A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors

Completed · Phase 3

Conditions studied: Hemophilia A, Hemophilia B

In brief

The purpose of this study was to determine the frequency of bleeding episodes in participants receiving fitusiran as prophylactic treatment of hemophilia compared to participants who were assigned to continue with their regular medication. In addition, the study assessed safety, quality of life, pharmacodynamics (PD), and pharmacokinetics (PK).

Key facts

Study ID

NCT03417102

Run by

Genzyme, a Sanofi Company

People needed

60

Starts

2018-02-14

Expected to finish

2021-06-23

Last updated by the study team

2022-03-28

Who can join

Age: 12 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Males, greater than or equal to (>=) 12 years of age.
• Severe hemophilia A or B with inhibitors.
• (Severity confirmed by a central laboratory where coagulation factor VIII (FVIII) level was less than (<)1% or factor IX (FIX) level was less than or equal to [<=]2% at Screening; Inhibitors defined as inhibitor titer of >=0.6 Bethesda units per milliliter [BU/mL] or as evidenced by medical records).
• A minimum of 6 bleeding episodes requiring BPA 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.

You may not qualify if…

• Known co-existing bleeding disorders other than hemophilia A or B.
• Antithrombin (AT) activity <60% at Screening.
• Co-existing thrombophilic disorder.
• Clinically significant liver disease.
• Active hepatitis C virus infection.
• HIV positive with a cluster of differentiation-4 count of <200 cells/microliter.
• History of arterial or venous thromboembolism.
• Inadequate renal function.
• History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine.
• History of intolerance to SC injection(s).
• Any other conditions or comorbidities that would make the participant unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgement.

Where it is running

• Investigational Site Number 0139 — Los Angeles, California, United States
• Investigational Site Number 0135 — Orange, California, United States
• Investigational Site Number 0137 — San Diego, California, United States
• Investigational Site Number 0128 — Gainesville, Florida, United States
• Investigational Site Number 0115 — Jacksonville, Florida, United States
• Investigational Site Number 0105 — Miami, Florida, United States
• Investigational Site Number 0103 — Tampa, Florida, United States
• Investigational Site Number 0119 — New Orleans, Louisiana, United States
• Investigational Site Number 0136 — Baltimore, Maryland, United States
• Investigational Site Number 0111 — Las Vegas, Nevada, United States
• Investigational Site Number 0104 — Philadelphia, Pennsylvania, United States
• Investigational Site Number 6101 — Camperdown, Australia
• Investigational Site Number 6104 — Clayton, Australia
• Investigational Site Number 1102 — Montreal, Canada
• Investigational Site Number 8604 — Beijing, China
• Investigational Site Number 8602 — Guangzhou, China
• Investigational Site Number 8605 — Hangzhou, China
• Investigational Site Number 8603 — Shanghai, China
• Investigational Site Number 8601 — Tianjin, China
• Investigational Site Number 3303 — Lyon, France
• Investigational Site Number 3301 — Rouen, France
• Investigational Site Number 4905 — Frankfurt am Main, Germany
• Investigational Site Number 4906 — Leipzig, Germany
• Investigational Site Number 9102 — Bangalore, India
• Investigational Site Number 0117 — Phoenix, Arizona, United States

Full record on ClinicalTrials.gov

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