AfibrinogenemiaPhase 3NCT00916656
Fibrinogen Concentrate (Human) - Efficacy and Safety Study
A Phase 3 study testing Fibrinogen Concentrate, Human (FCH) and Cryoprecipitate in people with Afibrinogenemia and Hypofibrinogenemia, run by CSL Behring.
Trial summary
This is a multinational, multicenter, prospective, open-label historically controlled Phase IIIb non-inferiority clinical trial on the efficacy and safety of Fibrinogen Concentrate (Human). It is estimated that 150-300 patients in the U.S. suffer from afibrinogenemia. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Afibrinogenemia, Hypofibrinogenemia, Fibrinogen Deficiency
- What is being tested?
- Fibrinogen Concentrate, Human (FCH) and Cryoprecipitate
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Oct 2009to Mar 2014
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Documented congenital fibrinogen deficiency (afibrinogenemia and hypofibrinogenemia), expected to require treatment for bleeding
- Presenting with an episode of acute bleeding (either spontaneous or after trauma) not requiring surgery
- Provide informed consent
Who cannot join
- Life expectancy < 6 months
- Bleeding disorder other than congenital fibrinogen deficiency, but including dysfibrinogenemia
- Treatment with any investigational medicinal product (IMP) in the 30 days prior to enrollment
- Treatment with any fibrinogen concentrate or other fibrinogen containing blood product in the 2 weeks prior to enrollment
- Treatment with any coagulation active drug (i.e., non-steroidal-antirheumatics, warfarin, cumarin derivates, platelet aggregation inhibitors) in 1 week prior to enrollment or as a planned or expected medication during the time period from Day 1 until 24 hours after the last FCH infusion
- Presence or history of hypersensitivity to FCH
Show all 19Show fewer
- Presence or history of deep vein thrombosis or pulmonary embolism within 1 year prior to enrollment
- Presence or history of arterial thrombosis within 1 year prior to enrollment
- Presence or history of hypersensitivity to human plasma proteins
- Presence or history of esophageal varicose bleeding
- End stage liver disease (i.e., Child Pugh score B or C)
- Planned or expected surgery (i.e., for bleedings from aneurysm or splenic rupture)
- Pregnancy, or an intention to become pregnant during the study
- Currently breast-feeding, or with the intention of breast-feeding during the study
- Human immunodeficiency virus (HIV) positive
- Polytrauma, present or within 6 months prior to enrollment
- Suspicion of an anti-fibrinogen inhibitor as indicated by previous in-vivo recovery (IVR), if available (< 0.5 (mg/dL)/(mg/kg))
- Previous inclusion and treatment in the prospective part of the study
- Participation in any clinical study in the 30 days prior to enrollment
About this study
Official title: Efficacy and Safety of Fibrinogen Concentrate (Human) (FCH) for On-demand Treatment of Acute Bleeding in Subjects With Congenital Fibrinogen Deficiency
This is a multinational, multicenter, prospective, open-label historically controlled Phase IIIb non-inferiority clinical trial on the efficacy and safety of Fibrinogen Concentrate (Human).
It is estimated that 150-300 patients in the U.S. suffer from afibrinogenemia. Substitution with cryoprecipitate or alternative treatments have limited safety and efficacy.
The primary purpose of the study is to demonstrate the hemostatic efficacy of Fibrinogen Concentrate (Human) by adequately controlling acute bleeding (spontaneous or after trauma) in patients with congenital fibrinogen deficiency (afibrinogenemia and hypofibrinogenemia). Cryoprecipitate hemostatic efficacy data from a retrospective physician survey will be used as a historical control.
- Conditions
- Afibrinogenemia, Hypofibrinogenemia, Fibrinogen Deficiency
- Being tested
- Fibrinogen Concentrate, Human (FCH), Cryoprecipitate
- Keywords
- Congenital fibrinogen deficiency
- Medical subject headings
- Afibrinogenemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fibrinogen Concentrate, Human (FCH)Biological
Also called Haemocomplettan P, RIASTAP
Intravenous (IV) infusion to reach the peak target levels of 100 mg/dL with an accepted lower limit of 80 mg/dL on at least 3 subsequent days for minor bleeding episodes and 150 mg/dL with an accepted lower limit of 130 mg/dL on at least 7 subsequent days for major bleeding episodes.
If a subject's fibrinogen level is not known on Day 1, at the time treatment is initiated for the acute bleed (e.g., because they did not have a screening visit), the starting dose is to be 70 mg/kg b.w. Otherwise, the dose will be calculated individually.
CryoprecipitateBiological
Patients that received on-demand treatment with Cryoprecipitate for a classified bleeding event (minor or major) with a documented hemostatic efficacy assessment.
| Study group | Role | What they receive |
|---|---|---|
| Prospective Arm | Experimental | Not described Receives: Biological: Fibrinogen Concentrate, Human (FCH) |
| Historical Control | Other | Not described Receives: Biological: Cryoprecipitate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical assessment of hemostatic efficacy
Measured: 24 hours after last infusion or at Day 14 (whichever occurs first)
Secondary outcomes (4)
Maximum clot firmness (MCF)
Measured: Prior to and 60 minutes after the end of each infusion
Fibrinogen plasma level
Measured: 60 minutes, 3 hours, 6 hours, and 12 hours after the end of the first infusion; before and 60 minutes after each subsequent infusion
In vivo recovery of fibrinogen
Measured: 60 minutes, 3 hours, 6 hours and 12 hours after the end of the first infusion; before and 60 minutes after the end of each subsequent infusion and at the time of the overall clinical assessment of hemostatic efficacy
Virus safety markers
Measured: Day 1 to Day 45
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jun 2009 (estimated)
- Study startOct 2009
- Primary completionMar 2014
- Study completionMar 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AfibrinogenemiaStudies of the same condition, open and under way.
