This is a parallel, Phase 3, two-arm, open-label study to evaluate the efficacy and safety of treatment with fitusiran prophylaxis administered to male pediatric participants (aged 1 to \<12 years) who have severe hemophilia A or B, with or without inhibitory antibodies to FVIII or FIX. Number of participants: Approximately 85 participants will be enrolled into the study: * Approximately 60 fitusiran-naïve participants with severe hemophilia A or B, with or without inhibitors (fitusiran-naïve arm), and * Approximately 25 participants with severe hemophilia A or B with inhibitors rolling over from the EFC15467\* dose confirmation study (roll-over arm). * Fitusiran has been investigated in the pediatric population in study EFC15467, which enrolled male participants aged 1 to \<12 years with hemophilia A or B with inhibitors to examine the safety and tolerability of fitusiran in the pediatric population. Participants will be enrolled into 1 of 2 arms: * Fitusiran-naïve: these participants have not previously received fitusiran, and they will undergo screening and study eligibility assessments. Once enrolled, they will go through a 24-week standard of care (SOC) period before starting fitusiran prophylaxis. * Roll-over participants from the EFC15467 study: only participants who are still on active treatment in study EFC15467 and consenting to study EFC17905 will be eligible to roll over. They will not need to undergo screening or further eligibility assessments. They will directly enroll into the fitusiran treatment period and continue treatment on their current fitusiran dose. The duration of fitusiran treatment will be up to 160 weeks for the fitusiran-naïve arm and up to 60 weeks for the roll-over arm.
The duration of fitusiran treatment will be up to 160 weeks for the fitusiran-naïve arm and up to 60 weeks for the roll-over arm.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
85
Pharmaceutical form: solution for injection in PBS Route of administration: subcutaneous
Pharmaceutical form: solution for injection Route of administration: intravenous injection
Pharmaceutical form: solution for injection Route of administration: intravenous injection
Investigational Site Number : 7920001
Istanbul, Turkey (Türkiye)
RECRUITINGInvestigational Site Number : 7920002
Izmir, Turkey (Türkiye)
RECRUITINGAnnualized treated bleeding rate (ABR) in the fitusiran primary efficacy period and in the SOC period
A bleeding episode is defined as any occurrence of hemorrhage that requires administration of CFCs or BPAs
Time frame: Day 85 to Day 421 (fitusiran primary efficacy period); Day -168 to Day -1 (SOC period)
Annualized spontaneous bleeding rate (AsBR) in the fitusiran primary efficacy period and in the SOC period
A spontaneous bleeding episode is a bleeding event that occurs for no apparent or known reason, particularly into the joints, muscles, and soft tissues
Time frame: Day 85 to Day 421 (fitusiran primary efficacy period); Day -168 to Day -1 (SOC period)
Annualized joint bleeding rate (AjBR) in the fitusiran primary efficacy period and in the SOC period
A joint bleeding episode is characterized by an unusual sensation in the joint ("aura") in combination with 1) increasing swelling or warmth over the skin over the joint, 2) increasing pain, or 3) progressive loss of range of motion or difficulty in using the limb as compared with baseline
Time frame: Day 85 to Day 421 (fitusiran primary efficacy period); Day -168 to Day -1 (SOC period)
ABR in the fitusiran treatment period (160 weeks) for fitusiran-naïve participants
A bleeding episode is defined as any occurrence of hemorrhage that requires administration of CFCs or BPAs
Time frame: Day 1 to Day 1121
ABR in the fitusiran treatment period (60 weeks) for rolled-over participants
A bleeding episode is defined as any occurrence of hemorrhage that requires administration of CFCs or BPAs
Time frame: Day 1 to Day 421
Change in physical activity
Change in physical activity as measured via the PROMIS questionnaire
Time frame: From Day 1 to Day 421; From Week -24 to Day -1
Change in pain intensity
Change in pain intensity as measured via the PROMIS questionnaire
Time frame: From Day 1 to Day 421; From Week -24 to Day -1
Change in HRQoL
Change in HRQoL as measured by the EuroQoL 5-dimension questionnaire
Time frame: From Day 1 to Day 421; From Week -24 to Day -1
Incidence, severity, seriousness, and relatedness of adverse events (AEs)
All AEs are collected from the signing of the informed consent form (ICF) until last AT follow up visit
Time frame: Date of signed ICF (Day -228 to Day -169) until last AT follow-up visit (Day 1121 + approximately 24 weeks)
Change in total score and domain scores
Change in total score and domain scores is assessed by the Hemophilia Joint Health Score
Time frame: From Day 1 to Day 421; From Week -24 to Day -1
Target joints resolution
Target joints resolution assessed per ISTH criteria
Time frame: At Day 421
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CONTACT
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