A study of the effectiveness and safety of Octafibrin, a replacement for fibrinogen, in patients with inherited fibrinogen deficiency causing episodes of bleeding.

Phase 1

• Conditions: Congenital fibrinogen deficiency.; MedDRA version: 18.1 Level: LLT Classification code 10066357 Term: Congenital hypofibrinogenemia System Organ Class: 100000004850; MedDRA version: 18.1 Level: LLT Classification code 10066356 Term: Congenital hypofibrinogenaemia System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2011-002419-27-GB
• Lead Sponsor: Octapharma AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-04-30
• Study Completion: 2018-02-14
• Last Update Posted: 28 February 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 25

Inclusion Criteria

Subjects who meet all of the following criteria are eligible for the study:
1.Aged =12 years (only 18 and above in Russia).
2.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.
3.Expected to have an acute bleeding episode (spontaneous or after trauma) or planning to undergo elective surgery.
4.Informed consent signed by the subject or legal guardian.

Are the trial subjects under 18? yes
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 19
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

Subjects who meet any of the following criteria are not eligible for the study:
1.Life expectancy <6 months.
2.Bleeding disorder other than congenital fibrinogen deficiency, including dysfibrinogenaemia.
3.Prophylactic treatment with a fibrinogen concentrate.
4.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.
5.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).
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 neutralising anti-fibrinogen inhibitor currently or at 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
–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 drug licit or illicit substance.
12.Participation in another interventional clinical study currently or during the past 4 weeks.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified