Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency

  • STATUS
    Recruiting
  • End date
    Oct 24, 2027
  • participants needed
    25
  • sponsor
    Octapharma
Updated on 24 March 2022

Summary

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

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.

Details
Condition Congenital Fibrinogen Deficiency
Treatment Fibryga
Clinical Study IdentifierNCT03793426
SponsorOctapharma
Last Modified on24 March 2022

Eligibility

Yes No Not Sure

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
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