Fibrinogen Concentrate (Human) - Efficacy and Safety Study

NCT00916656 | Withdrawn Phase 3

• 3 other identifiers: BI3023_300114752007-004088-22
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is a multinational, multicenter, prospective, open-label historically controlled Phase IIIb non-inferiority clinical trial on the efficacy and safety of Fibrinogen Concentrate (Human). It is estimated that 150-300 patients in the U.S. suffer from afibrinogenemia. Substitution with cryoprecipitate or alternative treatments have limited safety and efficacy. The primary purpose of the study is to demonstrate the hemostatic efficacy of Fibrinogen Concentrate (Human) by adequately controlling acute bleeding (spontaneous or after trauma) in patients with congenital fibrinogen deficiency (afibrinogenemia and hypofibrinogenemia). Cryoprecipitate hemostatic efficacy data from a retrospective physician survey will be used as a historical control.

Conditions

Afibrinogenemia; Hypofibrinogenemia; Fibrinogen Deficiency

Keywords

Congenital fibrinogen deficiency

Outcome Measures

Primary Outcomes (1)

• Clinical assessment of hemostatic efficacy

  24 hours after last infusion or at Day 14 (whichever occurs first)

Secondary Outcomes (4)

• Maximum clot firmness (MCF)

  Prior to and 60 minutes after the end of each infusion

• Fibrinogen plasma level

  60 minutes, 3 hours, 6 hours, and 12 hours after the end of the first infusion; before and 60 minutes after each subsequent infusion

• In vivo recovery of fibrinogen

  60 minutes, 3 hours, 6 hours and 12 hours after the end of the first infusion; before and 60 minutes after the end of each subsequent infusion and at the time of the overall clinical assessment of hemostatic efficacy

• Virus safety markers

  Day 1 to Day 45

Study Arms (2)

Prospective Arm

EXPERIMENTAL

Biological: Fibrinogen Concentrate, Human (FCH)

Historical Control

OTHER

Biological: Cryoprecipitate

Interventions

Fibrinogen Concentrate, Human (FCH) BIOLOGICAL

Intravenous (IV) infusion to reach the peak target levels of 100 mg/dL with an accepted lower limit of 80 mg/dL on at least 3 subsequent days for minor bleeding episodes and 150 mg/dL with an accepted lower limit of 130 mg/dL on at least 7 subsequent days for major bleeding episodes. If a subject's fibrinogen level is not known on Day 1, at the time treatment is initiated for the acute bleed (e.g., because they did not have a screening visit), the starting dose is to be 70 mg/kg b.w. Otherwise, the dose will be calculated individually.

Also known as: Haemocomplettan P, RIASTAP

Prospective Arm

Cryoprecipitate BIOLOGICAL

Patients that received on-demand treatment with Cryoprecipitate for a classified bleeding event (minor or major) with a documented hemostatic efficacy assessment.

Historical Control

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Documented congenital fibrinogen deficiency (afibrinogenemia and hypofibrinogenemia), expected to require treatment for bleeding
• Presenting with an episode of acute bleeding (either spontaneous or after trauma) not requiring surgery
• Provide informed consent

You may not qualify if:

• Life expectancy \< 6 months
• Bleeding disorder other than congenital fibrinogen deficiency, but including dysfibrinogenemia
• Treatment with any investigational medicinal product (IMP) in the 30 days prior to enrollment
• Treatment with any fibrinogen concentrate or other fibrinogen containing blood product in the 2 weeks prior to enrollment
• Treatment with any coagulation active drug (i.e., non-steroidal-antirheumatics, warfarin, cumarin derivates, platelet aggregation inhibitors) in 1 week prior to enrollment or as a planned or expected medication during the time period from Day 1 until 24 hours after the last FCH infusion
• Presence or history of hypersensitivity to FCH
• Presence or history of deep vein thrombosis or pulmonary embolism within 1 year prior to enrollment
• Presence or history of arterial thrombosis within 1 year prior to enrollment
• Presence or history of hypersensitivity to human plasma proteins
• Presence or history of esophageal varicose bleeding
• End stage liver disease (i.e., Child Pugh score B or C)
• Planned or expected surgery (i.e., for bleedings from aneurysm or splenic rupture)
• Pregnancy, or an intention to become pregnant during the study
• Currently breast-feeding, or with the intention of breast-feeding during the study
• Human immunodeficiency virus (HIV) positive

Sponsors & Collaborators

• CSL Behring: lead

MeSH Terms

Conditions

Afibrinogenemia

Interventions

Fibrinogen; cryoprecipitate coagulum

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Acute-Phase Proteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Blood Coagulation Factors; Protein Precursors; Biological Factors

Study Officials

• Program Director, Clinical R&D

  CSL Behring

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 8, 2009
• First Posted: June 9, 2009
• Study Start: October 1, 2009
• Primary Completion: March 1, 2014
• Study Completion: March 1, 2014
• Last Updated: February 6, 2023

Record last verified: 2023-02