Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B
Completed · Phase 2, Phase 3 · NCT03974113
Conditions: Hemophilia
At a glance
- Research payments reported, 2025
- $52,352
- U.S. investigators named
- 3
- Sites paid
- 3
- Planned or actual enrollment
- 32
About the trial
- Sponsor
- Genzyme, a Sanofi Company
- Tested
- Fitusiran (drug)
- Dates
- Started 2020-01-28; completion 2026-08-04
- Results posted
- Not yet
Primary Objective: \- To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to \<12 years of age) with severe hemophilia A or B Secondary Objectives: * To characterize the safety and tolerability * To determine fitusiran plasma concentrations at selected time points
From ClinicalTrials.gov, last updated 2026-09-28. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Genzyme Corporation reported $52,352 in research payments for this study in 2025 (31 payments).
Products named: Qfitlia
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Guy A. Young, M.D. Pediatric Hematology & Oncology · Los Angeles, CA
- Stacey Rifkin-zenenberg, D.O. Hospice & Palliative Medicine · Hackensack, NJ
- Amma Owusu-ansah, MD Pediatric Hematology & Oncology · Cleveland, OH
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.