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A Study to Describe Patient REal-world FitusiRan ExpeRience
Sponsor: Sanofi
Summary
This is a real-world, mixed-methods observational study conducted among adolescent (ages 12-17 years) and adult participants diagnosed with hemophilia A or B, with or without Factor VIII (FVIII) or Factor IX (FIX) inhibitors, who are treated with fitusiran. The study includes a prospective longitudinal cohort (Cohort 1) and a cross-sectional cohort (Cohort 2). Data collection includes patient-reported outcomes via online surveys, qualitative interviews conducted via teleconference, and electronic medical record (EMR) review to describe real-world patient experiences, treatment satisfaction, adherence, and clinical management of fitusiran.
Official title: Patient Experience With Fitusiran Prophylaxis in Adolescents and Adults With Hemophilia A or B With or Without Factor VIII or Factor IX Inhibitors in a Real-world Setting
Key Details
Gender
All
Age Range
12 Years - Any
Study Type
OBSERVATIONAL
Enrollment
100
Start Date
2026-10-07
Completion Date
2029-03-02
Last Updated
2026-10-26
Healthy Volunteers
No
Conditions
Locations (1)
Concentrics Center for Research
Indianapolis, Indiana, United States