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New study aims to unlock why some children with hemophilia reject treatment

NCT ID NCT07539402

Knowledge-focused Sponsor: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) 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 This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This observational study will follow 500 children with hemophilia A who use non-factor therapy to prevent bleeding. Researchers want to understand why some children develop antibodies against factor VIII while others do not. By studying blood and stool samples over time, they hope to uncover the immune mechanisms behind tolerance. The study does not test a new treatment but aims to inform future therapies.

What this could mean

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

What this could lead to
If successful, this study could reveal how the immune system learns to accept factor VIII, potentially guiding better treatments for hemophilia A.
What could go wrong
This is an observational study, not a treatment trial. It will not directly test a new therapy, and results may take years to translate into clinical practice.

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

About 500 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

May 2026

An estimate. Start dates often move.

Expected to finish

Mar 2029

An estimate. End dates often move.

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

Children with hemophilia A, who are treated with non-factor therapy as prophylaxis, and who are aged \<18 years. The subjects will be recruited from Hemophilia Treatment Centres (HTCs) in the Netherlanss and internationally.

Ages

Up to 18 years

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: * Congenital hemophilia A of all severities * Using NFT for prophylaxis * Aged under 18 years * Written informed consent Exclusion Criteria: * Acquired hemophilia A * Any other 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 Hemophilia A, congenital

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Amsterdam UMC, locatie AMC

    Amsterdam, North Holland, 1105AZ, Netherlands

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