Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency

Congenital Fibrinogen Deficiency Clinical Trial

Official title:

Post-marketing Observational Study on the Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03793426
• Other study ID #: FORMA-07
• Status: Recruiting
• Start date: January 28, 2021
• Est. completion date: October 2027

Study information

• Verified date: February 2024
• Source: Octapharma
• Contact: Gita Pezeshki, PhD
• Phone: +12016041125
• Email: gita.pezeshki[@]octapharma.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 25
• Est. completion date: October 2027
• Est. primary completion date: October 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Afibrinogenemia
• Congenital Fibrinogen Deficiency

Intervention

Biological:
• Fibryga
Human plasma-derived fibrinogen concentrate

Locations

Country	Name	City	State
Germany	Gerinnungszentrum rhein-ruhr	Duisburg
Germany	Medizinische Klinik 2 / Institut für Transfusionsmedizin Universitätsklinikum	Frankfurt
Germany	Gerinnungszentrum Hochtaunus	Hamburg

Sponsors (1)

Lead Sponsor	Collaborator
Octapharma

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Day 0-28
Secondary	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'.	Within 2-24 hours following treatment of BEs
Secondary	Dosage of Fibryga	Fibryga will be individually dosed as per the locally approved package insert. Actual dosage administered will be documented	Within 2-24 hours following treatment of BEs
Secondary	Duration of BEs	Details of BE duration will be documented	Within 2-24 hours following treatment of BEs
Secondary	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	Day 0-28