Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A (NCT04644575)
A Phase 3 Open-label, Multicenter Study of the Long-term Safety and Efficacy of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients With Severe Hemophilia A
🩺 Plain-English Summary (8th-Grade Level)
Primary Objective: \- To evaluate the long-term safety of BIVV001 in previously treated subjects with hemophilia A Secondary Objectives: * To evaluate the efficacy of BIVV001 as a prophylaxis treatment. * To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes. * To evaluate BIVV001 consumption for prevention and treatment of bleeding episodes. * To evaluate the effect of BIVV001 prophylaxis on joint health outcomes. * To evaluate the effect of BIVV001 prophylaxis on Quality of Life (QoL) outcomes. * To evaluate the safety and tolerability of BIVV001 treatment. * To assess the PK of BIVV001 based on the one stage activated partial thromboplastin time (aPTT) and two-stage chromogenic FVIII activity assays (only applicable to Arm B). * To evaluate the efficacy of BIVV001 for perioperative management
Who can join: Review the eligibility criteria below.
Location: Orthopaedic Institute for Children Site Number : 8400003 — Los Angeles, California
🧪 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 (85 Sites)
Showing the first 25 of 85 sites.
- Orthopaedic Institute for Children Site Number : 8400003 — Los Angeles, California
- Children's Hospital Los Angeles Site Number : 8400009 — Los Angeles, California
- University of California San Diego Site Number : 8400007 — San Diego, California
- University of Florida Health Site Number : 8400008 — Gainesville, Florida
- Children's Healthcare of Atlanta Site Number : 8400016 — Atlanta, Georgia
- Rush University Medical Center Site Number : 8400010 — Chicago, Illinois
- Children's Hospital Of Iowa Site Number : 8400011 — Iowa City, Iowa
- University of Michigan Medical Center Site Number : 8400006 — Ann Arbor, Michigan
- Michigan State University School Of Med Site Number : 8400002 — East Lansing, Michigan
- Hemostasis and Thrombosis Center of Nevada Site Number : 8400001 — Las Vegas, Nevada
- New York Presbyterian Hospital/Weill Cornell Medical Center Site Number : 8400017 — New York, New York
- East Carolina University -2390 Hemby Ln Site Number : 8400015 — Greenville, North Carolina
- Cincinnati Children's Hospital Medical Center Site Number : 8400012 — Cincinnati, Ohio
- Children's Research Institute Site Number : 8400013 — Columbus, Ohio
- Bloodworks Northwest Site Number : 8400005 — Seattle, Washington
- Children's Hospital of Wisconsin Site Number : 8400014 — Milwaukee, Wisconsin
- Investigational Site Number : 0320001 — CABA, Buenos Aires F.D.
- Investigational Site Number : 0320002 — Godoy Cruz, Mendoza Province
- Investigational Site Number : 0320003 — Buenos Aires,
- Investigational Site Number : 0360004 — Camperdown, New South Wales
- Investigational Site Number : 0360001 — Westmead, New South Wales
- Investigational Site Number : 0360002 — South Brisbane, Queensland
- Investigational Site Number : 0360003 — Murdoch, Western Australia
- Investigational Site Number : 0560003 — Woluwe-Saint-Lambert,
- Hemocentro Campinas - UNICAMP Site Number : 0760001 — Campinas, São Paulo
💰 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
- 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.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.