Congenital AfibrinogenemiaPhase 3NCT04636268
FIB Grifols Congenital Deficiency for On-demand Treatment and Surgical Prophylaxis
A Phase 3 study testing FIB Grifols in people with Congenital Afibrinogenemia and Hypofibrinogenemia, run by Instituto Grifols, S.A..
Trial summary
This is a phase 3, multi-center, prospective, open-label, single-arm, clinical trial to be carried out in subjects with congenital fibrinogen deficiency manifested as afibrinogenemia or severe hypofibrinogenemia. This clinical trial is planned to be performed at study sites in multiple countries. Read more about this study
- Who is it for?
- Ages 6 to 70 years
- Conditions the study is for
- Congenital Afibrinogenemia, Hypofibrinogenemia
- What is being tested?
- FIB Grifols
- 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
- Dec 2023to Mar 2025
- 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 6 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female subject between 6 to 70 years of age.
- Signed and dated written ICF, or the subject's parent or legal guardian signs and dates the ICF where applicable, and the Subject Authorization Form (SAF) where applicable. Pediatric subjects, as defined by local regulations, will be asked to sign an age appropriate assent form.
- Diagnosed with congenital fibrinogen deficiency manifested as afibrinogenemia or severe hypofibrinogenemia (fibrinogen <50 mg/dL) and expected to require treatment for acute bleeding (either spontaneous or after trauma [defined as any accidental event leading to acute bleeding]), or prophylaxis of bleeding before a surgical intervention or invasive procedure.
- Fibrinogen level < 50 mg/dL determined by Clauss method at baseline (sample drawn within 24 hours prior to infusion on Infusion 1 Day 1 Visit).
- Female subjects of child-bearing potential must have a negative test for pregnancy blood or urine human chorionic gonadotropin (HCG)-based assay at baseline (sample drawn within 24 hours prior to infusion on Infusion 1 Day 1 Visit).
- Willing to comply with all aspects of the clinical trial protocol, including blood sampling, for the entire duration of the study.
Who cannot join
- Has acquired (secondary) fibrinogen deficiency.
- Diagnosed with dysfibrinogenemia.
- Has known antibodies against fibrinogen.
- Has history of anaphylaxis or severe systemic response to any drug or blood-derived product.
- Has history of intolerance to any component of the IP.
- Documented history of immunoglobulin A (IgA) deficiency and antibodies against IgA.
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- Is a female who is pregnant, breastfeeding or, if of child-bearing potential, unwilling to practice a highly effective method of contraception (eg, oral, injectable, or implantable hormonal methods of contraception, placement of an IUD or intrauterine system, condom or occlusive cap with spermicidal foam/gel/film/cream/suppository, male sterilization, or true abstinancea) throughout the study.
- Has any medical condition which is likely to interfere with the evaluation of the IP and/or the satisfactory conduct of the clinical trial according to the investigator's judgment.
- Has congenital or acquired bleeding disorders other than congenital fibrinogen deficiency.
- Has life expectancy of less than 6 months.
- Received FRT within 21 days prior to the Screening Visit.
- Receiving, or having received within 3 months prior to the Screening Visit of this clinical trial, any investigational drug or device.
- Is unlikely to adhere the protocol requirements, or is likely to be uncooperative, or unable to provide a storage sample prior to IP infusion.
About this study
Official title: A Prospective, Multicenter, Open-label, Single-arm Study to Evaluate the Efficacy and Safety of Human Plasma-derived Fibrinogen Concentrate (FIB Grifols) in Subjects With Congenital Afibrinogenaemia and Severe Hypofibrinogenemia Requiring Either On-demand Treatment for Acute Bleeding or Surgical Prophylaxis
This is a phase 3, multi-center, prospective, open-label, single-arm, clinical trial to be carried out in subjects with congenital fibrinogen deficiency manifested as afibrinogenemia or severe hypofibrinogenemia.
This clinical trial is planned to be performed at study sites in multiple countries. It is planned to include a maximum of 32 adult and pediatric subjects with congenital fibrinogen deficiency in order to provide at least 28 evaluable acute bleeding episodes and/or surgical procedures.
- Conditions
- Congenital Afibrinogenemia, Hypofibrinogenemia
- Being tested
- FIB Grifols
- Medical subject headings
- Afibrinogenemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FIB GrifolsBiological
Also called Fibrinogen
FIB Grifols is the IP and will be administered via slow intravenous (IV) infusion at a rate not to exceed 5 mL/minute.
Dosing will be individually calculated for each subject based on the target plasma fibrinogen level according to the type of bleeding, measured actual plasma fibrinogen level before infusion, and body weight. The IP will be administered according to the nominal potency of the product.
| Study group | Role | What they receive |
|---|---|---|
| FIB Grifols | Experimental | FIB Grifols is the IP and will be administered via slow intravenous (IV) infusion at a rate not to exceed 5 mL/minute.
Dosing will be individually calculated for each subject based on the target plasma fibrinogen level according to the type of bleeding, measured actual plasma fibrinogen level before infusion, and body weight. The IP will be administered according to the nominal potency of the product. Receives: Biological: FIB Grifols |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hemostatic efficacy of FIB Grifols in on-demand treatment of all documented acute bleeding episodes as assessed by the Independent Endpoint Adjudication Committee (IEAC) Using a 4-Point Scale
To clinically evaluate the hemostatic efficacy of FIB Grifols in on-demand treatment of all documented acute bleeding episodes in subjects with congenital fibrinogen deficiency as determined by the the proportion of successful treatment responses (Yes/No) indicated by a hemostatic efficacy rating of "Excellent" or "Good" as assessed by the IEAC using a 4 point scale. Hemostatic efficacy will be determined at end of treatment observation period (either at Day 3 for non-life threatening bleeding episodes, at Day 7 for life-threatening bleeding episodes, or at 24 hours after the end of the last infusion of FIB Grifols, whichever is later).
Measured: Up to Day 7
Hemostatic efficacy of FIB Grifols in perioperative management of bleeding during and after all documented surgical procedures as assessed by the IEAC Using a 4-Point Scale
To clinically evaluate the hemostatic efficacy of FIB Grifols in the perioperative management of bleeding during and after all documented surgical procedures in subjects with congenital fibrinogen deficiency as determined by the the proportion of successful treatment responses (Yes/No) indicate by an adjudicated hemostatic efficacy rating of "Success" in preventing excessive bleeding during and after all documented surgical procedures as assessed by the IEAC using a 4 point scale. Hemostatic efficacy will be determined at end of treatment observation period (either at Day 3 for minor surgical procedures, at Day 7 for major surgical procedures, or at 24 hours after the end of the last infusion of FIB Grifols, whichever is later).
Measured: Up to Day 7
Secondary outcomes (6)
Hemostatic Efficacy of FIB Grifols in Treating the First Documented Acute Bleeding Episode as Assessed by the IEAC Using a 4-Point Scale.
Overall clinical assessment of the hemostatic efficacy of FIB Grifols in treating the first documented acute bleeding episode 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'. This secondary efficacy endpoint will be assessed by the IEAC.
Hemostatic efficacy will be determined at end of treatment observation period (either at Day 3 for non-life threatening bleeding episodes, at Day 7 for life-threatening bleeding episodes, or at 24 hours after the end of the last infusion of FIB Grifols, whichever is later).
Measured: Up to Day 7
Hemostatic Efficacy of FIB Grifols in Preventing Excessive Bleeding During and After the First Documented Surgical Procedure as Assessed by the IEAC Using a 4-Point Scale
Overall clinical assessment of the hemostatic efficacy of FIB Grifols in preventing excessive bleeding during and after the first documented surgical procedure 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'. This secondary efficacy endpoint will be assessed by the IEAC.
Hemostatic efficacy will be determined at end of treatment observation period (either at Day 3 for minor surgical procedures, at Day 7 for major surgical procedures, or at 24 hours after the end of the last infusion of FIB Grifols, whichever is later).
Measured: Up to Day 7
Hemostatic Efficacy of FIB Grifols in Treating the First Acute Bleeding Episode
Overall clinical assessment of the hemostatic efficacy of FIB Grifols in treating the first acute bleeding episode as assessed by the principal investigator at each trial site 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'.
Hemostatic efficacy will be determined at end of treatment observation period (either at Day 3 for non-life threatening bleeding episodes, at Day 7 for life-threatening bleeding episodes, or at 24 hours after the end of the last infusion of FIB Grifols, whichever is later).
Measured: Up to Day 7
Hemostatic Efficacy of FIB Grifols in Treating All Acute Bleeding Episodes
Overall clinical assessment of the hemostatic efficacy of FIB Grifols in treating all acute bleeding episodes as assessed by the principal investigator at each trial site 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'.
Hemostatic efficacy will be determined at end of treatment observation period (either at Day 3 for non-life threatening bleeding episodes, at Day 7 for life-threatening bleeding episodes, or at 24 hours after the end of the last infusion of FIB Grifols, whichever is later).
Measured: Up to Day 7
Hemostatic Efficacy of FIB Grifols in Preventing Excessive Bleeding Intra-operatively for All Surgical Procedures.
Clinical assessment of the hemostatic efficacy of FIB Grifols in preventing excessive bleeding intra-operatively for all surgical procedures as assessed by the surgeon (defined as the licensed medical professional performing the invasive procedure [eg, dentist]) 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'.
Hemostatic efficacy will be determined at end of treatment observation period (either at Day 3 for minor surgical procedures, at Day 7 for major surgical procedures, or at 24 hours after the end of the last infusion of FIB Grifols, whichever is later).
Measured: Up to Day 7
Hemostatic Efficacy of FIB Grifols in Preventing Excessive Bleeding After All Surgical Procedures (ie, Post-operative)
Clinical assessment of the hemostatic efficacy of FIB Grifols in preventing excessive bleeding after all surgical procedures (ie, post-operative) as assessed by the principal investigator at each trial site 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'.
Hemostatic efficacy will be determined at end of treatment observation period (either at Day 3 for minor surgical procedures, at Day 7 for major surgical procedures, or at 24 hours after the end of the last infusion of FIB Grifols, whichever is later).
Measured: Up to Day 7
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2020
- Study startDec 2023 (estimated)
- Primary completionMar 2025 (estimated)
- Study completionMar 2025 (estimated)
Why it stopped: The study was stopped due to a business decision.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital AfibrinogenemiaStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Instituto Grifols, S.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04636268
- Sponsor's protocol ID
- GC1801
- Other IDs
- 2018-004005-81, 2018
- Status last verified
- Oct 2023
- First submitted
- 16 Mar 2020
- Last updated
- 23 Oct 2023
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Multicenter, Open-label, Single-arm Study to Evaluate the Efficacy and Safety of Human Plasma-derived Fibrinogen Concentrate (FIB Grifols) in Subjects With Congenital Afibrinogenaemia and Severe Hypofibrinogenemia Requiring Either On-demand Treatment for Acute Bleeding or Surgical Prophylaxis
- ClinicalTrials.gov ID
- NCT04636268
- Acronym / sponsor ID
- GC1801
- Conditions
- Congenital Afibrinogenemia, Hypofibrinogenemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: The study was stopped due to a business decision.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Instituto Grifols, S.A.Industry (pharma, biotech, devices)
- Study start
- Dec 2023 (estimated)
- Primary completion
- Mar 2025 (estimated)
- Study completion
- Mar 2025 (estimated)
- First posted
- 19 Nov 2020 (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.
This is a phase 3, multi-center, prospective, open-label, single-arm, clinical trial to be carried out in subjects with congenital fibrinogen deficiency manifested as afibrinogenemia or severe hypofibrinogenemia.
This clinical trial is planned to be performed at study sites in multiple countries. It is planned to include a maximum of 32 adult and pediatric subjects with congenital fibrinogen deficiency in order to provide at least 28 evaluable acute bleeding episodes and/or surgical procedures.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FIB Grifols | Experimental | Biological: FIB GrifolsFIB Grifols is the IP and will be administered via slow intravenous (IV) infusion at a rate not to exceed 5 mL/minute. Dosing will be individually calculated for each subject based on the target plasma fibrinogen level according to the type of bleeding, measured actual plasma fibrinogen level before infusion, and body weight. The IP will be administered according to the nominal potency of the product. |
Who may qualify
- Ages
- 6 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Male or female subject between 6 to 70 years of age.
- Signed and dated written ICF, or the subject's parent or legal guardian signs and dates the ICF where applicable, and the Subject Authorization Form (SAF) where applicable. Pediatric subjects, as defined by local regulations, will be asked to sign an age appropriate assent form.
- Diagnosed with congenital fibrinogen deficiency manifested as afibrinogenemia or severe hypofibrinogenemia (fibrinogen <50 mg/dL) and expected to require treatment for acute bleeding (either spontaneous or after trauma [defined as any accidental event leading to acute bleeding]), or prophylaxis of bleeding before a surgical intervention or invasive procedure.
