Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency
Post-marketing Observational Study on the Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency
Study Overview
Status
Conditions
Intervention / Treatment
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
Enrollment (Actual)
Contacts and Locations
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Sampling Method
Study Population
Description
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
Study Plan
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Prospective
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
|---|---|
|
Fibryga
Fibryga (human plasma-derived fibrinogen concentrate)
|
Human plasma-derived fibrinogen concentrate
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
The incidence of thromboembolic adverse drug reactions (ADRs)
Time Frame: Day 0-28
|
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 Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
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
Time Frame: Within 2-24 hours following treatment of BEs
|
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
|
|
Dosage of Fibryga
Time Frame: Within 2-24 hours following treatment of BEs
|
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
|
|
Duration of BEs
Time Frame: Within 2-24 hours following treatment of BEs
|
Details of BE duration will be documented
|
Within 2-24 hours following treatment of BEs
|
|
Incidence of treatment-emergent adverse events (safety)
Time Frame: Day 0-28
|
All ADRs in patients receiving Fibryga for on-demand treatment of BEs, including major BEs, will be documented
|
Day 0-28
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- FORMA-07
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
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