Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B (NCT03974113) | Clinical Trial Compass
Active — Not RecruitingPhase 3
Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B
United States, Canada32 participantsStarted 2020-01-28
Plain-language 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 Objectives:
* To characterize the safety and tolerability
* To determine fitusiran plasma concentrations at selected time points
Who can participate
Age range1 Year – 11 Years
SexMALE
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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…
What they're measuring
1
Plasma antithrombin (AT) activity levels
Timeframe: Day 1 to the AT analysis time point at the optimal therapeutic dose (approximately 256 weeks)