Congenital Fibrinogen DeficiencyNCT03793426
Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency
An observational study of Congenital Fibrinogen Deficiency, run by Octapharma at 4 sites in 2 countries.
Trial summary
Open-label, Uncontrolled, Multicenter Observational Study on the Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Congenital Fibrinogen Deficiency
- What is being tested?
- Fibryga
- How many people
- 7 participants
- Where
- 4 sitesin 2 countries
- Study dates
- 28 Jan 2021to 14 May 2025
- Phase
- Observational
- 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
Study population: Approximately 25 patients with a documented diagnosis of congenital afibrinogenemia or hypofibrinogenemia are planned to be documented in this study
Who may be able to join
- Patients of any age with a documented diagnosis of congenital afibrinogenemia or hypofibrinogenemia expected to require on-demand in-hospital treatment for BEs with Fibryga
Who cannot join
- Bleeding disorder other than congenital fibrinogen deficiency
- Patients with acquired fibrinogen deficiency or dysfibrinogenemia
- Suspicion of an anti-fibrinogen inhibitor as indicated by previous in vivo recovery, if available, of <0.5 (mg/dL)/(mg/kg); there is currently no standard test for inhibitors
- Participation in an interventional clinical study at the time of or within 4 weeks prior to enrolment
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
Germany3 sites
Gerinnungszentrum rhein-ruhr
Duisburg
Medizinische Klinik 2 / Institut für Transfusionsmedizin Universitätsklinikum
Frankfurt
Gerinnungszentrum Hochtaunus
Hamburg
About this study
Official title: Post-marketing Observational Study on the Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency
Open-label, Uncontrolled, Multicenter Observational Study on the Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency
Read the detailed description
There is a need to increase the body of data on treatment effectiveness and safety in the ultra-rare setting of congenital fibrinogen deficiency. Real-world evidence (RWE) derived from non-interventional studies can describe product utilization, demonstrate value, and facilitate benefit-risk assessments; RWE can only be fully assessed once a product is launched and used in a real-life setting.
This post-marketing, observational study is designed to collect information concerning safety, efficacy, and outcomes of Fibryga administration in routine clinical use in patients of any age with congenital afibrinogenemia or hypofibrinogenemia. Documentation of the administration of Fibryga in clinical practice for the treatment of both minor and major bleeding events (BEs) will not only enhance the knowledge on the efficacy and safety profile of Fibryga, but will also gather information that cannot be obtained in the same way in controlled clinical studies. These observational data will support the safety and efficacy data generated with Fibryga in good clinical practice (GCP) clinical studies, providing benefit for both physicians and patients.
- Conditions
- Congenital Fibrinogen Deficiency
- Being tested
- Fibryga
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FibrygaBiological
Human plasma-derived fibrinogen concentrate
| Study group | Role | What they receive |
|---|---|---|
| Fibryga | Fibryga (human plasma-derived fibrinogen concentrate) Receives: Biological: Fibryga |
What is being measured
The primary outcome is the main question the study is designed to answer.
The incidence of thromboembolic adverse drug reactions (ADRs)
The incidence of thromboembolic ADRs in patients receiving Fibryga for on-demand treatment of bleeding, including major bleeding, will be documented
Measured: Day 0-28
Secondary outcomes (4)
Hemostatic efficacy of Fibryga for all bleeding events (BEs) collected in the study will be assessed by the investigator using a 4-point hemostatic efficacy scale
The hemostatic efficacy of Fibryga for all BEs collected in the study will be assessed by the investigator using a 4-point hemostatic efficacy scale including the four items: 'excellent,' 'good,' moderate,' and 'none'. These data will be transformed into a dichotomous result, with 'treatment success-yes' defined as a rating of 'excellent' or 'good' and 'treatment success-no' defined as a rating of 'moderate' or 'none'.
Measured: Within 2-24 hours following treatment of BEs
Dosage of Fibryga
Fibryga will be individually dosed as per the locally approved package insert. Actual dosage administered will be documented
Measured: Within 2-24 hours following treatment of BEs
Duration of BEs
Details of BE duration will be documented
Measured: Within 2-24 hours following treatment of BEs
Incidence of treatment-emergent adverse events (safety)
All ADRs in patients receiving Fibryga for on-demand treatment of BEs, including major BEs, will be documented
Measured: Day 0-28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jan 2019
- Study start28 Jan 2021
- Primary completion14 May 2025
- Study completion14 May 2025
Why it stopped: FORMA-07 was initiated as a requirement imposed by the U.S. FDA as part of Octapharma's regulatory obligations. After approving Fibryga for treatment of acquired fibrinogen deficiency in 2024, FDA has agreed that the study may now be discontinued.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03793426
- Sponsor's protocol ID
- FORMA-07
- Status last verified
- Jun 2025
- First submitted
- 19 Dec 2018
- Last updated
- 24 Jun 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Post-marketing Observational Study on the Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency
- ClinicalTrials.gov ID
- NCT03793426
- Acronym / sponsor ID
- FORMA-07
- Conditions
- Congenital Fibrinogen Deficiency
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: FORMA-07 was initiated as a requirement imposed by the U.S. FDA as part of Octapharma's regulatory obligations. After approving Fibryga for treatment of acquired fibrinogen deficiency in 2024, FDA has agreed that the study may now be discontinued.
