New shot could cut bleeds in kids with hemophilia
NCT ID NCT07285460
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 3 trial is testing a drug called fitusiran in about 85 boys aged 1 to under 12 with severe hemophilia A or B, including those with inhibitors that make standard treatments less effective. The drug is given as a regular shot under the skin to help prevent bleeding episodes. The study compares how many bleeds occur during fitusiran treatment versus standard care.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
fitusiran (a drug given as a shot under the skin to help prevent bleeding)
What this could lead to
If it works, this could give young boys with severe hemophilia a new option to prevent bleeds with fewer injections than current treatments.
What could go wrong
This is still being tested in a relatively small group, and fitusiran may not work for everyone. There are also risks like blood clots or liver problems that need careful monitoring.
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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Cure 4 The Kids Foundation- Site Number : 8400001
RECRUITINGLas Vegas, Nevada, 89147, United States
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Hackensack Meridian Health - Hackensack University Medical Center- Site Number : 8400008
RECRUITINGHackensack, New Jersey, 07601, United States
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Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo- Site Number : 0760004
RECRUITINGSão Paulo, 05403-000, Brazil
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Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto- Site Number : 0760003
RECRUITINGRibeirão Preto, São Paulo, 14051140, Brazil
Contact Phone: •••-•••-••••
Contact
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Investigational Site Number : 0560001
RECRUITINGBrussels, 1200, Belgium
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Investigational Site Number : 0560002
RECRUITINGBrussels, 1020, Belgium
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Investigational Site Number : 1240001
RECRUITINGHamilton, Ontario, L8N 3Z5, Canada
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Investigational Site Number : 1240003
RECRUITINGMontreal, Quebec, H3T 1C5, Canada
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Investigational Site Number : 1560001
RECRUITINGBeijing, 100045, China
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Investigational Site Number : 1580001
RECRUITINGTaipei, 100, Taiwan
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Investigational Site Number : 1580002
RECRUITINGTaichung, 402, Taiwan
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Investigational Site Number : 3560001
RECRUITINGPune, 411004, India
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Investigational Site Number : 3560002
RECRUITINGMumbai, 400022, India
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Investigational Site Number : 3560003
RECRUITINGVellore, 632004, India
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Investigational Site Number : 3560004
RECRUITINGBengaluru, 560034, India
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Investigational Site Number : 3800001
RECRUITINGMilan, Milano, 20122, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Investigational Site Number : 3800002
RECRUITINGFlorence, Firenze, 50134, Italy
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Investigational Site Number : 3800003
RECRUITINGRozzano, Milano, 20089, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Investigational Site Number : 6160002
RECRUITINGWroclaw, Lower Silesian Voivodeship, 50-556, Poland
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Investigational Site Number : 6420001
RECRUITINGIași, 700309, Romania
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Investigational Site Number : 6420003
RECRUITINGTimișoara, 300011, Romania
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Investigational Site Number : 6820001
RECRUITINGRiyadh, 12746, Saudi Arabia
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Investigational Site Number : 7240001
RECRUITINGEsplugues de Llobregat, Barcelona [Barcelona], 08950, Spain
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Investigational Site Number : 7240002
RECRUITINGMadrid, 28046, Spain
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Investigational Site Number : 7240003
RECRUITINGZaragoza, 50009, Spain
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Investigational Site Number : 7920001
RECRUITINGIstanbul, 34093, Turkey (Türkiye)
Contact
Contact Phone: •••-•••-••••
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Investigational Site Number : 7920002
RECRUITINGIzmir, 35100, Turkey (Türkiye)
Contact Phone: •••-•••-••••
Contact
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The Center for Inherited Blood Disorders- Site Number : 8400009
RECRUITINGOrange, California, 92868, United States
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The Luskin Orthopaedic Institute for Children- Site Number : 8400013
RECRUITINGLos Angeles, California, 90007, United States
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University of Texas - Southwestern Medical Center- Site Number : 8400015
RECRUITINGDallas, Texas, 75390, United States