A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B

Study acronym: ATLAS-KIDS
StatusRecruiting
PhasePhase 3
SponsorSanofi
Started

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Record History

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  1. Minor Trial sites expanded: 30 → 41 locations
    • Eligibility criteria expanded, about 34 words
    • Secondary endpoints: details revised at 1 of 10 entries
  2. Minor Study sites revised (30)v15
  3. Minor Trial sites expanded: 27 → 30 locationsv14
  4. Minor Trial sites expanded: 24 → 27 locationsv13
  5. Minor Site details revised at 8 of 24 sitesv12
  6. Minor Trial sites expanded: 21 → 24 locationsv11
  7. Minor Study sites: 20 to 21v10
  8. Minor Study sites: 18 to 20v9
  9. Minor Trial sites expanded: 13 → 18 locationsv8
  10. Minor Study sites: 12 to 13v7
  11. Minor Study sites: 11 to 12v6
  12. Minor Study sites: 9 to 11v5
  13. Minor Study sites: 8 to 9v4
  14. Minor Study sites: 2 to 8
    • Eligibility criteria expanded, about 20 words
    v3
  15. Notable Trial 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
  16. Minor Study 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.
285 trials monitored
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241 trials monitored
See 12 trials at risk
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.
Trial Details
NCT Number NCT07285460
Lead Sponsor Sanofi
Conditions
(from sponsor)
Hemophilia
Related terms Hemophilia A
Enrollment 85 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov