NCT02615691

A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A

Phase 3, Prospective, Multi-center, Open Label Study to Investigate Safety, Immunogenicity and Hemostatic Efficacy of PEGylated Factor VIII (BAX 855) in Previously Untreated Patients (PUPs) < 6 Years With Severe Hemophilia A (FVIII < 1%)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 120 (actual)
• Sponsor: Baxalta now part of Shire · Industry
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

This study is for young children with severe hemophilia A who have previously not been treated with BAX855 or other FVIII concentrates. The main aim of the study is to check for side effects from treatment with BAX855. This includes the buildup of antibodies against FVIII which may stop BAX855 from working properly. Another aim is to learn how well BAX855 controls bleeding. In this study, the children can receive BAX855 either as preventative treatment (prophylaxis), or as needed to treat bleeding (on-demand). In case a participant develops antibodies, treatment will be provided as part of the study.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
BIOLOGICAL	PEGylated Recombinant Factor VIII	Polyethylene glycol (PEG)-ylated full-length recombinant FVIII (rFVIII).
BIOLOGICAL	ITI	Immune tolerance induction therapy

Timeline

• Start date: 2015-11-12
• Primary completion: 2024-10-29
• Completion: 2024-10-29
• First posted: 2015-11-26
• Last updated: 2025-07-28
• Results posted: 2025-07-28

Locations

91 sites across 23 countries: United States, Austria, Belgium, Bulgaria, Canada, Denmark, Finland, France, Germany, Hong Kong, Hungary, Italy, Malaysia, Netherlands, Norway, Singapore, South Korea, Spain, Taiwan, Thailand, Turkey (Türkiye), Ukraine, United Kingdom

Regulatory

• FDA-regulated drug study