- Fibrinogen level < 50 mg/dL determined by Clauss method at baseline (sample drawn within 24 hours prior to infusion on Infusion 1 Day 1 Visit).
- Female subjects of child-bearing potential must have a negative test for pregnancy blood or urine human chorionic gonadotropin (HCG)-based assay at baseline (sample drawn within 24 hours prior to infusion on Infusion 1 Day 1 Visit).
Key exclusion criteria (13 listed)
- Has acquired (secondary) fibrinogen deficiency.
- Diagnosed with dysfibrinogenemia.
- Has known antibodies against fibrinogen.
- Has history of anaphylaxis or severe systemic response to any drug or blood-derived product.
- Has history of intolerance to any component of the IP.
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 |
|---|---|---|
| FIB GrifolsAlso: FibrinogenFIB Grifols is the IP and will be administered via slow intravenous (IV) infusion at a rate not to exceed 5 mL/minute. Dosing will be individually calculated for each subject based on the target plasma fibrinogen level according to the type of bleeding, measured actual plasma fibrinogen level before infusion, and body weight. The IP will be administered according to the nominal potency of the product. | Biological | FIB Grifols |
Primary outcome
Hemostatic efficacy of FIB Grifols in on-demand treatment of all documented acute bleeding episodes as assessed by the Independent Endpoint Adjudication Committee (IEAC) Using a 4-Point Scale
Time frame: Up to Day 7
To clinically evaluate the hemostatic efficacy of FIB Grifols in on-demand treatment of all documented acute bleeding episodes in subjects with congenital fibrinogen deficiency as determined by the the proportion of successful treatment responses (Yes/No) indicated by a hemostatic efficacy rating of "Excellent" or "Good" as assessed by the IEAC using a 4 point scale. Hemostatic efficacy will be determined at end of treatment observation period (either at Day 3 for non-life threatening bleeding episodes, at Day 7 for life-threatening bleeding episodes, or at 24 hours after the end of the last infusion of FIB Grifols, whichever is later).
Hemostatic efficacy of FIB Grifols in perioperative management of bleeding during and after all documented surgical procedures as assessed by the IEAC Using a 4-Point Scale
Time frame: Up to Day 7
To clinically evaluate the hemostatic efficacy of FIB Grifols in the perioperative management of bleeding during and after all documented surgical procedures in subjects with congenital fibrinogen deficiency as determined by the the proportion of successful treatment responses (Yes/No) indicate by an adjudicated hemostatic efficacy rating of "Success" in preventing excessive bleeding during and after all documented surgical procedures as assessed by the IEAC using a 4 point scale. Hemostatic efficacy will be determined at end of treatment observation period (either at Day 3 for minor surgical procedures, at Day 7 for major surgical procedures, or at 24 hours after the end of the last infusion of FIB Grifols, whichever is later).
6 secondary outcomes
Hemostatic Efficacy of FIB Grifols in Treating the First Documented Acute Bleeding Episode as Assessed by the IEAC Using a 4-Point Scale.
Time frame: Up to Day 7
Hemostatic Efficacy of FIB Grifols in Preventing Excessive Bleeding During and After the First Documented Surgical Procedure as Assessed by the IEAC Using a 4-Point Scale
Time frame: Up to Day 7
Hemostatic Efficacy of FIB Grifols in Treating the First Acute Bleeding Episode
Time frame: Up to Day 7
Hemostatic Efficacy of FIB Grifols in Treating All Acute Bleeding Episodes
Time frame: Up to Day 7
Hemostatic Efficacy of FIB Grifols in Preventing Excessive Bleeding Intra-operatively for All Surgical Procedures.
Time frame: Up to Day 7
Hemostatic Efficacy of FIB Grifols in Preventing Excessive Bleeding After All Surgical Procedures (ie, Post-operative)
Time frame: Up to Day 7
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 23 Oct 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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing FIB Grifols in people with Congenital Afibrinogenemia and Hypofibrinogenemia, run by Instituto Grifols, S.A..
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 13 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 23 Oct 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?
Hemostatic efficacy of FIB Grifols in on-demand treatment of all documented acute bleeding episodes as assessed by the Independent Endpoint Adjudication Committee (IEAC) Using a 4-Point Scale (time frame: Up to Day 7). 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 23 Oct 2023.