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Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03793426
Recruitment Status : Not yet recruiting
First Posted : January 4, 2019
Last Update Posted : April 6, 2020
Sponsor:
Information provided by (Responsible Party):
Octapharma

Tracking Information
First Submitted Date December 19, 2018
First Posted Date January 4, 2019
Last Update Posted Date April 6, 2020
Estimated Study Start Date June 2020
Estimated Primary Completion Date January 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: January 3, 2019)
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
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: January 3, 2019)
  • 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'.
  • 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
  • Duration of BEs [ Time Frame: Within 2-24 hours following treatment of BEs ]
    Details of BE duration will be documented
  • 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
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
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 Adults and Adolescents (12-17 years of age) with 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 ≥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.
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
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
Condition Congenital Fibrinogen Deficiency
Intervention Biological: Fibryga
Human plasma-derived fibrinogen concentrate
Study Groups/Cohorts Fibryga
Fibryga (human plasma-derived fibrinogen concentrate)
Intervention: Biological: Fibryga
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Not yet recruiting
Estimated Enrollment
 (submitted: January 3, 2019)
25
Original Estimated Enrollment Same as current
Estimated Study Completion Date January 2024
Estimated Primary Completion Date January 2024   (Final data collection date for primary outcome measure)
Eligibility 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
Sex/Gender
Sexes Eligible for Study: All
Ages 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Bruce Schwartz, PhD +12016041112 bruce.schwartz@octapharma.com
Contact: Sylvia Werner, MS +12016041149 sylvia.werner@octapharma.com
Listed Location Countries Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number NCT03793426
Other Study ID Numbers FORMA-07
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Responsible Party Octapharma
Study Sponsor Octapharma
Collaborators Not Provided
Investigators
Study Director: Bruce Schwartz, PhD Octapharma
PRS Account Octapharma
Verification Date April 2020