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New hope for tiniest patients: emicizumab cuts bleeding in infants with hemophilia

NCT ID NCT05248594

First seen Nov 01, 2025 · Last updated May 22, 2026 · Updated 27 times

Summary

This study tested the real-world use of emicizumab in 71 infants and children aged 0-3 years with hemophilia A, a bleeding disorder. The goal was to see how many children could use the drug to prevent bleeding and how often breakthrough bleeds occurred. Results help doctors understand how well this treatment works in very young patients.

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Contacts and locations

Locations

  • Children's Hospital at Montefiore

    The Bronx, New York, 10467, United States

  • Comprehensive Center for Hemophilia and Coagulation Disorders (Cornell)

    New York, New York, 10065, United States

  • Connecticut Children's Hemophilia Treatment Center - Connecticut Children's Medical Center

    Hartford, Connecticut, 06106, United States

  • Dartmouth Hitchcock Hemophilia Center - Mary Hitchcock Memorial Hospital

    Lebanon, New Hampshire, 03766, United States

  • Mary M Gooley Hemophilia Center, Inc.

    Rochester, New York, 14621, United States

  • Newark Beth Israel

    Newark, New Jersey, 07112, United States

  • Northwell Health Hemostasis and Thrombosis Center

    Staten Island, New York, 11030, United States

  • SUNY Upstate Hemophilia Treatment Center - HTC 058

    Syracuse, New York, 13210, United States

  • Western NY Blood Care - Research Foundation for SUNY

    Buffalo, New York, 14202, United States

  • Yale Hemophilia Treatment Center

    New Haven, Connecticut, 06510, United States

Conditions

Explore the condition pages connected to this study.