NCT03793426

Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency

Post-marketing Observational Study on the Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency

• Status: Terminated
• Phase: —
• Study type: Observational
• Enrollment: 7 (actual)
• Sponsor: Octapharma · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

Open-label, Uncontrolled, Multicenter Observational Study on the Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency

Detailed description

There is a need to increase the body of data on treatment effectiveness and safety in the ultra-rare setting of congenital fibrinogen deficiency. Real-world evidence (RWE) derived from non-interventional studies can describe product utilization, demonstrate value, and facilitate benefit-risk assessments; RWE can only be fully assessed once a product is launched and used in a real-life setting. This post-marketing, observational study is designed to collect information concerning safety, efficacy, and outcomes of Fibryga administration in routine clinical use in patients of any age with congenital afibrinogenemia or hypofibrinogenemia. Documentation of the administration of Fibryga in clinical practice for the treatment of both minor and major bleeding events (BEs) will not only enhance the knowledge on the efficacy and safety profile of Fibryga, but will also gather information that cannot be obtained in the same way in controlled clinical studies. These observational data will support the safety and efficacy data generated with Fibryga in good clinical practice (GCP) clinical studies, providing benefit for both physicians and patients.

Conditions

• Congenital Fibrinogen Deficiency

Interventions

Type	Name	Description
BIOLOGICAL	Fibryga	Human plasma-derived fibrinogen concentrate

Timeline

• Start date: 2021-01-28
• Primary completion: 2025-05-14
• Completion: 2025-05-14
• First posted: 2019-01-04
• Last updated: 2025-06-24

Locations

4 sites across 2 countries: Czechia, Germany

Regulatory

• FDA-regulated drug study