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.
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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
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
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Investigational Site Number : 0560001
RECRUITINGBrussels, 1200, Belgium
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Investigational Site Number : 0560002
RECRUITINGBrussels, 1020, Belgium
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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
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Investigational Site Number : 3800002
RECRUITINGFlorence, Firenze, 50134, Italy
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Investigational Site Number : 3800003
RECRUITINGRozzano, Milano, 20089, Italy
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Investigational Site Number : 6160002
RECRUITINGWroclaw, Lower Silesian Voivodeship, 50-556, Poland
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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)
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Investigational Site Number : 7920002
RECRUITINGIzmir, 35100, Turkey (Türkiye)
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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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- A Once-a-Week shot could transform hemophilia Care—Even for those with inhibitors
- Can a clotting boost make surgery safer for hemophilia patients?
- Can a new clotting factor offer better bleed protection for severe hemophilia?
- A facelift without the bruising? tranexamic acid put to the test.
- Deep gum cleaning may boost quality of life for hemophilia patients
- New clotting factor aims to cut bleeding episodes in severe hemophilia a