Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency

Octapharma7 enrolled

Overview

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

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Congenital Fibrinogen Deficiency

Interventions

FibrygaBIOLOGICAL

Human plasma-derived fibrinogen concentrate

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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

Locations (4)

Fakultní nemocnice Ostrava

Ostrava, Czechia

Gerinnungszentrum rhein-ruhr

Duisburg, Germany

Medizinische Klinik 2 / Institut für Transfusionsmedizin Universitätsklinikum

Frankfurt, Germany

Gerinnungszentrum Hochtaunus

Hamburg, Germany

Outcomes

Primary Outcomes

The incidence of thromboembolic adverse drug reactions (ADRs)

The incidence of thromboembolic ADRs in patients receiving Fibryga for on-demand treatment of bleeding, including major bleeding, will be documented

Time frame: Day 0-28

Secondary Outcomes

Hemostatic efficacy of Fibryga for all bleeding events (BEs) collected in the study will be assessed by the investigator using a 4-point hemostatic efficacy scale

The hemostatic efficacy of Fibryga for all BEs collected in the study will be assessed by the investigator using a 4-point hemostatic efficacy scale including the four items: 'excellent,' 'good,' moderate,' and 'none'. These data will be transformed into a dichotomous result, with 'treatment success-yes' defined as a rating of 'excellent' or 'good' and 'treatment success-no' defined as a rating of 'moderate' or 'none'.

Time frame: Within 2-24 hours following treatment of BEs

Dosage of Fibryga

Fibryga will be individually dosed as per the locally approved package insert. Actual dosage administered will be documented

Time frame: Within 2-24 hours following treatment of BEs

Duration of BEs

Details of BE duration will be documented

Time frame: Within 2-24 hours following treatment of BEs

Incidence of treatment-emergent adverse events (safety)

All ADRs in patients receiving Fibryga for on-demand treatment of BEs, including major BEs, will be documented

Time frame: Day 0-28

Data from ClinicalTrials.gov

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