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

NCT06998524 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 75

Last updated 2026-05-18

No results posted yet for this study

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).

Conditions

  • Von Willebrand Disease, Type 3

Interventions

DRUG

Emicizumab

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.

DRUG

von Willebrand Factor (VWF) Concentrates

Used according to local labeling or local treatment guidelines.

DRUG

Factor VIII (FVIII) Concentrates

Used according to local labeling or local treatment guidelines.

DRUG

von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates

Used according to local labeling or local treatment guidelines.

DRUG

Bypassing Agents

Used according to local labeling or local treatment guidelines.

Sponsors & Collaborators

Principal Investigators

  • Clinical Trials · Hoffmann-La Roche

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
1 Month
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-06-27
Primary Completion
2027-04-30
Completion
2029-04-30
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Canada
  • Colombia
  • France
  • Germany
  • Italy
  • Japan
  • Netherlands
  • Poland
  • South Africa
  • Spain
  • Sweden
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06998524 on ClinicalTrials.gov