Health Condition 1: D682- Hereditary deficiency of other clotting factors
Conditions
Interventions
Intervention1: Octafibrin (a highly purified, lyophilised, human plasma fibrinogen concentrate, without added albumins): Dosage form: Freeze dried powder with active ingredient to be reconstituted wit
Octafibrin will be individually dosed to achieve a recommended target fibrinogen plasma level dependent on the bleeding type (minor or major)
Duration of treatment: The individual observation period
Sponsors
Octapharma AG
JSS Medical Research India Private Limited
Eligibility
Inclusion criteria
Inclusion criteria: 1. Aged 2. Documented diagnosis of congenital fibrinogen deficiency, expected to require on-demand treatment for bleeding or surgical prophylaxis: â?? Fibrinogen deficiency manifested as afibrinogenaemia or severe hypofibrino-genaemia. â?? Historical plasma fibrinogen activity of 3. Expected to have an acute bleeding episode (spontaneous or after trauma) or plan-ning to undergo elective surgery. 4. Informed consent signed by the subjectâ??s legal guardian.
Exclusion criteria
Exclusion criteria: 1. Life expectancy 2. Bleeding disorder other than congenital fibrinogen deficiency, including dysfi-brinogenaemia. 3. Prophylactic treatment with a fibrinogen concentrate. 4. Treatment with: â?? Any fibrinogen concentrate or other fibrinogen-containing blood product within 2 weeks prior to start of treatment for the PK phase, a bleeding episode, or sur-gery. â?? Any coagulation-active drug (i.e., non-steroidal anti-inflammatory drugs, war-farin, coumarin derivatives, platelet aggregation inhibitors) within 1 week prior to start of the PK phase or treatment for the bleeding episode or surgery, or as a planned or expected medication during the time period from Day 1 until 24 hours. 5. Presence or history of: â?? Hypersensitivity to study medication. â?? Deep vein thrombosis or pulmonary embolism within 1 year prior to start of treatment for the bleeding episode or surgery. â?? Arterial thrombosis within 1 year prior to start of treatment for the bleeding episode or surgery â?? Hypersensitivity to human plasma proteins. â?? Oesophageal varicose bleeding. â?? End-stage liver disease (i.e., Child-Pugh score B or C). 6. Known positive HIV infection with a viral load >200 particles/μL or >400,000 copies/mL. 7. Polytrauma 1 year prior to start of treatment for the bleeding episode or surgery. 8. Diagnosis or suspicion of a neutralizing anti-fibrinogen inhibitor currently or any time in the past. 9. 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, predni-sone (equivalent to >10 mg/day), or similar drugs, at study start. 10. Treatment with IMP 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 bleed-ing episode covers the time period from the first Octafibrin infusion for the treatment of a bleeding episode until 24 hours (i.e., 1 day) after the last infusion | — |
Secondary
| Measure | Time frame |
|---|---|
| Efficacy of Octafibrin in all bleeding episodes & surgical prophylaxisTimepoint: Clinical assessment of haemostatic efficacy for bleeding based on a 4-point haemostatic efficacy scale. To be assessed at the end of surgery by the surgeon and post-operatively by the haematologist ;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 efficacyTimepoint: 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 infusion of each documented bleeding episode.Timepoint: Before first infusion and 1 hour after end of first infusion of each documented bleeding episode.;Response after the first infusion of each bleeding episode as indicated by incremental IVR, calculated as the maximum increase in plasma fibrinogen between the pre-infusion and the 3-hour post-infusion.Timepoint: Between the pre-infusion and the 3-hour post-infusion. | — |
Countries
India, Lebanon, United States of America
Contacts
Public ContactDr Shariq Anwar
JSS Medical Research India Private Limited
Outcome results
None listed