Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX
NCT ID: NCT03754790
Last Updated: 2025-12-17
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
281 participants
INTERVENTIONAL
2019-01-09
2026-11-05
Brief Summary
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To characterize the long-term safety and tolerability of fitusiran
Secondary Objectives:
* To characterize the efficacy and long-term efficacy of fitusiran as assessed by the frequency of:
* Bleeding episodes
* Spontaneous bleeding episodes
* Joint bleeding episodes
* To characterize the effects of fitusiran on health-related quality of life (HRQOL) measures in participants ≥17 years of age
Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Fitusiran
Participants will be administered fitusiran as a subcutaneous injection once monthly or every other month for up to 48 months post initiation of modified IMP dose/frequency or until fitusiran becomes commercially available, whichever comes first.
Fitusiran
Pharmaceutical form:solution for injection Route of administration: subcutaneous
Interventions
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Fitusiran
Pharmaceutical form:solution for injection Route of administration: subcutaneous
Eligibility Criteria
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Inclusion Criteria
* Participants with severe hemophilia A or B who have completed a Phase 3 fitusiran clinical trial
* Male
* Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol. In countries where legal age of majority is above 18 years, a specific ICF must also be signed by the participant's legally authorized representative
Exclusion Criteria
* Current participation in immune tolerance induction treatment (ITI)
* Current use of factor concentrates or bypassing agents (BPAs) as regularly administered prophylaxis designed to prevent spontaneous bleeding episodes except for participants requiring factor concentrates or BPAs prophylaxis during the study dosing pause period
* Use of compounds other than factor concentrates or BPAs for hemophilia treatment
* Current or prior participation in a gene therapy trial
* Alanine aminotransferase (ALT) and/or Aspartate aminotransferase (AST) \>1.5 × upper limit of normal reference range (ULN) for patients who are naïve to fitusiran at study start; ALT and/or AST \> 5 x ULN for patients who were in the fitusiran arm in the parent study
* Additional exclusions for participants not currently participating in a fitusiran trial at the time of enrollment in the lower dose cohort:
* Clinically significant liver disease
* History of arterial or venous thromboembolism
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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Genzyme, a Sanofi Company
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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Phoenix Childrens Hospital Site Number : 8400009
Phoenix, Arizona, United States
Children's Hospital Los Angeles Site Number : 8400019
Los Angeles, California, United States
Center for Inherited Blood Disorders (CIBD) Site Number : 8400016
Orange, California, United States
Nemours Children's Clinic Site Number : 8400008
Jacksonville, Florida, United States
St Joseph's Children's Hospital of Tampa Site Number : 8400002
Tampa, Florida, United States
Rush University Medical Center -1725 W Harrison St Site Number : 8400001
Chicago, Illinois, United States
~Massachusetts General Hospital Site Number : 8400011
Boston, Massachusetts, United States
University of Michigan Hospital - 1500 E Medical Center Dr Site Number : 8400012
Ann Arbor, Michigan, United States
Alliance for Childhood Diseases Site Number : 8400007
Las Vegas, Nevada, United States
Childrens Hospital Medical Center of Akron Site Number : 8400006
Akron, Ohio, United States
Investigational Site Number : 0360001
Camperdown, New South Wales, Australia
Investigational Site Number : 0360003
Prahran, Victoria, Australia
Investigational Site Number : 0360002
Murdoch, Western Australia, Australia
Investigational Site Number : 1240001
Montreal, Quebec, Canada
Investigational Site Number : 1560004
Beijing, , China
Investigational Site Number : 1560007
Beijing, , China
Investigational Site Number : 1560014
Changsha, , China
Investigational Site Number : 1560009
Chengdu, , China
Investigational Site Number : 1560002
Guangzhou, , China
Investigational Site Number : 1560011
Guiyang, , China
Investigational Site Number : 1560005