- Enrolment
- 7 participants (actual)
- Lead sponsor
- OctapharmaIndustry (pharma, biotech, devices)
- Study start
- 28 Jan 2021 (actual)
- Primary completion
- 14 May 2025 (actual)
- Study completion
- 14 May 2025 (actual)
- First posted
- 4 Jan 2019 (actual)
- 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.
Open-label, Uncontrolled, Multicenter Observational Study on the Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency
Detailed description
There is a need to increase the body of data on treatment effectiveness and safety in the ultra-rare setting of congenital fibrinogen deficiency. Real-world evidence (RWE) derived from non-interventional studies can describe product utilization, demonstrate value, and facilitate benefit-risk assessments; RWE can only be fully assessed once a product is launched and used in a real-life setting.
This post-marketing, observational study is designed to collect information concerning safety, efficacy, and outcomes of Fibryga administration in routine clinical use in patients of any age with congenital afibrinogenemia or hypofibrinogenemia. Documentation of the administration of Fibryga in clinical practice for the treatment of both minor and major bleeding events (BEs) will not only enhance the knowledge on the efficacy and safety profile of Fibryga, but will also gather information that cannot be obtained in the same way in controlled clinical studies. These observational data will support the safety and efficacy data generated with Fibryga in good clinical practice (GCP) clinical studies, providing benefit for both physicians and patients.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fibryga | – | Biological: FibrygaFibryga (human plasma-derived fibrinogen concentrate) |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Approximately 25 patients with a documented diagnosis of congenital afibrinogenemia or hypofibrinogenemia are planned to be documented in this study
Key inclusion criteria (1 listed)
- Patients of any age with a documented diagnosis of congenital afibrinogenemia or hypofibrinogenemia expected to require on-demand in-hospital treatment for BEs with Fibryga
Key exclusion criteria (4 listed)
- Bleeding disorder other than congenital fibrinogen deficiency
- Patients with acquired fibrinogen deficiency or dysfibrinogenemia
- Suspicion of an anti-fibrinogen inhibitor as indicated by previous in vivo recovery, if available, of <0.5 (mg/dL)/(mg/kg); there is currently no standard test for inhibitors
- Participation in an interventional clinical study at the time of or within 4 weeks prior to enrolment
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 |
|---|---|---|
| FibrygaHuman plasma-derived fibrinogen concentrate | Biological | Fibryga |
Primary outcome
The incidence of thromboembolic adverse drug reactions (ADRs)
Time frame: Day 0-28
The incidence of thromboembolic ADRs in patients receiving Fibryga for on-demand treatment of bleeding, including major bleeding, will be documented
4 secondary outcomes
Hemostatic efficacy of Fibryga for all bleeding events (BEs) collected in the study will be assessed by the investigator using a 4-point hemostatic efficacy scale
Time frame: Within 2-24 hours following treatment of BEs
Dosage of Fibryga
Time frame: Within 2-24 hours following treatment of BEs
Duration of BEs
Time frame: Within 2-24 hours following treatment of BEs
Incidence of treatment-emergent adverse events (safety)
Time frame: Day 0-28
Results and safety
The study was stopped early (reason given: FORMA-07 was initiated as a requirement imposed by the U.S. FDA as part of Octapharma's regulatory obligations. After approving Fibryga for treatment of acquired fibrinogen deficiency in 2024, FDA has agreed that the study may now be discontinued.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2025.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Congenital Fibrinogen Deficiency, run by Octapharma at 4 sites in 2 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 1 inclusion and 4 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: stopped early, as of the sponsor's last update on 24 Jun 2025. 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 Germany, Czechia. 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.
Are results available for this study?
The study was stopped early (reason given: FORMA-07 was initiated as a requirement imposed by the U.S. FDA as part of Octapharma's regulatory obligations. After approving Fibryga for treatment of acquired fibrinogen deficiency in 2024, FDA has agreed that the study may now be discontinued.). Results are not available in the source record.
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?
The incidence of thromboembolic adverse drug reactions (ADRs) (time frame: Day 0-28). 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 24 Jun 2025.