FIB Grifols Congenital Deficiency for On-demand Treatment... | NCT04636268

NCT04636268

Protocol Section

Identification Module

Results Section

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Annotation Section

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Document Section

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Derived Section

Miscellaneous Info Module

ID	Type	Description	Link
2018-004005-81	EudraCT Number

Status Module

Description Module

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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.	Biological: FIB Grifols

Interventions

Name	Type	Description	Arm Group Labels	Other Names
FIB Grifols	Biological	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.

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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).	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).	Up to Day 7

Secondary Outcomes

Measure	Description	Time Frame
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).

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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.

Exclusion Criteria:

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.
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.

ID	Term
D025861	Blood Coagulation Disorders, Inherited
D001778	Blood Coagulation Disorders
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases

ID	Term
D000209	Acute-Phase Proteins
D001798	Blood Proteins
D011506	Proteins
D000602	Amino Acids, Peptides, and Proteins