drugset / Trial / NCT03974113

Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B

NCT03974113 ↗

Phase 2/3 Completed 32 enrolled Genzyme, a Sanofi Company
NaSingle-groupOpen-labelTreatment

Summary

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

Timeline

Start
2020-01-28
Primary completion
2026-08-04
Completion
2026-08-04

Drugs

EvaluationDrugModalityDoseRoute
Subject Fitusiran Small interfering RNA (siRNA) — Subcutaneous

Indications