- More trials run by CSL BehringThe 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Program Director, Clinical R&D, Study director, CSL Behring
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00916656
- Sponsor's protocol ID
- BI3023_3001
- Other IDs
- 1475, 2007-004088-22
- Status last verified
- Feb 2023
- First submitted
- 8 Jun 2009
- Last updated
- 6 Feb 2023
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Fibrinogen Concentrate (Human) (FCH) for On-demand Treatment of Acute Bleeding in Subjects With Congenital Fibrinogen Deficiency
- ClinicalTrials.gov ID
- NCT00916656
- Acronym / sponsor ID
- BI3023_3001
- Conditions
- Afibrinogenemia, Hypofibrinogenemia, Fibrinogen Deficiency
- Keywords
- Congenital fibrinogen deficiency
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- Oct 2009
- Primary completion
- Mar 2014 (actual)
- Study completion
- Mar 2014 (actual)
- First posted
- 9 Jun 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multinational, multicenter, prospective, open-label historically controlled Phase IIIb non-inferiority clinical trial on the efficacy and safety of Fibrinogen Concentrate (Human).
It is estimated that 150-300 patients in the U.S. suffer from afibrinogenemia. Substitution with cryoprecipitate or alternative treatments have limited safety and efficacy.
The primary purpose of the study is to demonstrate the hemostatic efficacy of Fibrinogen Concentrate (Human) by adequately controlling acute bleeding (spontaneous or after trauma) in patients with congenital fibrinogen deficiency (afibrinogenemia and hypofibrinogenemia). Cryoprecipitate hemostatic efficacy data from a retrospective physician survey will be used as a historical control.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Prospective Arm | Experimental | Biological: Fibrinogen Concentrate, Human (FCH) |
| Historical Control | Other | Biological: Cryoprecipitate |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Documented congenital fibrinogen deficiency (afibrinogenemia and hypofibrinogenemia), expected to require treatment for bleeding
- Presenting with an episode of acute bleeding (either spontaneous or after trauma) not requiring surgery
- Provide informed consent
Key exclusion criteria (19 listed)
- Life expectancy < 6 months
- Bleeding disorder other than congenital fibrinogen deficiency, but including dysfibrinogenemia
- Treatment with any investigational medicinal product (IMP) in the 30 days prior to enrollment
- Treatment with any fibrinogen concentrate or other fibrinogen containing blood product in the 2 weeks prior to enrollment
- Treatment with any coagulation active drug (i.e., non-steroidal-antirheumatics, warfarin, cumarin derivates, platelet aggregation inhibitors) in 1 week prior to enrollment or as a planned or expected medication during the time period from Day 1 until 24 hours after the last FCH 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 |
|---|---|---|
| Fibrinogen Concentrate, Human (FCH)Also: Haemocomplettan P, RIASTAPIntravenous (IV) infusion to reach the peak target levels of 100 mg/dL with an accepted lower limit of 80 mg/dL on at least 3 subsequent days for minor bleeding episodes and 150 mg/dL with an accepted lower limit of 130 mg/dL on at least 7 subsequent days for major bleeding episodes. If a subject's fibrinogen level is not known on Day 1, at the time treatment is initiated for the acute bleed (e.g., because they did not have a screening visit), the starting dose is to be 70 mg/kg b.w. Otherwise, the dose will be calculated individually. | Biological | Prospective Arm |
| CryoprecipitatePatients that received on-demand treatment with Cryoprecipitate for a classified bleeding event (minor or major) with a documented hemostatic efficacy assessment. | Biological | Historical Control |
Primary outcome
Clinical assessment of hemostatic efficacy
Time frame: 24 hours after last infusion or at Day 14 (whichever occurs first)
4 secondary outcomes
Maximum clot firmness (MCF)
Time frame: Prior to and 60 minutes after the end of each infusion
Fibrinogen plasma level
Time frame: 60 minutes, 3 hours, 6 hours, and 12 hours after the end of the first infusion; before and 60 minutes after each subsequent infusion
In vivo recovery of fibrinogen
Time frame: 60 minutes, 3 hours, 6 hours and 12 hours after the end of the first infusion; before and 60 minutes after the end of each subsequent infusion and at the time of the overall clinical assessment of hemostatic efficacy
Virus safety markers
Time frame: Day 1 to Day 45
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Feb 2023.
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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.
Program Director, Clinical R&D study director
CSL Behring
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Fibrinogen Concentrate, Human (FCH) and Cryoprecipitate in people with Afibrinogenemia and Hypofibrinogenemia, run by CSL Behring.
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 3 inclusion and 19 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: withdrawn, as of the sponsor's last update on 6 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Clinical assessment of hemostatic efficacy (time frame: 24 hours after last infusion or at Day 14 (whichever occurs first)). 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 6 Feb 2023.