NCT03974113

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

ATLAS-PEDS: An Open-label, Multinational Study of Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 32 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: Male
• Age: 1 Year – 11 Years
• Healthy volunteers: Not accepted

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

Detailed description

The estimated total time on study is up to 256 weeks for participants who roll over into the extension study and up to 280 weeks for participants who do not roll over into the extension study (due to the requirement for up to an additional 6 months of follow-up for monitoring of AT levels).

Conditions

• Hemophilia

Interventions

Type	Name	Description
DRUG	Fitusiran	Pharmaceutical form:Solution for injection-Route of administration:Subcutaneous

Timeline

• Start date: 2020-01-28
• Primary completion: 2026-08-15
• Completion: 2026-12-15
• First posted: 2019-06-04
• Last updated: 2025-08-08

Locations

16 sites across 6 countries: United States, Canada, India, Italy, Spain, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study