Fitusiran: Pharmaceutical form: solution for injection in PBS Route of administration: subcutaneous
Clotting factor concentrates (CFC) or bypassing agents (BPA): Pharmaceutical form: solution for injection Route of administration: intravenous injection
Antithrombin concentrate (ATIIIC): Pharmaceutical form: solution for injection Route of administration: intravenous injection
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.
| Facility | City | Region | Status |
|---|---|---|---|
| The Luskin Orthopaedic Institute for Children- Site Number : 8400013 | Los Angeles | California | Recruiting |
| The Center for Inherited Blood Disorders- Site Number : 8400009 | Orange | California | Recruiting |
| Cure 4 The Kids Foundation- Site Number : 8400001 | Las Vegas | Nevada | Recruiting |
| Hackensack Meridian Health - Hackensack University Medical Center- Site Number : 8400008 | Hackensack | New Jersey | Recruiting |
| University of Texas - Southwestern Medical Center- Site Number : 8400015 | Dallas | Texas | Recruiting |
| Investigational Site Number : 0560002 | Brussels | Belgium | Recruiting |
| Investigational Site Number : 0560001 | Brussels | Belgium | Recruiting |
| Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto- Site Number : 0760003 | Ribeirão Preto | São Paulo | Recruiting |
| Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo- Site Number : 0760004 | São Paulo | Brazil | Recruiting |
| Investigational Site Number : 1240001 | Hamilton | Ontario | Recruiting |
| Investigational Site Number : 1240003 | Montreal | Quebec | Recruiting |
| Investigational Site Number : 1560001 | Beijing | China | Recruiting |
| Investigational Site Number : 3560004 | Bengaluru | India | Recruiting |
| Investigational Site Number : 3560002 | Mumbai | India | Recruiting |
| Investigational Site Number : 3560001 | Pune | India | Recruiting |
| Investigational Site Number : 3560003 | Vellore | India | Recruiting |
| Investigational Site Number : 3800002 | Florence | Firenze | Recruiting |
| Investigational Site Number : 3800001 | Milan | Milano | Recruiting |
| Investigational Site Number : 3800003 | Rozzano | Milano | Recruiting |
| Klinika Transplantacji Szpiku, Onkologii i Hematologii Dziecięcej - Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu-Site Number: 6160002 | Wroclaw | Lower Silesian Voivodeship | Recruiting |
| Spitalul Clinic De Urgenta Pentru Copii Sfanta Maria Iasi, Department: St. Mary Children's Emergency Clinical Hospital-Site Number : 6420001 | Iași | Romania | Recruiting |
| Spitalul Clinic De Urgenta pentru Copii "Louis Turcanu"-Site Number : 6420003 | Timișoara | Romania | Recruiting |
| Investigational Site Number : 6820001 | Riyadh | Saudi Arabia | Recruiting |
| Investigational Site Number : 7240001 | Esplugues de Llobregat | Barcelona [Barcelona] | Recruiting |
| Investigational Site Number : 7240002 | Madrid | Spain | Recruiting |
| Investigational Site Number : 7240003 | Zaragoza | Spain | Recruiting |
| The division of Hematology, National Taiwan University Hospital-Site Number : 1580001 | Taipei City | Taipei | Recruiting |
| Department of pediatrics, Chung Shan University Hospital-Site Number : 1580002 | Taichung | Taiwan | Recruiting |
| Investigational Site Number : 7920001 | Istanbul | Turkey (Türkiye) | Recruiting |
| Investigational Site Number : 7920002 | Izmir | Turkey (Türkiye) | Recruiting |
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT07285460 on ClinicalTrials.gov ↗ ← All trials in India