New shot could cut bleeding episodes in severe hemophilia

NCT ID NCT05662319

Ongoing Disease control Sponsor: Sanofi Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 3 trial tests fitusiran, an injectable drug, in 91 males aged 12 and older with severe hemophilia A or B, with or without inhibitors. Participants first receive standard care for 6 months, then switch to fitusiran for about 3 years. The goal is to see if fitusiran reduces the number of bleeding episodes that require treatment.

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 new preventive treatment that reduces bleeding episodes in people with severe hemophilia, potentially reducing the need for frequent clotting factor infusions.

What could go wrong

This is a single-arm, open-label study with no placebo group, so results may be less definitive. Fitusiran carries risks like blood clotting issues, and long-term safety is still being evaluated.

Disclaimer Read more

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.

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 severe hemophilia A severe hemophilia B

As listed by the trial registrant

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

HEMOPHILIA

Contacts and locations

Show contact details

Locations

Center for Inherited Blood Disorders (CIBD) Site Number : 8400012

Orange, California, 92868-4306, United States
Children's Medical Center Dallas- Site Number : 8400018

Dallas, Texas, 75235, United States
Gulf States Hemophilia and Thrombophilia Center- Site Number : 8400002

Houston, Texas, 77030, United States
Hackensack University Site Number : 8400009

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

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

Hamilton, Ontario, L8N 3Z5, Canada
Investigational Site Number : 1240004

Toronto, Ontario, M5G 1X8, Canada
Investigational Site Number : 1560001

Guangzhou, 510515, China
Investigational Site Number : 1560002

Jinan, 250013, China
Investigational Site Number : 1560003

Beijing, 100045, China
Investigational Site Number : 1580001

Taipei, 100, Taiwan
Investigational Site Number : 1580003

Taipei, 11031, Taiwan
Investigational Site Number : 2500001

Paris, 75015, France
Investigational Site Number : 2500002

Lille, 59037, France
Investigational Site Number : 2500003

Le Kremlin-Bicêtre, 94275, France
Investigational Site Number : 2760001

Berlin, 10249, Germany
Investigational Site Number : 2760002

Hamburg, 20246, Germany
Investigational Site Number : 3000001

Athens, 115 27, Greece
Investigational Site Number : 3000002

Athens, 11527, Greece
Investigational Site Number : 3560001

Pune-411011, 411 011, India
Investigational Site Number : 3560003

Vellore, 632004, India
Investigational Site Number : 3560004

Bangalore, 560 034, India
Investigational Site Number : 3560006

Punjab, 141008, India
Investigational Site Number : 3560007

Bhubaneswar, 751019, India
Investigational Site Number : 3800001

Milan, 20122, Italy
Investigational Site Number : 3800003

Rozzano, Milano, 20089, Italy
Investigational Site Number : 3920001

Kashihara-shi, Nara, 634-8522, Japan
Investigational Site Number : 3920002

Saitama-shi, Saitama, 330-8777, Japan
Investigational Site Number : 3920003

Nagoya, Aichi-ken, 4668560, Japan
Investigational Site Number : 4100001

Seoul, Seoul-teukbyeolsi, 03722, South Korea
Investigational Site Number : 4100002

Seoul, Seoul-teukbyeolsi, 05278, South Korea
Investigational Site Number : 4840001

Veracruz, 91900, Mexico
Investigational Site Number : 4840002

Monterrey, Nuevo León, 64460, Mexico
Investigational Site Number : 4840004

Chihuahua City, 31000, Mexico
Investigational Site Number : 6160001

Warsaw, Masovian Voivodeship, 02-776, Poland
Investigational Site Number : 6160002

Krakow, Lesser Poland Voivodeship, 30-688, Poland
Investigational Site Number : 6160004

Lodz, Lódzkie, 93-510, Poland
Investigational Site Number : 6820002

Jeddah, 21423, Saudi Arabia
Investigational Site Number : 7100001

Parktown, 2193, South Africa
Investigational Site Number : 7100003

Johannesburg, 1501, South Africa
Investigational Site Number : 7240002

A Coruña, A Coruña [La Coruña], 15006, Spain
Investigational Site Number : 7240003

Zaragoza, 50009, Spain
Investigational Site Number : 7920001

Çapa, 34390, Turkey (Türkiye)
Investigational Site Number : 7920002

Adana, 01130, Turkey (Türkiye)
Investigational Site Number : 7920003

Izmir, TR-35100, Turkey (Türkiye)
Investigational Site Number : 7920004

Akdeniz, 07059, Turkey (Türkiye)
M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 8400016

Minneapolis, Minnesota, 55454, United States
Northwell Health Hemostasis and Thrombosis Center Site Number : 8400015

New Hyde Park, New York, 11040, United States
UPMC Children's Hospital of Pittsburgh-4401 Penn Ave Site Number : 8400017

Pittsburgh, Pennsylvania, 15224-1334, United States
University Hospitals of Cleveland Site Number : 8400001

Cleveland, Ohio, 44106, United States