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Trial intelligence / Profile preview
Phase 3 open-label fitusiran prophylaxis study in boys aged 1 to under 12 years with hemophilia A or B
2025-521858-42-00Phase 3Authorised
- Sponsor
- Sanofi
- Start
- Mar 2026
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 15
01
Overview
Phase 3, open-label trial of fitusiran in hemophilia A and hemophilia B (HB) (2 arms), sponsored by Sanofi. Primary endpoint: Annualized treated bleeding rate. Planned start Mar 2026; status: authorised.
02
Drugs
fitusiran
03
Conditions
hemophilia Ahemophilia B (HB)
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