FIB Grifols Congenital Deficiency for On-demand Treatment and Surgical Prophylaxis

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.

Inclusion Criteria:

1. Male or female subject between 6 to 70 years of age.
2. 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.
3. 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.
4. 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).
5. 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).
6. Willing to comply with all aspects of the clinical trial protocol, including blood sampling, for the entire duration of the study.

Exclusion Criteria:

1. Has acquired (secondary) fibrinogen deficiency.
2. Diagnosed with dysfibrinogenemia.
3. Has known antibodies against fibrinogen.
4. Has history of anaphylaxis or severe systemic response to any drug or blood-derived product.
5. Has history of intolerance to any component of the IP.
6. Documented history of immunoglobulin A (IgA) deficiency and antibodies against IgA.
7. 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.
8. 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.
9. Has congenital or acquired bleeding disorders other than congenital fibrinogen deficiency.
10. Has life expectancy of less than 6 months.
11. Received FRT within 21 days prior to the Screening Visit.
12. Receiving, or having received within 3 months prior to the Screening Visit of this clinical trial, any investigational drug or device.
13. Is unlikely to adhere the protocol requirements, or is likely to be uncooperative, or unable to provide a storage sample prior to IP infusion.