Monthly shot could free kids with hemophilia from frequent infusions
NCT ID NCT03974113
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests fitusiran, a monthly injection, in boys aged 1 to under 12 with severe hemophilia A or B who have developed inhibitors (antibodies that block standard treatment). The goal is to find the right dose to safely lower a protein called antithrombin, which helps prevent bleeding episodes. Researchers will monitor side effects and drug levels in the blood.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
fitusiran
What this could lead to
If successful, fitusiran could offer a convenient, monthly injection to prevent bleeding episodes in young children with hemophilia who have developed inhibitors to standard treatments.
What could go wrong
This is an early-to-mid-stage trial with only 32 participants, so results may not apply to all children. Risks include potential side effects from lowering antithrombin levels, such as blood clots.
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 Los Angeles Site Number : 8400002
Los Angeles, California, 90027, United States
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Hackensack University Medical Center- Site Number : 8400008
Hackensack, New Jersey, 07601, United States
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Investigational Site Number : 1240001
Hamilton, Ontario, L8N 3Z5, Canada
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Investigational Site Number : 1240002
Ottawa, Ontario, K1H 8L1, Canada
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Investigational Site Number : 3560001
Pune-411011, 411 011, India
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Investigational Site Number : 3560002
Mumbai, 400 022, India
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Investigational Site Number : 3560004
Vellore, 632004, India
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Investigational Site Number : 3560006
Bangalore, 560034, India
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Investigational Site Number : 3800001
Milan, 20122, Italy
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Investigational Site Number : 3800002
Florence, 50134, Italy
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Investigational Site Number : 7240002
Madrid, Madrid, Comunidad de, 28046, Spain
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Investigational Site Number : 7920001
Adana, 01130, Turkey (Türkiye)
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Investigational Site Number : 7920002
Istanbul, 34093, Turkey (Türkiye)
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Investigational Site Number : 7920003
Izmir, TR-35100, Turkey (Türkiye)
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Penn State Milton S. Hershey Medical Center- Site Number : 8400006
Hershey, Pennsylvania, 17033, United States
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University Hospitals of Cleveland- Site Number : 8400007
Cleveland, Ohio, 44106, United States