Hangzhou, , China
Investigational Site Number : 1560008
Jinan, , China
Investigational Site Number : 1560012
Kunming, , China
Investigational Site Number : 1560013
Lanzhou, , China
Investigational Site Number : 1560010
Qingdao, , China
Investigational Site Number : 1560003
Shanghai, , China
Investigational Site Number : 1560006
Suzhou, , China
Investigational Site Number : 2080001
Copenhagen, , Denmark
Investigational Site Number : 2500002
Lyon, , France
Investigational Site Number : 2500003
Paris, , France
Investigational Site Number : 2760001
Berlin, , Germany
Investigational Site Number : 2760003
Leipzig, , Germany
Investigational Site Number : 3480002
Budapest, , Hungary
Investigational Site Number : 3560001
Bangalore, , India
Investigational Site Number : 3560005
Lucknow, , India
Investigational Site Number : 3560007
Mumbai, , India
Investigational Site Number : 3560002
Pune, , India
Investigational Site Number : 3560010
Pune, , India
Investigational Site Number : 3560004
Ranipet, , India
Investigational Site Number : 3720001
Crumlin, Dublin, Ireland
Investigational Site Number : 3720002
Dublin, , Ireland
Investigational Site Number : 3760001
Ramat Gan, , Israel
Investigational Site Number : 3800002
Milan, Lombardy, Italy
Investigational Site Number : 3800004
Padua, Veneto, Italy
Investigational Site Number : 3920002
Nishinomiya, Hyōgo, Japan
Investigational Site Number : 3920005
Isehara, Kanagawa, Japan
Investigational Site Number : 3920003
Kitakyushu, , Japan
Investigational Site Number : 3920001
Nagoya, , Japan
Investigational Site Number : 3920004
Saitama, , Japan
Investigational Site Number : 3920008
Tokyo, , Japan
Investigational Site Number : 4580003
Ampang, , Malaysia
Investigational Site Number : 4580001
Johor Bahru, , Malaysia
Investigational Site Number : 4580002
Kota Kinabalu, , Malaysia
Investigational Site Number : 7100002
Port Elizabeth, , South Africa
Investigational Site Number : 4100001
Busan, Busan, South Korea
Investigational Site Number : 4100002
Daejeon, Daejeon, South Korea
Investigational Site Number : 4100003
Seoul, Seoul-teukbyeolsi, South Korea
Investigational Site Number : 4100004
Seoul, , South Korea
Investigational Site Number : 1580002
Changhua, , Taiwan
Investigational Site Number : 1580008
Taichung, , Taiwan
Investigational Site Number : 1580004
Taichung, , Taiwan
Investigational Site Number : 1580003
Taipei, , Taiwan
Investigational Site Number : 1580001
Taipei, , Taiwan
Investigational Site Number : 1580005
Taipei, , Taiwan
Investigational Site Number : 1580007
Taoyuan, , Taiwan
Investigational Site Number : 7920002
Adana, , Turkey (Türkiye)
Investigational Site Number : 7920004
Akdeniz, , Turkey (Türkiye)
Investigational Site Number : 7920012
Bornova, , Turkey (Türkiye)
Investigational Site Number : 7920006
Gaziantep, , Turkey (Türkiye)
Investigational Site Number : 7920005
Istanbul, , Turkey (Türkiye)
Investigational Site Number : 7920003
Izmir, , Turkey (Türkiye)
Investigational Site Number : 7920008
Kayseri, , Turkey (Türkiye)
Investigational Site Number : 7920007
Samsun, , Turkey (Türkiye)
Investigational Site Number : 8040001
Kyiv, , Ukraine
Investigational Site Number : 8040003
Kyiv, , Ukraine
Investigational Site Number : 8040002
Lviv, , Ukraine
Investigational Site Number : 8040004
Mykolaiv, , Ukraine
Investigational Site Number : 8260001
London, London, City of, United Kingdom
Investigational Site Number : 8260004
Glasgow, , United Kingdom
Countries
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References
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Young G, Kavakli K, Klamroth R, Matsushita T, Peyvandi F, Pipe SW, Rangarajan S, Shen MC, Srivastava A, Sun J, Tran H, You CW, Zulfikar B, Menapace LA, Zhang C, Shen Y, Puurunen M, Demissie M, Kenet G. Safety and efficacy of a fitusiran antithrombin-based dose regimen in people with hemophilia A or B: the ATLAS-OLE study. Blood. 2025 Jun 19;145(25):2966-2977. doi: 10.1182/blood.2024027008.
Related Links
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LTE15174 Plain Language Results Summaries
Other Identifiers
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U1111-1210-0018
Identifier Type: REGISTRY
Identifier Source: secondary_id
2023-508884-59
Identifier Type: REGISTRY
Identifier Source: secondary_id
2018-002880-25
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
LTE15174
Identifier Type: -
Identifier Source: org_study_id