Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A (NCT05500807)
Emicizumab for Severe VON Willebrand Disease (VWD) and VWD/Hemophilia A
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: The Center for Comprehensive Care and Diagnosis of Inherited Blood Disorders (CIBD) — Orange, California
Age range: 0 Years to 90 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (12 Sites)
- The Center for Comprehensive Care and Diagnosis of Inherited Blood Disorders (CIBD) — Orange, California
- Stanford University: Stanford Children's Health — Redwood City, California
- University of Miami - Miller School of Medicine — Coral Gables, Florida
- St. Joseph's Children's Hospital - Center for Bleeding and Clotting Disorders — Tampa, Florida
- Bleeding and Clotting Disorders Institute (BCDI) — Peoria, Illinois
- Innovative Hematology, Inc. (IHI) — Indianapolis, Indiana
- University of Michigan Medical School — Ann Arbor, Michigan
- Central Michigan University: Children's Hospital of Michigan — Mount Pleasant, Michigan
- Children's Mercy Hospital — Kansas City, Missouri
- Penn State College of Medicine — Hershey, Pennsylvania
- Washington Center for Bleeding Disorders — Seattle, Washington
- UW Health Comprehensive Program for Bleeding Disorders — Madison, Wisconsin
📞 Contact the Study Coordinator
Phone: (309) 692-5337
Email: meganw@ilbcdi.org
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
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💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.