Efficacy and Safety Study of Octafibrin for On-demand Treatment of Acute Bleeding and to Prevent Bleeding During and After Surgery

• Status: Completed
• Trial end date: 2018-02-14
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Octapharma

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.