Monthly shot could free kids with hemophilia from frequent infusions

NCT ID NCT03974113

Ongoing Disease control Sponsor: Genzyme, a Sanofi Company Source: ClinicalTrials.gov ↗

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

hemophilia hemophilia A hemophilia B

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HEMOPHILIA

Contacts and locations

Locations

Children's Hospital Los Angeles Site Number : 8400002

Los Angeles, California, 90027, United States
Hackensack University Medical Center- Site Number : 8400008

Hackensack, New Jersey, 07601, United States
Investigational Site Number : 1240001

Hamilton, Ontario, L8N 3Z5, Canada
Investigational Site Number : 1240002

Ottawa, Ontario, K1H 8L1, Canada
Investigational Site Number : 3560001

Pune-411011, 411 011, India
Investigational Site Number : 3560002

Mumbai, 400 022, India
Investigational Site Number : 3560004

Vellore, 632004, India
Investigational Site Number : 3560006

Bangalore, 560034, India
Investigational Site Number : 3800001

Milan, 20122, Italy
Investigational Site Number : 3800002

Florence, 50134, Italy
Investigational Site Number : 7240002

Madrid, Madrid, Comunidad de, 28046, Spain
Investigational Site Number : 7920001

Adana, 01130, Turkey (Türkiye)
Investigational Site Number : 7920002

Istanbul, 34093, Turkey (Türkiye)
Investigational Site Number : 7920003

Izmir, TR-35100, Turkey (Türkiye)
Penn State Milton S. Hershey Medical Center- Site Number : 8400006

Hershey, Pennsylvania, 17033, United States
University Hospitals of Cleveland- Site Number : 8400007

Cleveland, Ohio, 44106, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.