A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebran… (NCT06998524) | Clinical Trial Compass
RecruitingPhase 3
A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease
United States, Belgium75 participantsStarted 2025-06-27
Plain-language 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).
Who can participate
Age range1 Month
SexALL
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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
* O…
What they're measuring
1
Annualized Bleed Rate (ABR) for Treated Bleeds in the Randomized Arms
Timeframe: From Baseline to at least 24 weeks
Trial details
NCT IDNCT06998524
SponsorHoffmann-La Roche
Sponsor typeINDUSTRY
Study typeINTERVENTIONAL
Primary completion2027-03-30
Contact for this trial
Reference Study ID Number: WP45338 https://forpatients.roche.com/