New drug helps prevent bleeds in toddlers with hemophilia
NCT ID NCT05248594
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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Locations
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Children's Hospital at Montefiore
The Bronx, New York, 10467, United States
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Comprehensive Center for Hemophilia and Coagulation Disorders (Cornell)
New York, New York, 10065, United States
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Connecticut Children's Hemophilia Treatment Center - Connecticut Children's Medical Center
Hartford, Connecticut, 06106, United States
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Dartmouth Hitchcock Hemophilia Center - Mary Hitchcock Memorial Hospital
Lebanon, New Hampshire, 03766, United States
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Mary M Gooley Hemophilia Center, Inc.
Rochester, New York, 14621, United States
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Newark Beth Israel
Newark, New Jersey, 07112, United States
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Northwell Health Hemostasis and Thrombosis Center
Staten Island, New York, 11030, United States
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SUNY Upstate Hemophilia Treatment Center - HTC 058
Syracuse, New York, 13210, United States
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Western NY Blood Care - Research Foundation for SUNY
Buffalo, New York, 14202, United States
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Yale Hemophilia Treatment Center
New Haven, Connecticut, 06510, United States