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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 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.
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.
Conditions Studied
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
| Field | Value |
|---|---|
| Enrollment Target | 85 participants |
| Start Date | 2025-12-18 |
| Est. Completion | 2031-12-30 |
| Phase | Phase 3 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07285460
The ClinicalTrials.gov registry entry for NCT07285460 describes a study currently listed as recruiting, categorized as Phase 3. The registered enrollment target is 85 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 401-participant average among 9 other Hemophilia trials with a reported enrollment target (79% lower). The listed sponsor is Sanofi, which has 713 total studies on file at ClinicalTrials.gov.
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 reports 13 study locations spanning 5 distinct geographic areas - top geographies include 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.
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Related
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