New hope for rare bleeding disorder: emicizumab trial launches

NCT ID NCT05345197

First seen Jun 27, 2026 ยท Last updated Jun 27, 2026

Summary

This phase 2 trial tests the drug emicizumab (Hemlibra) as a regular injection to prevent bleeding in 51 adults with acquired hemophilia A, a rare condition where the immune system attacks clotting factors. Participants receive two loading doses then weekly shots, along with standard immune-suppressing therapy. The main goal is to see how many serious bleeds occur over 12 weeks.

What this could mean

Our plain-language read of the trial. This is informational only โ€” not medical advice or a prediction.

Active substance
emicizumab (Hemlibra)
What this could lead to
If successful, this could provide a new way to prevent dangerous bleeding episodes in people with acquired hemophilia A, reducing the need for frequent hospital visits.
What could go wrong
This is a small, early-phase trial with no comparison group. Risks include blood clots or other side effects, and the treatment may not work for everyone.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Bleeding and Clotting Disorders Institute

    Peoria, Illinois, 61614, United States

  • Emory University

    Atlanta, Georgia, 30308, United States

  • Georgetown University

    Washington D.C., District of Columbia, 20007, United States

  • Hemophilia Center of Western Pennsylvania

    Pittsburgh, Pennsylvania, 15213, United States

  • Indiana Hemophilia and Thrombosis Center, Inc.

    Indianapolis, Indiana, 46260, United States

  • Mayo Clinic

    Rochester, Minnesota, 55905, United States

  • Penn Blood Disorders Program, Hospital of the University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • Tulane University

    New Orleans, Louisiana, 70112-2632, United States

  • UCSD Hemophilia and Thrombosis Treatment Center

    San Diego, California, 92121, United States

  • UVA Comprehensive Cancer Center

    Charlottesville, Virginia, 22908, United States

  • University of North Carolina

    Chapel Hill, North Carolina, 27514, United States

  • University of Oklahoma Health Sciences Center

    Oklahoma City, Oklahoma, 73104, United States

  • University of Vermont Medical Center

    Burlington, Vermont, 05401, United States

  • Versiti Inc.

    Milwaukee, Wisconsin, 53226, United States

  • Washington Center for Bleeding Disorders

    Seattle, Washington, 98101, United States

  • Washington University

    St Louis, Missouri, 63110, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.