Emicizumab prophylaxis for people with Type 3 von Willebrand disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase III, multicenter, open-label study testing emicizumab as prophylaxis in participants with confirmed Type 3 von Willebrand disease. Participants currently using on-demand therapy are randomized to emicizumab prophylaxis or continuation of on-demand standard of care, while participants already on prophylactic standard therapy will receive emicizumab and be compared to their own prior non-interventional study data (WP45335). The trial measures bleeding outcomes along with safety, pharmacokinetics, and pharmacodynamics, and allows use of VWF/FVIII concentrates or bypassing agents as clinically needed. Eligible ages start at 1 month for the on-demand/randomized arms and at 2 years for the prophylaxis arm, with required baseline organ function and documentation of prior treatment patterns.

Why it matters

Who can join: the full rules

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

Where it's running

• UC Davis
  Sacramento, California, United States Recruiting
• University of Florida
  Gainesville, Florida, United States Recruiting
• University of Kentucky Children's Hospital
  Lexington, Kentucky, United States Recruiting
• University of Minnesota Medical Center
  Minneapolis, Minnesota, United States Recruiting
• Washington University School of Medicine
  St Louis, Missouri, United States Recruiting
• Virginia Commonwealth University
  Richmond, Virginia, United States Recruiting

Study contacts

• Study coordinator: Reference Study ID Number: WP45338 https://forpatients.roche.com/
• Email: global-roche-genentech-trials@gene.com
• Phone: 888-662-6728 (U.S. Only)

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Emicizumab treatment for severe Von Willebrand Disease and Hemophilia A

  This study is testing if a new injection called Emicizumab can help people with severe Von Willebrand Disease or both Von Willebrand Disease and Hemophilia A prevent serious bleeding better than current treatments.

  Recruiting at 12 sites Phase 1 Bleeding and Clotting Disorders Institute Peoria, Illinois · Orange, California and 11 other locations · NCT05500807

• NXT007 versus emicizumab to prevent bleeds in people with Hemophilia A

  This will test whether NXT007 prevents bleeding better than emicizumab in people aged 12 and older with Hemophilia A, including those with or without factor VIII inhibitors.

  Recruiting at 63 sites Phase 3 Hoffmann-La Roche · Orange, California and 62 other locations · NCT07416604

• Switching from Emicizumab to Marstacimab in Severe Hemophilia A

  This study is testing if switching from emicizumab to marstacimab is safe and how well the body handles the new medication in people with severe hemophilia A.

  Recruiting at 7 sites Phase 1 Pfizer · La Jolla, California and 9 other locations · NCT06703606

• Testing fitusiran to prevent bleeds in severe hemophilia A patients switching from emicizumab

  This study is testing if fitusiran can help prevent bleeding in men with severe hemophilia A who are switching from another treatment called emicizumab.

  Recruiting at 3 sites Phase 4 Sanofi · Los Angeles, California and 2 other locations · NCT06145373

• Using Nuwiq to manage bleeding in hemophilia A patients during major surgery

  This study is testing if Nuwiq can help prevent bleeding during major surgery for boys and men over 12 with severe hemophilia A who are already using another treatment called emicizumab.

  Recruiting at 17 sites Phase 4 Octapharma · Dallas, Texas and 17 other locations · NCT05935358

• Understanding antibody changes in hemophilia A patients treated with emicizumab

  This study looks at how the immune system reacts to emicizumab treatment in people with severe hemophilia A by checking leftover samples from their regular doctor visits.

  Recruiting Goethe University · Frankfurt am Main, Hesse · NCT05802836

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.