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

STATUS

Recruiting
participants needed

12
sponsor

Genzyme, a Sanofi Company

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Updated on 19 February 2024

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hemophilia

antihemophilic factor

factor ix

bethesda assay

fitusiran

haemophilia a

Summary

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 Objective:

To characterize the safety and tolerability
To characterize the pharmacokinetics (PK)

Description

Study duration per participant is approximately 160 weeks, including a 12-week fitusiran efficacy period

Details

Condition	Hemophilia A, hemophilia, hemophilia
Age	1-11 years
Treatment	Fitusiran
Clinical Study Identifier	NCT03974113
Sponsor	Genzyme, a Sanofi Company
Last Modified on	19 February 2024

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Eligibility		Yes	No	Not Sure
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 anamnestic response
	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
	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/L
	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)
	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

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