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NCT07285460 · ClinicalTrials.gov registry record · Phase 3

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

A Phase 3 study of Hemophilia, sponsored by Sanofi.

Recruiting
Registry status
Phase 3
Development phase
85
Enrollment target
13
Study locations

NCT07285460: Recruiting Phase 3 study of Hemophilia, sponsored by Sanofi.

NCT07285460 is a Phase 3 study of Hemophilia that is actively recruiting participants, run by Sanofi. The registered enrollment target is 85 participants, below the 401-participant average among 9 other Hemophilia trials with a reported enrollment target (79% lower). The trial reports 13 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT07285460, a Phase 3 study of Hemophilia, is actively recruiting participants, sponsored by Sanofi.

RECRUITING
Registry status
Phase 3
Development phase
85 participants
Enrollment target
13
Study locations

Study Summary

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.

Primary Outcome

A bleeding episode is defined as any occurrence of hemorrhage that requires administration of CFCs or BPAs

Conditions Studied

Hemophilia

Interventions

  • DRUG Fitusiran
  • BIOLOGICAL Clotting factor concentrates (CFC) or bypassing agents (BPA)
  • BIOLOGICAL Antithrombin concentrate (ATIIIC)

Study Locations (13)

Other

  • Investigational Site Number : 0560002 - Brussels
  • Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo- Site Number : 0760004 - São Paulo
  • Investigational Site Number : 3560002 - Mumbai
  • Investigational Site Number : 3560001 - Pune
  • Investigational Site Number : 7240002 - Madrid
  • Investigational Site Number : 7240003 - Zaragoza
  • Investigational Site Number : 7920001 - Istanbul
  • Investigational Site Number : 7920002 - Izmir

California

  • The Luskin Orthopaedic Institute for Children- Site Number : 8400013 - Los Angeles
  • The Center for Inherited Blood Disorders- Site Number : 8400009 - Orange

Quebec

  • Investigational Site Number : 1240003 - Montreal

Lower Silesian Voivodeship

  • Investigational Site Number : 6160002 - Wroclaw

Barcelona [Barcelona]

  • Investigational Site Number : 7240001 - Esplugues de Llobregat

Trial Details

FieldValue
Enrollment Target 85 participants
Start Date 2025-12-18
Est. Completion 2031-12-30
Phase Phase 3
Sanofi

713 total trials

What NCT07285460 shows while recruiting

NCT07285460 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 85 participants, a relatively small participant target, below the 401-participant average among 9 other Hemophilia trials with a reported enrollment target (79% lower).

The record links to 1 condition, with Hemophilia appearing as the primary indexed condition, and to 3 interventions - of which Fitusiran is the first listed.

NCT07285460 lists 13 locations in 5 states (Other, California, Quebec).

Frequently Asked Questions

What is clinical trial NCT07285460 about?

NCT07285460 is a clinical study titled "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". 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 p...

What is the current status of trial NCT07285460?

This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 85 participants. The study started on 2025-12-18. Estimated completion is 2031-12-30.

What conditions does trial NCT07285460 study?

This clinical trial studies the following conditions: Hemophilia.

What interventions are being tested in trial NCT07285460?

The interventions under investigation include: Fitusiran (DRUG), Clotting factor concentrates (CFC) or bypassing agents (BPA) (BIOLOGICAL), Antithrombin concentrate (ATIIIC) (BIOLOGICAL).

Who is sponsoring clinical trial NCT07285460?

This trial is sponsored by Sanofi, which has 713 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT07285460 being conducted?

This trial has 13 study locations across California, Quebec, Lower Silesian Voivodeship, Barcelona [Barcelona]. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Learn More About Clinical Trials

Similar trials for Hemophilia

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Where NCT07285460's enrollment target sits among peer trials

85 4th of 9 higher than 6 of 9 other Hemophilia trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Hemophilia trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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More recruiting context for NCT07285460

Source: ClinicalTrials.gov NCT07285460, the US trial registry maintained by the National Library of Medicine. NCT07285460 (small enrollment · multi site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.