Recruiting
Phase 3

Emicizumab

Sponsor:

Hoffmann-La Roche

Code:

NCT06998524

Conditions

Von Willebrand Disease, Type 3

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Interventions

Emicizumab

von Willebrand Factor (VWF) Concentrates

Factor VIII (FVIII) Concentrates

von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates

Bypassing Agents

Study Details

Brief summary:

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).

Conditions

Von Willebrand Disease, Type 3

Study ID

NCT06998524

Start date

Jun 27, 2025

Status verified date

Aug, 2026

Completion date

Apr 30, 2029

Anticipated

Primary completion date

Apr 30, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

75 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Arm A (Prior On-Demand SOC): Emicizumab Prophylaxis for 24 Weeks

Participants who are randomized to Arm A, taking on-demand standard of care (SOC) treatment at the time of study entry (and for at least 24 weeks prior to enrollment), will receive emicizumab SC prophylaxis.

active comparator: Arm B (Prior On-Demand SOC): On-Demand SOC for 24 Weeks

Participants who are randomized to Arm B, taking on-demand standard of care (SOC) treatment at the time of study entry (and for at least 24 weeks prior to enrollment), will continue to receive their current SOC on-demand treatment until Week 24.

experimental: Arm C (Prior Prophylaxis SOC): Emicizumab Prophylaxis for 24 Weeks

Participants who enroll in Arm C, taking SOC prophylactic treatment at the time of study entry and for at least 24 weeks of observation during the preceding NIS WP45335, will receive emicizumab SC prophylaxis.

experimental: Treatment Extension Period for All Arms: Emicizumab Prophylaxis

Participants in Arms A and C who have completed 24 weeks of emicizumab prophylaxis and who derive benefit from emicizumab will have the opportunity to continue to receive emicizumab prophylaxis in the extension period. Participants in Arm B who have completed 24 weeks of SOC on-demand treatment will have the opportunity to receive emicizumab prophylaxis in the extension period.

Interventions

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.

von Willebrand Factor (VWF) Concentrates

Used according to local labeling or local treatment guidelines.

Factor VIII (FVIII) Concentrates

Used according to local labeling or local treatment guidelines.

von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates

Used according to local labeling or local treatment guidelines.

Bypassing Agents

Used according to local labeling or local treatment guidelines.

Primary outcome measure

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

Central Contacts and Locations

Central contacts

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

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

Locations

UC Davis

Recruiting

Sacramento, California, United States, 95817

University of Florida

Recruiting

Gainesville, Florida, United States, 32610

University of Minnesota Medical Center

Recruiting

Minneapolis, Minnesota, United States, 55455

Washington University School of Medicine

Recruiting

St Louis, Missouri, United States, 63110

Virginia Commonwealth University

Recruiting

Richmond, Virginia, United States, 23298-0461

The Hospital for Sick Children

Recruiting

Toronto, Ontario, Canada, M5G 1X8

McGill University Health Center

Recruiting

Montreal, Quebec, Canada, H4A 3J1

More Information

Sponsor

Hoffmann-La Roche

Last update posted

Aug 10, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Hoffmann-La Roche on 2026-08-10.