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Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency

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ClinicalTrials.gov Identifier: NCT03793426
Recruitment Status : Not yet recruiting
First Posted : January 4, 2019
Last Update Posted : August 6, 2019
Sponsor:
Information provided by (Responsible Party):
Octapharma

Brief Summary:
Open-label, Uncontrolled, Multicenter Observational Study on the Safety and Efficacy of Fibryga in Adults and Adolescents (12-17 years of age) with Congenital Fibrinogen Deficiency

Condition or disease Intervention/treatment
Congenital Fibrinogen Deficiency Biological: Fibryga

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 ≥12 years of 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.

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Study Type : Observational
Estimated Enrollment : 25 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Post-marketing Observational Study on the Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency
Estimated Study Start Date : October 2019
Estimated Primary Completion Date : January 2024
Estimated Study Completion Date : January 2024


Group/Cohort Intervention/treatment
Fibryga
Fibryga (human plasma-derived fibrinogen concentrate)
Biological: Fibryga
Human plasma-derived fibrinogen concentrate




Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. 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'.

  2. 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

  3. Duration of BEs [ Time Frame: Within 2-24 hours following treatment of BEs ]
    Details of BE duration will be documented

  4. 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



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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
A minimum of 25 patients with a documented diagnosis of congenital afibrinogenemia or hypofibrinogenemia are planned to be documented in this study
Criteria

Inclusion Criteria:

  • Patients ≥12 years of 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03793426


Contacts
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Contact: Bruce Schwartz, PhD +12016041112 bruce.schwartz@octapharma.com
Contact: Sylvia Werner, MS +12016041149 sylvia.werner@octapharma.com

Sponsors and Collaborators
Octapharma
Investigators
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Study Director: Bruce Schwartz, PhD Octapharma

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Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT03793426     History of Changes
Other Study ID Numbers: FORMA-07
First Posted: January 4, 2019    Key Record Dates
Last Update Posted: August 6, 2019
Last Verified: August 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Afibrinogenemia
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn