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

Prospective, open-label, uncontrolled, Phase III study to assess the efficacy and safety of Octafibrin for on-demand treatment of acute bleeding and to prevent bleeding during and after surgery in subjects with congenital fibrinogen deficiency. - Octafibrin in Congenital Fibrinogen Deficiency - FORMA-02

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2011-002419-27-GB

Enrollment

Registered

2014-04-30

Start date

2014-06-11

Completion date

Unknown

Last updated

2019-02-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

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

Interventions

Product Name: Octafibrin Pharmaceutical Form: Powder for solution for injection INN or Proposed INN: Fibrinogen (as clottable protein) CAS Number: 9001-

Eligibility

Sex/Gender

All

Inclusion criteria

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=65 years) yes F.1.3.1 Number of subjects for this age range 1

Exclusion criteria

Exclusion criteria: Subjects who meet any of the following criteria are not eligible for the study: 1. Life expectancy 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.

Design outcomes

Primary

Measure	Time frame
Main Objective: The study aims to demonstrate the efficacy of Octafibrin for on-demand treatment of acute bleeding episodes (spontaneous or after trauma).;Primary end point(s): The primary endpoint is the overall clinical assessment of haemostatic efficacy in acute bleeding at 24 hours (i.e., 1 day) after last infusion or the end of the treatment observation period (whichever comes last) of the IMP for the first bleeding episode of each patient.; Secondary Objective: • To show an association between the overall clinical assessment of the effectiveness of Octafibrin and the laboratory endpoint ‘clot strength’ or ‘clot firmness’ (referred to as ‘maximum clot firmness’ [MCF] in this protocol) that was used as a "surrogate" measure of effectiveness in the previous study FORMA-01. • To achieve a peak target level of fibrinogen in plasma of 100 mg/dL in minor bleeds and 150 mg/dL for major bleeds 1 hour post-infusion. • To determine the response to Octafibrin based on incremental in vivo recovery (IVR). • To demonstrate the efficacy of Octafibrin in preventing bleeding during and after surgery. • To assess the safety of Octafibrin in subjects with congenital fibrinogen deficiency, including immunogenicity, thromboembolic complications, and early signs of allergic or hypersensitivity reactions. ;Timepoint(s) of evaluation of this end point: 24 hours (i.e., 1 day) after last infusion or the end of the treatment observation period (whichever comes last) of the IMP for the first bleeding episode of each patient.	—

Measure

Time frame

Main Objective: The study aims to demonstrate the efficacy of Octafibrin for on-demand treatment of acute bleeding episodes (spontaneous or after trauma).;Primary end point(s): The primary endpoint is the overall clinical assessment of haemostatic efficacy in acute bleeding at 24 hours (i.e., 1 day) after last infusion or the end of the treatment observation period (whichever comes last) of the IMP for the first bleeding episode of each patient.; Secondary Objective: • To show an association between the overall clinical assessment of the effectiveness of Octafibrin and the laboratory endpoint ‘clot strength’ or ‘clot firmness’ (referred to as ‘maximum clot firmness’ [MCF] in this protocol) that was used as a "surrogate" measure of effectiveness in the previous study FORMA-01. • To achieve a peak target level of fibrinogen in plasma of 100 mg/dL in minor bleeds and 150 mg/dL for major bleeds 1 hour post-infusion. • To determine the response to Octafibrin based on incremental in vivo recovery (IVR). • To demonstrate the efficacy of Octafibrin in preventing bleeding during and after surgery. • To assess the safety of Octafibrin in subjects with congenital fibrinogen deficiency, including immunogenicity, thromboembolic complications, and early signs of allergic or hypersensitivity reactions. ;Timepoint(s) of evaluation of this end point: 24 hours (i.e., 1 day) after last infusion or the end of the treatment observation period (whichever comes last) of the IMP for the first bleeding episode of each patient.

—

Secondary

Measure	Time frame
Secondary end point(s): Clot strength (MCF) Peak target level of fibrinogen in plasma 1 hour post-infusion Octafibrin use - The dose of the IMP used per day and in total. In-vivo recovery (IVR)- Response and classical IVR will be calculated for each infusion of each subject. Surgical prophylaxis Efficacy of Octafibrin in surgical prophylaxis will be assessed intra-operatively (at the end of surgery = after last suture) by the surgeon and post-operatively by the haematologist using two 4-point efficacy scales. The overall surgical efficacy will be adjudicated by the IDMEAC. Safety analysis ; Timepoint(s) of evaluation of this end point: - MCF before 1st infusion and 1h after the end of the 1st and last infusion of each bleeding episode - Fibrinogen levels 1 hour after infusion of the investigational medicinal product (IMP - In vivo recovery (IVR)for each infusion - Octafibrin use per day and in total - Efficacy of Octafibrin in surgical prophylaxis will be assessed intra-operatively by the surgeon and post-operatively by the haematologist	—

Measure

Time frame

Secondary end point(s): Clot strength (MCF) Peak target level of fibrinogen in plasma 1 hour post-infusion Octafibrin use - The dose of the IMP used per day and in total. In-vivo recovery (IVR)- Response and classical IVR will be calculated for each infusion of each subject. Surgical prophylaxis Efficacy of Octafibrin in surgical prophylaxis will be assessed intra-operatively (at the end of surgery = after last suture) by the surgeon and post-operatively by the haematologist using two 4-point efficacy scales. The overall surgical efficacy will be adjudicated by the IDMEAC. Safety analysis ; Timepoint(s) of evaluation of this end point: - MCF before 1st infusion and 1h after the end of the 1st and last infusion of each bleeding episode - Fibrinogen levels 1 hour after infusion of the investigational medicinal product (IMP - In vivo recovery (IVR)for each infusion - Octafibrin use per day and in total - Efficacy of Octafibrin in surgical prophylaxis will be assessed intra-operatively by the surgeon and post-operatively by the haematologist

—

Countries

Bulgaria, Egypt, India, Lebanon, Russian Federation, Saudi Arabia, Turkey, United Kingdom

Contacts

Public ContactNazira Maruf

QED Clinical Services Ltd

NMaruf@qed-clinical.com+44 1908 251 480

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026