Clinical trial NCT03793426: Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency
Official title: Post-marketing Observational Study on the Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Congenital Fibrinogen Deficiency
Lead sponsor: Octapharma
Sponsor class: INDUSTRY
First posted: 2019-01-04
Enrollment: 25
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Approximately 25 patients with a documented diagnosis of congenital afibrinogenemia or hypofibrinogenemia are planned to be documented in this study

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.

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

Primary outcomes:
measure: The incidence of thromboembolic adverse drug reactions (ADRs)
timeFrame: Day 0-28
Secondary outcomes:
measure: 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
timeFrame: Within 2-24 hours following treatment of BEs
measure: Dosage of Fibryga
timeFrame: Within 2-24 hours following treatment of BEs
measure: Duration of BEs
timeFrame: Within 2-24 hours following treatment of BEs
measure: Incidence of treatment-emergent adverse events (safety)
timeFrame: Day 0-28
Other outcomes:
Locations:
city: Ostrava
country: Czechia
facility: Fakultní nemocnice Ostrava
city: Duisburg
country: Germany
facility: Gerinnungszentrum rhein-ruhr
city: Frankfurt
country: Germany
facility: Medizinische Klinik 2 / Institut für Transfusionsmedizin Universitätsklinikum
city: Hamburg
country: Germany
facility: Gerinnungszentrum Hochtaunus
Study officials:
References: