An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B

Completed · Phase 1/Phase 2

Conditions studied: Hemophilia A, Hemophilia B

In brief

Primary Objective: 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

Key facts

Study ID

NCT02554773

Run by

Genzyme, a Sanofi Company

People needed

34

Starts

2015-09-18

Expected to finish

2023-03-21

Last updated by the study team

2024-06-05

Who can join

Age: 18 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Completed and tolerated study drug dosing in study TDR14767 (ALN-AT3SC-001)
• 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

You may not qualify if…

• Clinically significant liver disease
• 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

Where it is running

• 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

Full record on ClinicalTrials.gov

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