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Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B

Active Not RecruitingPhase 2, Phase 3

Sponsor: Genzyme, a Sanofi Company

Conditions: Hemophilia

Interventions: Fitusiran

Countries: United States, Canada, India, Italy, Spain, Turkey (Türkiye)

Primary Objective: \- To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to \<12 years of age) with severe hemophilia A or B Secondary Objectives: * To characterize the safety and tolerability * To determine fitusiran plasma concentrations at selected time points

Eligibility overview

Sex: MALE

Age: 1 Year to 11 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria

Inclusion Criteria:

* Male, aged 1 to \<12 years at the time of enrollment.
* Severe hemophilia A or B (Factor VIII (FVIII) \<1% or Factor IX (FIX) ≤2%)
* Participants must have inhibitory antibodies to FVIII or FIX and must meet one of the following Nijmegen-modified Bethesda assay results criteria:

  * Inhibitor titer of ≥0.6 BU/mL at screening, OR
  * Inhibitor titer of \<0.6 BU/mL at screening with medical record evidence of 2 consecutive titers ≥0.6 BU/mL, OR
  * Inhibitor titer of \<0.6 BU/mL at screening with medical record evidence of 1 inhibitor titer ≥0.6 BU/mL and a history of anamnestic response or severe allergic reaction (anaphylaxis or nephrotic syndrome)
* Adequate peripheral venous access, as determined by the Investigator, to allow the blood draws required by the study protocol
* Weight requirements at the time of enrollment: 8 to \<45 kg
* Willing and able to comply with the study requirements and to provide signed written informed consent obtained from parent(s)/legal guardian (hereinafter the "parent") and written or oral assent obtained from participant, per local and national requirements

Exclusion Criteria:

Participants are excluded from the study if any of the following criteria apply:

* 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
* Acute or chronic Hepatitis B virus infection
* Acute Hepatitis A or hepatitis E infection
* HIV positive with a CD4 count of \<400 cells/μL
* History of arterial or venous thromboembolism, unrelated to an indwelling venous access
* Inadequate renal function
* History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc)
* Subjects with central or peripheral indwelling catheters, with history of venous access complications leading to hospitalization and/or systemic anticoagulation therapy.
* History of intolerance to subcutaneous (SC) injection(s)
* Use of emicizumab (Hemlibra®) within 6 months prior to screening
* Any other conditions or comorbidities that would make the patient unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgment

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Locations (16)

Los Angeles, California, United States
Hackensack, New Jersey, United States
Cleveland, Ohio, United States
Hershey, Pennsylvania, United States
Hamilton, Ontario, Canada
Ottawa, Ontario, Canada
Bangalore, India
Mumbai, India
Pune-411011, India
Vellore, India
Florence, Italy
Milan, Italy
Madrid, Madrid, Comunidad de, Spain
Adana, Turkey (Türkiye)
Istanbul, Turkey (Türkiye)
Izmir, Turkey (Türkiye)