Condition: Hemophilia · Sponsor: Sanofi
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…
This description comes directly from the study's public registry record.
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| The Luskin Orthopaedic Institute for Children- Site Number : 8400013 | Los Angeles, California, United States | Recruiting |
| The Center for Inherited Blood Disorders- Site Number : 8400009 | Orange, California, United States | Recruiting |
| Cure 4 The Kids Foundation- Site Number : 8400001 | Las Vegas, Nevada, United States | Recruiting |
| Hackensack Meridian Health - Hackensack University Medical Center- Site Number : 8400008 | Hackensack, New Jersey, United States | Recruiting |
| University of Texas - Southwestern Medical Center- Site Number : 8400015 | Dallas, Texas, United States | 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, Brazil | 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, Canada | Recruiting |
| Investigational Site Number : 1240003 | Montreal, Quebec, Canada | Recruiting |
| Investigational Site Number : 1560001 | Beijing, China | Recruiting |
+ 18 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT07285460