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

Conditions

• Von Willebrand Disease, Type 3

Eligibility Criteria

Sex

ALL

Age

1 Month - N/A

Healthy Volunteers

Not accepted

Interventions

• Emicizumab — DRUG
  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 — DRUG
  Used according to local labeling or local treatment guidelines.
• Factor VIII (FVIII) Concentrates — DRUG
  Used according to local labeling or local treatment guidelines.
• von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates — DRUG
  Used according to local labeling or local treatment guidelines.
• Bypassing Agents — DRUG
  Used according to local labeling or local treatment guidelines.

Study Details

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).

Key Dates

Start date

Jun 27, 2025

Status verified

May 2026

Primary completion

Apr 30, 2027

Completion

Apr 30, 2029

Study Design

Enrollment

75 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

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.

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

• Reference Study ID Number: WP45338 https://forpatients.roche.com/
  888-662-6728 (U.S. Only)
  [email protected]

Locations (4)

Find similar trials in Sacramento, CA

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