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 (NCT07285460)
An Open-label, Parallel, Phase 3, Two-arm 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 With or Without Inhibitory Antibodies to Factors VIII or IX
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: The Luskin Orthopaedic Institute for Children- Site Number : 8400013 — Los Angeles, California
Age range: 1 Year to 11 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (30 Sites)
Showing the first 25 of 30 sites.
- The Luskin Orthopaedic Institute for Children- Site Number : 8400013 — Los Angeles, California
- The Center for Inherited Blood Disorders- Site Number : 8400009 — Orange, California
- Cure 4 The Kids Foundation- Site Number : 8400001 — Las Vegas, Nevada
- Hackensack Meridian Health - Hackensack University Medical Center- Site Number : 8400008 — Hackensack, New Jersey
- University of Texas - Southwestern Medical Center- Site Number : 8400015 — Dallas, Texas
- Investigational Site Number : 0560002 — Brussels,
- Investigational Site Number : 0560001 — Brussels,
- Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto- Site Number : 0760003 — Ribeirão Preto, São Paulo
- Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo- Site Number : 0760004 — São Paulo,
- Investigational Site Number : 1240001 — Hamilton, Ontario
- Investigational Site Number : 1240003 — Montreal, Quebec
- Investigational Site Number : 1560001 — Beijing,
- Investigational Site Number : 3560004 — Bengaluru,
- Investigational Site Number : 3560002 — Mumbai,
- Investigational Site Number : 3560001 — Pune,
- Investigational Site Number : 3560003 — Vellore,
- Investigational Site Number : 3800002 — Florence, Firenze
- Investigational Site Number : 3800001 — Milan, Milano
- Investigational Site Number : 3800003 — Rozzano, Milano
- Klinika Transplantacji Szpiku, Onkologii i Hematologii Dziecięcej - Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu-Site Number: 6160002 — Wroclaw, Lower Silesian Voivodeship
- Spitalul Clinic De Urgenta Pentru Copii Sfanta Maria Iasi, Department: St. Mary Children's Emergency Clinical Hospital-Site Number : 6420001 — Iași,
- Spitalul Clinic De Urgenta pentru Copii "Louis Turcanu"-Site Number : 6420003 — Timișoara,
- Investigational Site Number : 6820001 — Riyadh,
- Investigational Site Number : 7240001 — Esplugues de Llobregat, Barcelona [Barcelona]
- Investigational Site Number : 7240002 — Madrid,
📞 Contact the Study Coordinator
Phone: (800) 633-1610
Email: Contact-US@sanofi.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Hemophilia
- Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX — Phoenix, Arizona
- A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia — Orange, California
- Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B — Los Angeles, California
- Patient Reported Outcomes Burdens and Experiences - Phase 3 — Hamilton, Ontario
- ATHNdataset Registry — Hickory, North Carolina
Explore by condition: All Hemophilia clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.