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New drug helps prevent bleeds in toddlers with hemophilia

NCT ID NCT05248594

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Participants

71 people

The number who actually took part.

Started

Aug 2024

Finished

Jul 2025

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Patients 0 to 36 months of age, diagnosed with severe, moderate or mild Hemophlia A at the time of initiating treatment with Emicizumab.

Ages

Up to 36 months

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: Patients must meet the following criteria for study entry: * Patients who have been prescribed Emicizumab * Patients who are 0-36 months of age at the time of starting treatment with Emicizumab * Diagnosis of congenital mild, moderate or severe hemophilia with or without an inhibitor Exclusion Criteria: * Patients with acquired Hemophilia A * Patients with Hemophilia A and another congenital or acquired bleeding disorder.

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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

More trials for these conditions

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