To study the effect of Octafibrin (i.e. human plasma fibrinogen concentrate, which is a product derived from blood) in paediatric subjects with congenital fibrinogen deficiency (a rare bleeding disorder).
- Conditions
- Health Condition 1: D682- Hereditary deficiency of other clotting factors
- Registration Number
- CTRI/2015/07/005953
- Lead Sponsor
- Octapharma AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Closed to Recruitment of Participants
- Sex
- Not specified
- Target Recruitment
- 4
1.Aged <12 years (at the start of treatment). This will include two subgroups of subjects aged between 0 and <6 years and between 6 and <12 years of either gender.
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 <50 mg/dL or levels below the limit of detection of the local assay method.
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.
1. Life expectancy <6 months.
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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method