Emicizumab for Type 3 Von Willebrand Disease

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

Recruiting Phase 3 Interventional Study
Von Willebrand Disease, Type 3
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Pivotal Trial This treatment is in the last trial phase before FDA approval. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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Review the details below, then apply to join this clinical trial.

At a Glance
Age
1 Month and older
Sex
Any
Trial phase
Phase 3
Study type
Interventional
Purpose
Treatment
Participants needed
75 (estimated)
Sponsor
Hoffmann-La Roche · Industry
Who this trial is looking for

This trial is looking for people aged 1 month and older who have been diagnosed with Type 3 von Willebrand disease. Participants will receive either emicizumab treatment or continue their current therapy, depending on their previous treatment history.

Are You a Good Fit for This Trial?

You may be able to join if

  • I have been diagnosed with Type 3 von Willebrand disease
  • I have a medical record showing my von Willebrand factor inhibitor status
  • I have adequate blood, liver, and kidney function
  • I will follow the birth control requirements if I can get pregnant
  • I am at least 1 month old
  • I have used on-demand therapy for my condition recently
  • I have had at least 2 treated bleeds in the last 24 weeks

You may not be able to join if

  • I have another bleeding disorder besides Type 3 von Willebrand disease
  • I have had gastrointestinal bleeding in the last 18 months
  • I have a history of bleeding in the brain
  • I have been treated for blood clots or have signs of blood clots
  • I have conditions that increase my bleeding or blood clot risk
  • I have had severe allergic reactions to similar treatments
  • I am using certain immune-modulating medications

Summarized in plain language from this trial's official eligibility criteria. The full criteria are further down this page — only the research team can confirm whether you qualify.

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About This Trial
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 - emicizuma…
Trial Locations
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Eligibility Criteria
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 funct…
Contacts

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

888-662-6728 (U.S. Only)

global-roche-genentech-trials@gene.com

CONTACT

Fastest response: use the inquiry form. No email attachments. https://www.gene.com/contact-us/submit-medical-inquiry

CONTACT