Efficacy and Safety Study of Octafibrin for On-demand Treatment of Acute Bleeding and to Prevent Bleeding During and After Surgery
Condition: Congenital Fibrinogen Deficiency
MeSH terms: Afibrinogenemia; Hemorrhage
Intervention: Octafibrin
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 25.0
Sex: All
Minimum age: 12 Years
Healthy volunteers: No

Summary:

      The purpose of the study is to assess the efficacy and safety of Octafibrin for on-demand
      treatment of acute bleeding in subjects with congenital fibrinogen deficiency.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Aged ≥12 years (only 18 and above in Russia)

          -  Documented diagnosis of congenital fibrinogen deficiency, expected to require
             on-demand treatment for bleeding or surgical prophylaxis:

          -  Fibrinogen deficiency manifested as afibrinogenaemia or severe hypofibrinogenaemia.

          -  Historical plasma fibrinogen activity of <50 mg/dL or levels below the limit of
             detection of the local assay method.

          -  Expected to have an acute bleeding episode (spontaneous or after trauma) or planning
             to undergo elective surgery.

          -  Informed consent signed by the subject or legal guardian.

        Exclusion Criteria:

          -  Life expectancy <6 months.

          -  Bleeding disorder other than congenital fibrinogen deficiency, including
             dysfibrinogenaemia.

          -  Prophylactic treatment with a fibrinogen concentrate.

        Treatment with:

          -  Any fibrinogen concentrate or other fibrinogen-containing blood product within 2 weeks
             prior to start of treatment for the bleeding episode or surgery.

          -  Any coagulation-active drug (i.e., non-steroidal anti-inflammatory drugs, warfarin,
             coumarin derivatives, platelet aggregation inhibitors) within 1 week prior to start of
             treatment for the bleeding episode or surgery, or as a planned or expected medication
             during the time period from Day 1 until 24 hours (i.e., 1 day) after the last
             Octafibrin infusion.

        Presence or history of:

          -  Hypersensitivity to study medication.

          -  Deep vein thrombosis or pulmonary embolism within 1 year prior to start of treatment
             for the bleeding episode or surgery.

          -  Arterial thrombosis within 1 year prior to start of treatment for the bleeding episode
             or surgery

          -  Hypersensitivity to human plasma proteins.

          -  Oesophageal varicose bleeding.

          -  End-stage liver disease (i.e., Child-Pugh score B or C).

        Pregnant women within the first 20 weeks of gestation.

        Currently breast-feeding.

        Known positive HIV infection with a viral load >200 particles/μL or >400,000 copies/mL.

        Polytrauma 1 year prior to start of treatment for the bleeding episode or surgery.

        Diagnosis or suspicion of a neutralizing anti-fibrinogen inhibitor currently or any time in
        the past.

        Acute or chronic medical condition which may, in the opinion of investigator, affect the
        conduct of the study, including

          -  Subjects receiving immune-modulating drugs (other than anti-retroviral chemotherapy)
             such as alpha-interferon, prednisone (equivalent to >10 mg/day), or similar drugs at
             study start.

          -  Subjects having evidence or a history (within the previous 12 months) of abuse of any
             licit or illicit drug substance.

        Participation in another interventional clinical study currently or during the past 4
        weeks.