willebrand Disease

Condition: Von Willebrand Disease, Type 3Sponsor: Hoffmann-La RocheTarget enrollment: 75

Phase 3
4 groups
Sites in Toronto and Montreal
Recruiting

Interventions (5)

Medication
Emicizumab
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). During the extension period, participants may remain on maintenance dose of emicizumab 3 mg/kg Q2W, or change their emicizumab maintenance regimen to 1.5 mg/kg once every week (QW) or 6 mg/kg once every 4 weeks (Q4W), if they prefer and if agreed by the investigators.
Medication
von Willebrand Factor (VWF) Concentrates
Used according to local labeling or local treatment guidelines.
Medication
Factor VIII (FVIII) Concentrates
Used according to local labeling or local treatment guidelines.
Medication
von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates
Used according to local labeling or local treatment guidelines.
Medication
Bypassing Agents
Used according to local labeling or local treatment guidelines.

Canadian Sites (2)

2 of 2 recruiting

The Hospital for Sick Children
Toronto, Ontario
Recruiting
McGill University Health Center
Montreal, Quebec
Recruiting

See who this study is looking for19 criteria

The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.

Inclusion

+Age ≥1 month at the time of signing Informed Consent/Assent Form
+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
+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
+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:
+Have completed all study requirements as defined in the WP45335 protocol for at least 24 weeks
+For participants of childbearing potential: agreement to remain abstinent or adhere to the contraception requirements

Exclusion

−History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
−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
−Use of systemic immunomodulators (e.g., interferon) at enrollment or planned use during the study, with the exception of anti-retroviral therapy

5 concepts to explore on this page · up to 1,300 pointsTap any term to learn what it means.

In plain language

Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.

Learning

What this study is

This study is testing a treatment for a condition.

Phase 3 — a large study comparing this against the current standard, usually across many hospitals.

From the trial registry

Built from these fields:

designModule.designInfo.primaryPurpose
designModule.phases

Who receives what

There are 4 groups in this study.

Groups A, C and D receive Emicizumab.

Registry label: A: Arm A (Prior On-Demand SOC): Emicizumab Prophylaxis for 24 Weeks · C: Arm C (Prior Prophylaxis SOC): Emicizumab Prophylaxis for 24 Weeks · D: Treatment Extension Period for All Arms: Emicizumab Prophylaxis

Group B, the comparison group, receives one or more of: von Willebrand Factor (VWF) Concentrates, Factor VIII (FVIII) Concentrates, von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates and Bypassing Agents.

Registry label: B: Arm B (Prior On-Demand SOC): On-Demand SOC for 24 Weeks

From the trial registry

Built from these fields:

armsInterventionsModule.armGroups[].label
armsInterventionsModule.armGroups[].type
armsInterventionsModule.armGroups[].interventionNames

How the study is run

Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.

This study is open-label: everyone knows which treatment is being given, including you and the study team.

From the trial registry

Built from these fields:

designModule.designInfo.allocation
designModule.designInfo.maskingInfo.masking

Is there a placebo?

This study does not list a placebo group.

One group receives an existing treatment, so the two can be compared.

From the trial registry

Built from these fields:

armsInterventionsModule.armGroups[].type
armsInterventionsModule.armGroups[].interventionNames

Who the study is looking for

The study lists a minimum age of 1 month, with no upper limit given.

The study is open to people of any sex.

The study does not accept healthy volunteers.

These are the criteria the study lists. Only the trial site can decide whether someone can take part.

From the trial registry

Built from these fields:

eligibilityModule.minimumAge
eligibilityModule.sex
eligibilityModule.healthyVolunteers

How big and how long

The study aims to enrol about 75 people.

The study is currently expected to finish around April 2029.

The main measurement is taken over: From Baseline to at least 24 weeks.

From the trial registry

Built from these fields:

designModule.enrollmentInfo.count
statusModule.completionDateStruct.date
outcomesModule.primaryOutcomes[].timeFrame

What the study measures

Annualized Bleed Rate (ABR) for Treated Bleeds in the Randomized Arms — measured over From Baseline to at least 24 weeks.

From the trial registry

Built from these fields:

outcomesModule.primaryOutcomes[].measure
outcomesModule.primaryOutcomes[].timeFrame

Source: NCT06998524 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.

Not medical advice. What do these terms mean?

What is being tested — in plain terms

About EmicizumabDrug

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). During the extension period, participants may remain on maintenance dose of emicizumab 3 mg/kg Q2W, or change their emicizumab maintenance regimen to 1.5 mg/kg once every week (QW) or 6 mg/kg once every 4 weeks (Q4W), if they prefer and if agreed by the investigators.