Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency

Brief Title

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

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.
    


Study Type

Observational


Primary Outcome

The incidence of thromboembolic adverse drug reactions (ADRs)

Secondary Outcome

 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

Condition

Congenital Fibrinogen Deficiency

Intervention

Fibryga

Study Arms / Comparison Groups

 Fibryga
Description:  Fibryga (human plasma-derived fibrinogen concentrate)

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

25

Start Date

January 28, 2021

Completion Date

October 2027

Primary Completion Date

October 2027

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
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Bruce Schwartz, PhD, +12016041112, [email protected]

Location Countries

Germany

Location Countries

Germany

Administrative Informations


NCT ID

NCT03793426

Organization ID

FORMA-07


Responsible Party

Sponsor

Study Sponsor

Octapharma


Study Sponsor

Bruce Schwartz, PhD, Study Director, Octapharma


Verification Date

September 2021