A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia
NCT ID: NCT05662319
Last Updated: 2026-02-04
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
91 participants
INTERVENTIONAL
2023-02-01
2029-01-25
Brief Summary
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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
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Detailed Description
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Conditions
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Study Design
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NA
CROSSOVER
TREATMENT
NONE
Study Groups
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All participants
In standard of care (SOC) period, participants will receive on-demand or prophylactic treatment with clotting factor concentrates (CFCs) or bypassing agents (BPAs) for 6 months (from Day -168 to Day -1). In fitusiran treatment period, participants will receive subcutaneous fitusiran prophylaxis once every other month (Q2M) or once monthly (QM) for 36 months (from Day 1 to Day 1009). In case of bleeding events participants will receive IV CFCs or BPAs. Participants may receive Antithrombin concentrate (ATIIIC) as rescue medicine.
.
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)
Interventions
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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)
Eligibility Criteria
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Inclusion Criteria
* For participants currently not on prophylaxis (CFC or BPA on-demand): A minimum of 4 bleeding episodes requiring BPA (inhibitor participants) or CFC (non-inhibitor participants) treatment within the last 6 months prior to screening.
* Willing and able to comply with the study requirements and to provide written informed consent and assent in the case of participants under the age of legal consent, per local and national requirements
Exclusion Criteria
* History of arterial or venous thromboembolism, not associated with an indwelling venous access
* History of intolerance to SC injection(s).
* Current participation in immune tolerance induction therapy (ITI)
* Prior gene therapy
* Current or prior participation in a fitusiran trial
* Current or prior participation in a gene therapy trial
* Received an investigational drug or device within 30 days prior to the screening visit or within 5 half-lives of the investigational drug (or device) prior to the screening visit, whichever is longer
* Presence of clinically significant liver disease AT activity \<60% at Screening
* Co-existing thrombophilic disorder
* Hepatitis C virus antibody positive, except participants who have negative Hepatitis C viral load and no evidence of cirrhosis
* Presence of acute hepatitis, ie, hepatitis A, hepatitis E.
* Presence of acute or chronic hepatitis B infection
* Known to be HIV positive with CD4 count \<200 cells/μL.
* Reduced renal function The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
12 Years
MALE
No
Sponsors
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Sanofi
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Sciences & Operations
Role: STUDY_DIRECTOR
Sanofi
Locations
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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)
Countries
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Related Links
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EFC17574 Plain Language Results Summaries
Other Identifiers
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U1111-1275-9584
Identifier Type: REGISTRY
Identifier Source: secondary_id
2022-500221-33
Identifier Type: REGISTRY
Identifier Source: secondary_id
EFC17574
Identifier Type: -
Identifier Source: org_study_id
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