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A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia
Sponsor: Sanofi
Summary
This is a multicenter, multinational, open-label, one-way cross-over, Phase 3, single-arm study for treatment of hemophilia. The purpose of this study is to measure the frequency of treated bleeding episodes with fitusiran in male adult and adolescent (≥12 years old) participants with hemophilia A or B, with or without inhibitory antibodies to factor VIII or IX who have switched from their prior standard of care treatment. The total study duration will be up to approximately 50 months (200 weeks, 1 study month is equivalent to 4 weeks) and will include: * A screening period up to approximately 60 days, * A standard of care (SOC) period of approximately 6 study months (24 weeks), * A fitusiran treatment period of approximately 36 study months (144 weeks), * An antithrombin (AT) follow-up period of approximately 6 study months (24 weeks) but may be shorter or longer depending on individual participants AT recovery. The frequency for telephone visits will be approximately every 2 weeks. For site visits the frequency will be approximately every 8 weeks during the SOC period and approximately every 4 weeks during the fitusiran treatment period. If applicable and if allowed by local regulation, home and/or remote visits may be conducted during the study
Official title: A Phase 3, Single-arm, Multicenter, Multinational, Open-label, One-way Crossover Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged ≥ 12 Years With Severe Hemophilia A or B With or Without Inhibitory Antibodies to Factor VIII or IX
Key Details
Gender
MALE
Age Range
12 Years - Any
Study Type
INTERVENTIONAL
Enrollment
91
Start Date
2023-02-01
Completion Date
2029-01-25
Last Updated
2026-02-04
Healthy Volunteers
No
Conditions
Interventions
Fitusiran
Pharmaceutical form: Solution for injection Route of administration: Subcutaneous (SC)
Clotting factor concentrates (CFC) or bypassing agents (BPA)
* Coagulation factor VIII (ATC code: B02BD02) * Coagulation factor IX (ATC code: B02BD04) * Coagulation factor VIIa (ATC code: B02BD08) * Factor VIII inhibitor bypassing activity (ATC code: B02BD03) Pharmaceutical form: Solution for infusion Route of administration: Intravenous (IV)
Antithrombin concentrate (ATIIIC)
Antithrombin III (ATC code: B01AB02) Pharmaceutical form: Solution for infusion Route of administration: Intravenous (IV)
Locations (50)
Center for Inherited Blood Disorders (CIBD) Site Number : 8400012
Orange, California, United States
M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 8400016
Minneapolis, Minnesota, United States
Hackensack University Site Number : 8400009
Hackensack, New Jersey, United States
Northwell Health Hemostasis and Thrombosis Center Site Number : 8400015
New Hyde Park, New York, United States
University Hospitals of Cleveland Site Number : 8400001
Cleveland, Ohio, United States
UPMC Children's Hospital of Pittsburgh-4401 Penn Ave Site Number : 8400017
Pittsburgh, Pennsylvania, United States
Children's Medical Center Dallas- Site Number : 8400018
Dallas, Texas, United States
Gulf States Hemophilia and Thrombophilia Center- Site Number : 8400002
Houston, Texas, United States
Investigational Site Number : 1240001
Hamilton, Ontario, Canada
Investigational Site Number : 1240002
Hamilton, Ontario, Canada
Investigational Site Number : 1240004
Toronto, Ontario, Canada
Investigational Site Number : 1560003
Beijing, China
Investigational Site Number : 1560001
Guangzhou, China
Investigational Site Number : 1560002
Jinan, China
Investigational Site Number : 2500003
Le Kremlin-Bicêtre, France
Investigational Site Number : 2500002
Lille, France
Investigational Site Number : 2500001
Paris, France
Investigational Site Number : 2760001
Berlin, Germany
Investigational Site Number : 2760002
Hamburg, Germany
Investigational Site Number : 3000001
Athens, Greece
Investigational Site Number : 3000002
Athens, Greece
Investigational Site Number : 3560004
Bangalore, India
Investigational Site Number : 3560007
Bhubaneswar, India
Investigational Site Number : 3560001
Pune-411011, India
Investigational Site Number : 3560006
Punjab, India
Investigational Site Number : 3560003
Vellore, India
Investigational Site Number : 3800003
Rozzano, Milano, Italy
Investigational Site Number : 3800001
Milan, Italy
Investigational Site Number : 3920003
Nagoya, Aichi-ken, Japan
Investigational Site Number : 3920001
Kashihara-shi, Nara, Japan
Investigational Site Number : 3920002
Saitama-shi, Saitama, Japan
Investigational Site Number : 4840002
Monterrey, Nuevo León, Mexico
Investigational Site Number : 4840004
Chihuahua City, Mexico
Investigational Site Number : 4840001
Veracruz, Mexico
Investigational Site Number : 6160002
Krakow, Lesser Poland Voivodeship, Poland
Investigational Site Number : 6160004
Lodz, Lódzkie, Poland
Investigational Site Number : 6160001
Warsaw, Masovian Voivodeship, Poland
Investigational Site Number : 6820002
Jeddah, Saudi Arabia
Investigational Site Number : 7100003
Johannesburg, South Africa
Investigational Site Number : 7100001
Parktown, South Africa
Investigational Site Number : 4100001
Seoul, Seoul-teukbyeolsi, South Korea
Investigational Site Number : 4100002
Seoul, Seoul-teukbyeolsi, South Korea
Investigational Site Number : 7240002
A Coruña, A Coruña [La Coruña], Spain
Investigational Site Number : 7240003
Zaragoza, Spain
Investigational Site Number : 1580001
Taipei, Taiwan
Investigational Site Number : 1580003
Taipei, Taiwan
Investigational Site Number : 7920002
Adana, Turkey (Türkiye)
Investigational Site Number : 7920004
Akdeniz, Turkey (Türkiye)
Investigational Site Number : 7920001
Çapa, Turkey (Türkiye)
Investigational Site Number : 7920003
Izmir, Turkey (Türkiye)