Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency

Octapharma

Status

Terminated

Conditions

Congenital Fibrinogen Deficiency

Treatments

Biological: Fibryga

Study type

Observational

Funder types

Industry

Identifiers

NCT03793426

FORMA-07

Details and patient eligibility

About

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

Full 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.

Enrollment

7 patients

Sex

All

Volunteers

No Healthy Volunteers

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