Fibrinogen Concentrate (Human) − Efficacy and Safety Study

This study has been withdrawn prior to enrollment.
Sponsor:
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT00916656
First received: June 8, 2009
Last updated: May 6, 2014
Last verified: May 2014
  Purpose

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.


Condition Intervention Phase
Afibrinogenemia
Hypofibrinogenemia
Fibrinogen Deficiency
Biological: Fibrinogen Concentrate, Human (FCH)
Biological: Cryoprecipitate
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by CSL Behring:

Primary Outcome Measures:
  • Clinical assessment of hemostatic efficacy [ Time Frame: 24 hours after last infusion or at Day 14 (whichever occurs first) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Maximum clot firmness (MCF) [ Time Frame: Prior to and 60 minutes after the end of each infusion ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • Virus safety markers [ Time Frame: Day 1 to Day 45 ] [ Designated as safety issue: Yes ]

Enrollment: 0
Study Start Date: October 2009
Study Completion Date: March 2014
Primary Completion Date: March 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

Other Names:
  • Haemocomplettan P
  • RIASTAP
Historical Control Biological: Cryoprecipitate
Patients that received on-demand treatment with Cryoprecipitate for a classified bleeding event (minor or major) with a documented hemostatic efficacy assessment.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00916656

Sponsors and Collaborators
CSL Behring
Investigators
Study Director: Program Director, Clinical R&D CSL Behring
  More Information

Additional Information:
No publications provided

Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT00916656     History of Changes
Other Study ID Numbers: BI3023_3001, 1475, 2007-004088-22
Study First Received: June 8, 2009
Last Updated: May 6, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by CSL Behring:
Congenital fibrinogen deficiency

Additional relevant MeSH terms:
Afibrinogenemia
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on August 28, 2014