Fibrinogen Concentrate (Human) − Efficacy and Safety Study
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.
|Afibrinogenemia Hypofibrinogenemia Fibrinogen Deficiency||Biological: Fibrinogen Concentrate, Human (FCH) Biological: Cryoprecipitate||Phase 3|
|Study Design:||Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||Efficacy and Safety of Fibrinogen Concentrate (Human) (FCH) for On-demand Treatment of Acute Bleeding in Subjects With Congenital Fibrinogen Deficiency|
- Clinical assessment of hemostatic efficacy [ Time Frame: 24 hours after last infusion or at Day 14 (whichever occurs first) ]
- Maximum clot firmness (MCF) [ Time Frame: Prior to and 60 minutes after the end of each infusion ]
- Fibrinogen plasma level [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: Day 1 to Day 45 ]
|Study Start Date:||October 2009|
|Study Completion Date:||March 2014|
|Primary Completion Date:||March 2014 (Final data collection date for primary outcome measure)|
|Experimental: Prospective Arm||
Biological: 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.
Patients that received on-demand treatment with Cryoprecipitate for a classified bleeding event (minor or major) with a documented hemostatic efficacy assessment.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00916656
|Study Director:||Program Director, Clinical R&D||CSL Behring|