Phase 3 study to evaluate the efficacy and safety of human plasma-derived fibrinogen concentrate (FIB Grifols) in subjects with congenital afibrinogenaemia and severe hypofibrinogenemia

Phase 3

• Conditions: Congenital fibrinogen deficiency (afibrinogenemia or severe hypofibrinogenemia); afibrinogenemia or severe hypofibrinogenemia; T45.6

• Registration Number: LBCTR2021024523
• Lead Sponsor: Instituto Grifols, S.A.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2021-03-21
• Last Update Posted: 19 August 2024

Recruitment & Eligibility

• Status: Pending
• Sex: All
• Target Recruitment: 32

Inclusion Criteria

A subject must meet all the following inclusion criteria to be eligible for participation in this study.
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).
a. Female subjects/partners of child-bearing potential include any female who has experienced menarche and who has not undergone successful surgical sterilization (eg, hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or are not postmenopausal (post menopausal is defined as amenorrhea for >12 consecutive months or women on hormone replacement therapy with documented serum follicle stimulating hormone level <35 mIU/mL). Even women who are using oral, implanted or injectable contraceptive hormones, mechanical products such as an intrauterine device [IUD], or barrier methods (eg, diaphragm, condoms, spermicides) to prevent pregnancy, or are practicing abstinence or where their partner is sterile (eg, vasectomy), should be considered to be of child bearing potential.
6. Willing to comply with all aspects of the clinical trial protocol, including blood sampling, for the entire duration of the study.

Exclusion Criteria

Exclusion Criteria
A subject meeting any of the following exclusion criteria is NOT eligible for participation in the study.
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.
a. True abstinance: when this is in line with the preferred and usual lifestyle of the subject (Periodic abstinance [eg, calendar, ovulation, symptothermal, post-ovulation methods], declaration of abstinance for the duration of a trial, and withdrawal are not acceptable methods of contraception.
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.

Study & Design

• Study Type: Interventional
• Study Design: Not specified