Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A (NCT04131036)
🩺 Plain-English Summary (8th-Grade Level)
The investigators propose to study longitudinal joint and bone density changes in patients with severe Hemophilia A. Per current standard of care, most patients are on prophylactic FVIII replacement therapy intravenously several times weekly with a goal of keeping the trough \>1% FVIII. Recent phase 3 data suggest superior bleed protection with emicizumab prophylaxis every 1-2 weeks. It is the purpose of this study to longitudinally assess joint health and bone density over 3 years and to compare the effect of routine factor VIII prophylaxis with emicizumab prophylaxis.
Who can join: Review the eligibility criteria below.
Location: Orthopedic Hemophilia Treatment Center — Los Angeles, California
Age range: 16 Years
🧪 Interventions in This Study
- assessment of joint health and bone density (OTHER)
📋 "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 (4 Sites)
- Orthopedic Hemophilia Treatment Center — Los Angeles, California
- Hemophilia and Thrombosis Treatment Center, University of California, San Diego — San Diego, California
- The University of Texas Health Science Center at San Antonio — San Antonio, Texas
- Washington Center for Bleeding Disorders at Washington Institute for Coagulation — Seattle, Washington
📞 Contact the Study Coordinator
Phone: (206) 614-1200
Email: RKJ@WACBD.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.
More studies in Hemophilia A
- Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle" — Little Rock, Arkansas
- Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A — Los Angeles, California
- Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A — Milwaukee, Wisconsin
- A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A — Gainesville, Florida
- A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A — Gilbert, Arizona
- Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults — Mountain View, California
Explore by condition: All Hemophilia A clinical trials
💡 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.