New drug helps prevent bleeds in toddlers with hemophilia

NCT ID NCT05248594

Completed Disease control Sponsor: Montefiore Medical Center Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This study looked at how the drug emicizumab (Hemlibra) works in real-world settings for infants and children up to 3 years old with hemophilia A, a bleeding disorder. Researchers tracked 71 children to see how many were using emicizumab to prevent bleeds and whether any breakthrough bleeding occurred. The goal was to understand how well the drug controls bleeding in very young patients.

What this could mean

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

Active substance: emicizumab (Hemlibra)
What this could lead to: If successful, this could show that emicizumab is a safe and effective option for preventing bleeds in very young children with hemophilia A.
What could go wrong: This is an observational study, not a controlled trial, so results may be less definitive. Breakthrough bleeding can still occur while on the drug.

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.

hemophilia A

As listed by the trial registrant

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

HEMOPHILIA A

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

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