Secondary endpoints: details revised at 1 of 10 entries
MinorStudy sites revised (30)v15
MinorTrial sites expanded: 27 → 30 locationsv14
MinorTrial sites expanded: 24 → 27 locationsv13
MinorSite details revised at 8 of 24 sitesv12
MinorTrial sites expanded: 21 → 24 locationsv11
MinorStudy sites: 20 to 21v10
MinorStudy sites: 18 to 20v9
MinorTrial sites expanded: 13 → 18 locationsv8
MinorStudy sites: 12 to 13v7
MinorStudy sites: 11 to 12v6
MinorStudy sites: 9 to 11v5
MinorStudy sites: 8 to 9v4
MinorStudy sites: 2 to 8
Eligibility criteria expanded, about 20 words
v3
NotableTrial status: Not Yet Recruiting to Recruiting
Study sites: 0 to 2
Start date changed from estimate of Dec 19, 2025 to Dec 18, 2025
v2
MinorStudy First Submitted
Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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.