Health Condition 1: null- Congenital fibrinogen deficiency - afibrinogenemia or Severe hypofibrinogenaemia- Historical Plasma fibrinogen activity of less than 50 mg/dl.
Conditions
Interventions
Intervention1: Octafibrin (Octafibrin is a highly purified, lyophilised, human plasma fibrinogen concentrate, without added albumins).: Name of the drug: Octafibrin
Dosage form: Freeze dried powder wi
Sponsors
Octapharma AG
JSS Medical Research India Private Limited
Eligibility
Inclusion criteria
Inclusion criteria: 1. Aged >=12 years. 2. Documented diagnosis of congenital fibrinogen deficiency, expected to require on-demand treatment for bleeding or surgical prophylaxis: ï? Fibrinogen deficiency manifested as afibrinogenaemia or severe hypofibrinogenaemia. ï? Historical plasma fibrinogen activity of 3. Having an acute bleeding episode (spontaneous or after trauma) or planning to undergo elective surgery. 4. Informed consent signed by the subject or legal guardian.
Exclusion criteria
Exclusion criteria: 1. Life expectancy 2. Bleeding disorder other than congenital fibrinogen deficiency, including dysfibrinogenaemia. 3. Prophylactic treatment with a fibrinogen concentrate. 4. Treatment with: ï? Any fibrinogen concentrate or other fibrinogen-containing blood product within 2 weeks prior to start of treatment for the bleeding episode or surgery. ï? 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. 5. Presence or history of: ï? Hypersensitivity to study medication. ï? Deep vein thrombosis or pulmonary embolism within 1 year prior to start of treatment for the bleeding episode or surgery. ï? Arterial thrombosis within 1 year prior to start of treatment for the bleeding episode or surgery ï? Hypersensitivity to human plasma proteins. ï? Oesophageal varicose bleeding. ï? End-stage liver disease (i.e., Child-Pugh score B or C). 6. Pregnant women within the first 20 weeks of gestation. 7. Currently breast-feeding. 8. Known positive HIV infection with a viral load >200 particles/μL or >400,000 copies/mL. 9. Polytrauma 1 year prior to start of treatment for the bleeding episode or surgery. 10. Diagnosis or suspicion of a neutralizing anti-fibrinogen inhibitor currently or any time in the past. 11. Acute or chronic medical condition which may, in the opinion of investigator, affect the conduct of the study, including ï? 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. ï? Subjects having evidence or a history (within the previous 12 months) of abuse of any licit or illicit drug substance. 12. Participation in another interventional clinical study currently or during the past 4 weeks.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoint is the overall clinical assessment of the haemostatic efficacy of Octafibrin in treating the first documented bleeding episode of each patient.Timepoint: The first bleeding episode covers the time period from the first Octafibrin infusion until 24 hours (i.e., 1 day) after the last infusion. | — |
Secondary
| Measure | Time frame |
|---|---|
| Efficacy of Octafibrin in all bleeding episodes and surgical prophylaxis (Detailed note has been given in brief summary).Timepoint: in all bleeding episodes, at the end of surgery and post-operatively;Fibrinogen plasma level before and 1 hour after the end of each infusion as well as at the time of the overall clinical assessment of haemostatic efficacy (Detailed note has been given in brief summary).Timepoint: before and 1 hour after the end of each infusion as well as at the time of the overall clinical assessment of haemostatic efficacy;MCF assessment before first infusion and 1 hour after end of first and last infusion of each documented bleeding episode. Timepoint: before first infusion and 1 hour after end of first and last infusion of each documented bleeding episode.;Response as indicated by incremental IVR, calculated as the maximum increase in plasma fibrinogen between pre-infusion and 1 and 3 hours post-infusion (Detailed note has been given in brief summary).Timepoint: between pre-infusion and 1 and 3 hours post-infusion | — |
Countries
Bulgaria, India, Iran (Islamic Republic of), United Kingdom, United States of America
Contacts
Public ContactDr Shariq Anwar
JSS Medical Research India Private Limited
Outcome results
None listed