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

ConditionVon Willebrand Disease, Type 3

Trial statusRecruiting

Trial phasePhase 3

Trial typeInterventional

Biological sexAll

Age1+

SponsorHoffmann-La Roche

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 - 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).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Parallel

Treatments tested in this trial

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.

Treatment groups

75 Participants

are divided into 4 treatment groups

Group A: Arm A (Prior On-Demand SOC): Emicizumab Prophylaxis for 24 WeeksExperimental treatment 1 intervention

Group B: Arm B (Prior On-Demand SOC): On-Demand SOC for 24 WeeksActive comparator 4 interventions

Group C: Arm C (Prior Prophylaxis SOC): Emicizumab Prophylaxis for 24 WeeksExperimental treatment 1 intervention

Group D: Treatment Extension Period for All Arms: Emicizumab ProphylaxisExperimental treatment 1 intervention