Hypofibrinogenemia, CongenitalPhase 2/3NCT02094430
Human Fibrinogen Concentrate (FGTW) in Pediatric Patients With Congenital Fibrinogen Deficiency
A Phase 2/3 study testing biological: human fibrinogen concentrate in people with Hypofibrinogenemia, Congenital and Afibrinogenemia, Congenital, run by Laboratoire français de Fractionnement et de Biotechnologies at 4 sites in 4 countries.
Trial summary
The aim of the study is to evaluate clinical pharmacology, efficacy and safety of FGTW in pediatric patients with congenital fibrinogen deficiency. Read more about this study
- Who is it for?
- Ages Up to 12 years
- Conditions the study is for
- Hypofibrinogenemia, Congenital, Afibrinogenemia, Congenital
- What is being tested?
- biological: human fibrinogen concentrate
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 4 sitesin 4 countries
- Study dates
- Jan 2014to Dec 2015
- Phase
- Phase 2/3
- 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 Up to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Signed and dated informed consent form by parents or a legal representative
- Age less or equal to 12 years old
- Patients with inherited afibrinogenemia or severe inherited hypofibrinogenemia
- Negative results on HCG-based pregnancy test for females of childbearing potential (presence of menstruation)
Who cannot join
- Dysfibrinogenemia
- Acquired fibrinogen deficiency
- Suspected present or past anticoagulation inhibitor
- Personal history of venous or arterial thrombosis or thromboembolic event
- Co-morbidity with other/unrelated coagulopathies
- Administration of any fibrinogen concentrate or fibrinogen containing blood product during the last 15 days
- Permanent treatment with antithrombotic or anti-platelet agents such as heparins, anti-IIa or anti-Xa agents, aspirin, clopidogrel and NSAIDs.
Trial locations and contacts
4 sites in 4 countries.
4 sites in 4 countries
Turkey (Türkiye)1 site
Faculty Ihsan Dogramaci Children's Hospital
Ankara
About this study
Official title: Clinical Pharmacology, Efficacy and Safety Study of FGTW in Paediatric Patients With Severe Congenital Fibrinogen Deficiency
The aim of the study is to evaluate clinical pharmacology, efficacy and safety of FGTW in pediatric patients with congenital fibrinogen deficiency.
- Being tested
- biological: human fibrinogen concentrate
- Medical subject headings
- Afibrinogenemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
biological: human fibrinogen concentrateDrug
| Study group | Role | What they receive |
|---|---|---|
| FGTW | Experimental | Not described Receives: Drug: biological: human fibrinogen concentrate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Investigator's overall assessment of efficacy of FGTW on hemostasis using a 4-point scale at the end of each bleeding or surgical episode.
Measured: 6 hours or up to 5 days
Terminal half life for Fibrinogen antigen and activity
Measured: 5 days
Secondary outcomes (1)
Adverse Events
Measured: Participants will be followed for the duration of their participation in the study, an expected average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2014 (estimated)
- Study startJan 2014
- Primary completionDec 2015
- Study completionDec 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hypofibrinogenemia, CongenitalStudies of the same condition, open and under way.
- 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 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02094430
- Sponsor's protocol ID
- FGTW-1004
- Status last verified
- Feb 2016
- First submitted
- 26 Feb 2014
- Last updated
- 12 Feb 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Feb 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Pharmacology, Efficacy and Safety Study of FGTW in Paediatric Patients With Severe Congenital Fibrinogen Deficiency
- ClinicalTrials.gov ID
- NCT02094430
- Acronym / sponsor ID
- FGTW-1004
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Laboratoire français de Fractionnement et de BiotechnologiesIndustry (pharma, biotech, devices)
- Study start
- Jan 2014
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 21 Mar 2014 (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.
The aim of the study is to evaluate clinical pharmacology, efficacy and safety of FGTW in pediatric patients 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 |
|---|---|---|
| FGTW | Experimental | Drug: biological: human fibrinogen concentrate |
Who may qualify
- Ages
- Up to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Signed and dated informed consent form by parents or a legal representative
- Age less or equal to 12 years old
- Patients with inherited afibrinogenemia or severe inherited hypofibrinogenemia
- Negative results on HCG-based pregnancy test for females of childbearing potential (presence of menstruation)
Key exclusion criteria (7 listed)
- Dysfibrinogenemia
- Acquired fibrinogen deficiency
- Suspected present or past anticoagulation inhibitor
- Personal history of venous or arterial thrombosis or thromboembolic event
- Co-morbidity with other/unrelated coagulopathies
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 |
|---|---|---|
| biological: human fibrinogen concentrate | Drug | FGTW |
Primary outcome
Investigator's overall assessment of efficacy of FGTW on hemostasis using a 4-point scale at the end of each bleeding or surgical episode.
Time frame: 6 hours or up to 5 days
Terminal half life for Fibrinogen antigen and activity
Time frame: 5 days
1 secondary outcome
Adverse Events
Time frame: Participants will be followed for the duration of their participation in the study, an expected average of 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Feb 2016.
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.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing biological: human fibrinogen concentrate in people with Hypofibrinogenemia, Congenital and Afibrinogenemia, Congenital, run by Laboratoire français de Fractionnement et de Biotechnologies at 4 sites in 4 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 4 inclusion and 7 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 12 Feb 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in France, Lebanon, Morocco, Turkey (Türkiye). 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Investigator's overall assessment of efficacy of FGTW on hemostasis using a 4-point scale at the end of each bleeding or surgical episode. (time frame: 6 hours or up to 5 days). The study lists 2 primary outcomes in all. 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 12 Feb 2016; AzTrials last refreshed its copy on 8 Nov 2026.