Fibrinogen Concentrate (Human) - Efficacy and Safety Study

NCT ID: NCT00916656

Last Updated: 2023-02-06

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE3
• Study Classification: INTERVENTIONAL
• Study Start Date: 2009-10-31
• Study Completion Date: 2014-03-31

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

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Prospective Arm

Group Type: EXPERIMENTAL

Fibrinogen Concentrate, Human (FCH)

Intervention Type: 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.

Historical Control

Group Type: OTHER

Cryoprecipitate

Intervention Type: BIOLOGICAL

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

Interventions

Fibrinogen Concentrate, Human (FCH)

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.

Intervention Type: BIOLOGICAL

Cryoprecipitate

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

Intervention Type: BIOLOGICAL

Other Intervention Names

Haemocomplettan P; RIASTAP

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

• 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
• Polytrauma, present or within 6 months prior to enrollment
• Suspicion of an anti-fibrinogen inhibitor as indicated by previous in-vivo recovery (IVR), if available (< 0.5 (mg/dL)/(mg/kg))
• Previous inclusion and treatment in the prospective part of the study
• Participation in any clinical study in the 30 days prior to enrollment

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

CSL Behring

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Program Director, Clinical R&D

Role: STUDY_DIRECTOR

CSL Behring

Other Identifiers

1475

Identifier Type: OTHER

Identifier Source: secondary_id

2007-004088-22

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

BI3023_3001

Identifier Type: -

Identifier Source: org_study_id