Congenital Fibrinogen DeficiencyPhase 3NCT02267226
Efficacy and Safety Study of Octafibrin for On-demand Treatment of Acute Bleeding and to Prevent Bleeding During and After Surgery
A Phase 3 study testing Octafibrin in people with Congenital Fibrinogen Deficiency, run by Octapharma at 12 sites in 9 countries.
Trial summary
The purpose of the study is to assess the efficacy and safety of Octafibrin for on-demand treatment of acute bleeding in subjects with congenital fibrinogen deficiency. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Congenital Fibrinogen Deficiency
- What is being tested?
- Octafibrin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 12 sitesin 9 countries
- Study dates
- Sep 2014to 14 Feb 2018
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Aged ≥12 years (only 18 and above in Russia)
- 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.
- Expected to have an acute bleeding episode (spontaneous or after trauma) or planning to undergo elective surgery.
- Informed consent signed by the subject or legal guardian.
Who cannot join
- Life expectancy <6 months.
- Bleeding disorder other than congenital fibrinogen deficiency, including dysfibrinogenaemia.
- Prophylactic treatment with a fibrinogen concentrate.
- 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.
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- 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).
- Pregnant women within the first 20 weeks of gestation.
- Currently breast-feeding.
- Known positive HIV infection with a viral load >200 particles/μL or >400,000 copies/mL.
- Polytrauma 1 year prior to start of treatment for the bleeding episode or surgery.
- Diagnosis or suspicion of a neutralizing anti-fibrinogen inhibitor currently or any time in the past.
- 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 licit or illicit drug substance.
- Participation in another interventional clinical study currently or during the past 4 weeks.
Trial locations and contacts
12 sites in 9 countries.
12 sites in 9 countries
India3 sites
St. John's Medical College Hospital
Bangalore
Sahyadri Specialty Hospital
Pune
Dept of Hematology, Christian Medical College
Vellore
Bulgaria1 site
Dept of Clinical Hematology for Hemorrhagic Diatheses and Anaemia, SHAT "Joan Pavel"
Sofia
Russia1 site
Haematological Scientific Center of Ministry of Healthcare of the Russian Federation
Moscow
Saudi Arabia1 site
Centre of Excellence in Thrombosis & Hemostasis, King Saud University
Riyadh
Turkey (Türkiye)1 site
Dept of Haematology, Ege University Children's Hospital
Izmir
United Kingdom1 site
Centre for Haemostasis & Thrombosis, St Thomas' Hospital
London
About this study
Official title: 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
The purpose of the study is to assess the efficacy and safety of Octafibrin for on-demand treatment of acute bleeding in subjects with congenital fibrinogen deficiency.
- Conditions
- Congenital Fibrinogen Deficiency
- Being tested
- Octafibrin
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OctafibrinDrug
| Study group | Role | What they receive |
|---|---|---|
| Octafibrin | Experimental | Not described Receives: Drug: Octafibrin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Clinical Assessment of the Haemostatic Efficacy of Octafibrin in Treating the First Documented Bleeding Episode of Each Patient.
The first bleeding episode covers the time period from the first Octafibrin infusion until 24 hours (i.e., 1 day) after the last infusion.
The investigator's overall clinical assessment of haemostatic efficacy for bleeding was based on a 4 point haemostatic efficacy scale. The final efficacy assessment of each patient was adjudicated by the Independent Data Monitoring \& Endpoint Adjudication Committee (IDMEAC).
Measured: 24 hours after last infusion for each bleeding episode
Secondary outcomes (5)
Maximum Clot Firmness (MCF) After Fibrinogen Infusion in Each Documented Bleeding Episode (BE), Measured in Frozen Plasma in a Central Laboratory.
MCF (mm) was determined using ROTEM and was used as a surrogate marker for haemostatic efficacy. ROTEM is a method for the continuous measurement of clot formation and clot firmness. It utilises a mechanical detection system which is based on the ability of the blood or plasma clot to form a mechanical coupling over a distance of 1 mm.
Measured: Before first infusion and 1 hour after end of first and last infusion of each documented bleeding episode
Fibrinogen Plasma Level
Fibrinogen plasma level was assessed using the Clauss fibrinogen assay
Measured: Before (pre-infusion), 1 hour and 3 hours after the end of each subsequent infusion as well as at the time of the overall clinical assessment of haemostatic efficacy (i.e., 24 hours after the last infusion of each documented bleeding episode)
Response as Indicated by Incremental in Vivo Recovery (IVR)
Incremental IVR (response): calculated as the maximum increase in plasma fibrinogen (i.e., Clauss data) between pre-infusion and 1 and 3 hours post-infusion, divided by the exact dose of Octafibrin.
