An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B
NCT ID: NCT02554773
Last Updated: 2024-06-05
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
PHASE1/PHASE2
34 participants
INTERVENTIONAL
2015-09-18
2023-03-21
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
To evaluate the long-term safety and tolerability of fitusiran in male patients with moderate or severe hemophilia A or B
Secondary Objectives:
* To investigate the long-term efficacy of fitusiran
* To characterize the safety and efficacy of concomitantly administered Factor VIII (FVIII), Factor IX (FIX) or bypassing agents (BPA) and fitusiran for treatment of bleeding episodes
* To assess changes in health-related quality of life (QOL) over time
* To characterize antithrombin (AT) reduction and thrombin generation (TG) increase
* To characterize the pharmacokinetics (PK) of fitusiran
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia
NCT05662319
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
NCT03754790
A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab
NCT06145373
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
NCT07285460
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors
NCT03417245
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Fitusiran
Patients will be administered subcutaneous (SC) fitusiran once monthly or every 2 months according to the dose selection rules defined in protocol.
Fitusiran (SAR439774)
Pharmaceutical form: solution for injection Route of administration : subcutaneous (sc)
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Fitusiran (SAR439774)
Pharmaceutical form: solution for injection Route of administration : subcutaneous (sc)
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Male aged ≥18 years
* Moderate or severe, clinically stable hemophilia A or B as evidenced by a laboratory FVIII or FIX level ≤5% at screening. Patients with a FVIII or FIX level \>5% at screening will be eligible on provision of a historic laboratory report indicating a trough level ≤5%
* Willing and able to comply with the study requirements and provide written informed consent
Exclusion Criteria
* Patients known to be human immunodeficiency virus seropositive and have a CD4 count \<200 cells/μL
* History of venous thromboembolism
* Current serious mental illness that, in the judgment of the Investigator, may compromise patient safety, ability to participate in all study assessments, or study integrity
* Clinically relevant history or presence of cardiovascular, respiratory, gastrointestinal, renal, neurological, inflammatory, or other diseases that, in the judgment of the Investigator, precludes study participation
18 Years
MALE
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Genzyme, a Sanofi Company
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Clinical Sciences & Operations, MD
Role: STUDY_DIRECTOR
Sanofi
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Clinical Trial Site
Ann Arbor, Michigan, United States
Clinical Trial Site
Pittsburgh, Pennsylvania, United States
Clinical Trial Site
Plovdiv, , Bulgaria
Clinical Trial Site
Sofia, , Bulgaria
Clinical Trial Site
Kirov, , Russia
Clinical Trial Site
Moscow, , Russia
Clinical Trial Site
Zurich, , Switzerland
Clinical Trial Site
Basingstoke, , United Kingdom
Clinical Trial Site
Glasgow, , United Kingdom
Clinical Trial Site
London, , United Kingdom
Clinical Trial Site
London, , United Kingdom
Clinical Trial Site
Truro, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
Pipe SW, Lissitchkov T, Georgiev P, Mangles S, Hegemann I, Trinchero A, Chowdary P, Forbes A, Feng L, Menapace LA, Kichou S, Andersson S, Demissie M, Ragni MV. Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management, in people with hemophilia A or B. Blood Adv. 2025 Mar 11;9(5):1147-1158. doi: 10.1182/bloodadvances.2024013900.
Provided Documents
Download supplemental materials such as informed consent forms, study protocols, or participant manuals.
Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
ALN-AT3SC-002
Identifier Type: OTHER
Identifier Source: secondary_id
2015-001395-21
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
U1111-1251-5204
Identifier Type: REGISTRY
Identifier Source: secondary_id
LTE14762
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.