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Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A

NCT05500807 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 40

Last updated 2026-04-03

No results posted yet for this study

Summary

Von Willebrand Disease (VWD) is the most common inherited bleeding disorder affecting up to 0.1% of the population, is usually characterized by mucocutaneous bleeding, HMB, surgical bleeding or other hemostatic challenges. Severe bleeding events require VWF concentrates administered solely through intravenous access. Emicizumab (Hemlibra) is a monoclonal bispecific antibody developed to bind activated FIX and FX and mimic FVIII cofactor functionality. Hemlibra is administered via subcutaneous injection rather than intravenous infusion. The hypothesis of this study is that Emicizumab is safe and efficacious for prophylaxis in severe VWD and concomitant VWD/hemophilia patients.

Conditions

  • Von Willebrand Disease, Type 3
  • Concomitant VWD and Hemophilia

Interventions

DRUG

Emicizumab

Subcutaneous injection of emicizumab for prophylaxis

Sponsors & Collaborators

  • Genentech, Inc.

    collaborator INDUSTRY

  • Bleeding and Clotting Disorders Institute Peoria, Illinois

    lead OTHER

Principal Investigators

  • Jonathan C Roberts, MD · Bleeding and Clotting Disorders Institute

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Years
Max Age
90 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-11-01
Primary Completion
2027-06-30
Completion
2028-02-29
FDA Drug
Yes

Countries

  • United States

Study Locations

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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 NCT05500807 on ClinicalTrials.gov