Measured: Pre-infusion and 1 and 3 hours post-infusion
Efficacy of Octafibrin for All Bleeding Episodes Collected in the Study
The investigator's overall clinical assessment of haemostatic efficacy for bleeding will be based on a 4-point haemostatic efficacy scale. The final efficacy assessment of each patient will be adjudicated by the Independent Data Monitoring \& Endpoint Adjudication Committee (IDMEAC)
Measured: 24 hours after last infusion for each bleeding episode
Efficacy of Octafibrin in Preventing Bleeding During and After Surgery
The efficacy of Octafibrin will be assessed at the end of surgery by the surgeon and post-operatively by the haematologist using two 4-point haemostatic efficacy scales. An overall efficacy assessment taking both the intra- and post-operative assessment into account will be adjudicated by the IDMEAC
Measured: First dose of Octafibrin administered prior to elective surgery to at least 3 post-operative days for minor and 7 post-operative days for major surgeries or last post-operative infusion, whichever comes last
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Oct 2014 (estimated)
- Study startSep 2014
- Primary completion14 Feb 2018
- Study completion14 Feb 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Congenital Fibrinogen DeficiencyStudies of the same condition, open and under way.
- More trials run by OctapharmaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Cristina Solomon, MD, Study director, Octapharma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02267226
- Sponsor's protocol ID
- FORMA-02
- Status last verified
- Dec 2020
- First submitted
- 7 Aug 2014
- Last updated
- 15 Jan 2021
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 15 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT02267226
- Acronym / sponsor ID
- FORMA-02
- Conditions
- Congenital Fibrinogen Deficiency
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- OctapharmaIndustry (pharma, biotech, devices)
- Study start
- Sep 2014
- Primary completion
- 14 Feb 2018 (actual)
- Study completion
- 14 Feb 2018 (actual)
- First posted
- 17 Oct 2014 (estimated)
- Results
- Posted 20 Feb 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the study is to assess the efficacy and safety of Octafibrin for on-demand treatment of acute bleeding in subjects with congenital fibrinogen deficiency.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Octafibrin | Experimental | Drug: Octafibrin |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Aged ≥12 years (only 18 and above in Russia)
- 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.
- Expected to have an acute bleeding episode (spontaneous or after trauma) or planning to undergo elective surgery.
Key exclusion criteria (22 listed)
- Life expectancy <6 months.
- Bleeding disorder other than congenital fibrinogen deficiency, including dysfibrinogenaemia.
- Prophylactic treatment with a fibrinogen concentrate.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Octafibrin | Drug | Octafibrin |
Primary outcome
Overall Clinical Assessment of the Haemostatic Efficacy of Octafibrin in Treating the First Documented Bleeding Episode of Each Patient.
Time frame: 24 hours after last infusion for each bleeding episode
The first bleeding episode covers the time period from the first Octafibrin infusion until 24 hours (i.e., 1 day) after the last infusion. The investigator's overall clinical assessment of haemostatic efficacy for bleeding was based on a 4 point haemostatic efficacy scale. The final efficacy assessment of each patient was adjudicated by the Independent Data Monitoring \& Endpoint Adjudication Committee (IDMEAC).
5 secondary outcomes
Maximum Clot Firmness (MCF) After Fibrinogen Infusion in Each Documented Bleeding Episode (BE), Measured in Frozen Plasma in a Central Laboratory.
Time frame: Before first infusion and 1 hour after end of first and last infusion of each documented bleeding episode
Fibrinogen Plasma Level
Time frame: Before (pre-infusion), 1 hour and 3 hours after the end of each subsequent infusion as well as at the time of the overall clinical assessment of haemostatic efficacy (i.e., 24 hours after the last infusion of each documented bleeding episode)
Response as Indicated by Incremental in Vivo Recovery (IVR)
Time frame: Pre-infusion and 1 and 3 hours post-infusion
Efficacy of Octafibrin for All Bleeding Episodes Collected in the Study
Time frame: 24 hours after last infusion for each bleeding episode
Efficacy of Octafibrin in Preventing Bleeding During and After Surgery
Time frame: First dose of Octafibrin administered prior to elective surgery to at least 3 post-operative days for minor and 7 post-operative days for major surgeries or last post-operative infusion, whichever comes last
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jan 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Phase 3 Study of Fibrinogen Concentrate (CSL511) in Subjects With Pseudomyxoma Peritonei Undergoing Cytoreductive Surgery
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Trauma Induced Coagulopathy, Trauma Coagulopathy
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Cristina Solomon, MD study director
Octapharma
12 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Octafibrin in people with Congenital Fibrinogen Deficiency, run by Octapharma at 12 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in India, Iran, United States, Bulgaria, Lebanon and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 20 Feb 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Clinical Assessment of the Haemostatic Efficacy of Octafibrin in Treating the First Documented Bleeding Episode of Each Patient. (time frame: 24 hours after last infusion for each bleeding episode). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jan 2021; AzTrials last refreshed its copy on 7 Nov 2026.