Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency

NCT ID: NCT03793426

Last Updated: 2025-06-24

Basic Information

• Recruitment Status: TERMINATED
• Total Enrollment: 7 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2021-01-28
• Study Completion Date: 2025-05-14

Brief 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

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Fibryga

Fibryga (human plasma-derived fibrinogen concentrate)

Fibryga

Intervention Type: BIOLOGICAL

Human plasma-derived fibrinogen concentrate

Interventions

Fibryga

Human plasma-derived fibrinogen concentrate

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

• Patients of any age with a documented diagnosis of congenital afibrinogenemia or hypofibrinogenemia expected to require on-demand in-hospital treatment for BEs with Fibryga

Exclusion Criteria

• Bleeding disorder other than congenital fibrinogen deficiency
• Patients with acquired fibrinogen deficiency or dysfibrinogenemia
• Suspicion of an anti-fibrinogen inhibitor as indicated by previous in vivo recovery, if available, of <0.5 (mg/dL)/(mg/kg); there is currently no standard test for inhibitors
• Participation in an interventional clinical study at the time of or within 4 weeks prior to enrolment

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Octapharma

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Fakultní nemocnice Ostrava

Ostrava, , Czechia

Gerinnungszentrum rhein-ruhr

Duisburg, , Germany

Medizinische Klinik 2 / Institut für Transfusionsmedizin Universitätsklinikum

Frankfurt, , Germany

Gerinnungszentrum Hochtaunus

Hamburg, , Germany

Countries

Czechia; Germany

Other Identifiers

FORMA-07

Identifier Type: -

Identifier Source: org_study_id