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Fibrinogen Concentrate (Human) - Efficacy and Safety Study

Primary Purpose

Afibrinogenemia, Hypofibrinogenemia, Fibrinogen Deficiency

Status
Withdrawn
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
Fibrinogen Concentrate, Human (FCH)
Cryoprecipitate
Sponsored by
CSL Behring
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Afibrinogenemia focused on measuring Congenital fibrinogen deficiency

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm Type

    Experimental

    Other

    Arm Label

    Prospective Arm

    Historical Control

    Arm Description

    Outcomes

    Primary Outcome Measures

    Clinical assessment of hemostatic efficacy

    Secondary Outcome Measures

    Maximum clot firmness (MCF)
    Fibrinogen plasma level
    In vivo recovery of fibrinogen
    Virus safety markers

    Full Information

    First Posted
    June 8, 2009
    Last Updated
    February 2, 2023
    Sponsor
    CSL Behring
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    1. Study Identification

    Unique Protocol Identification Number
    NCT00916656
    Brief Title
    Fibrinogen Concentrate (Human) - Efficacy and Safety Study
    Official Title
    Efficacy and Safety of Fibrinogen Concentrate (Human) (FCH) for On-demand Treatment of Acute Bleeding in Subjects With Congenital Fibrinogen Deficiency
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    February 2023
    Overall Recruitment Status
    Withdrawn
    Study Start Date
    October 2009 (undefined)
    Primary Completion Date
    March 2014 (Actual)
    Study Completion Date
    March 2014 (Actual)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    CSL Behring

    4. Oversight

    Data Monitoring Committee
    No

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Afibrinogenemia, Hypofibrinogenemia, Fibrinogen Deficiency
    Keywords
    Congenital fibrinogen deficiency

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 3
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    Non-Randomized
    Enrollment
    0 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Prospective Arm
    Arm Type
    Experimental
    Arm Title
    Historical Control
    Arm Type
    Other
    Intervention Type
    Biological
    Intervention Name(s)
    Fibrinogen Concentrate, Human (FCH)
    Other Intervention Name(s)
    Haemocomplettan P, RIASTAP
    Intervention Description
    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
    Intervention Name(s)
    Cryoprecipitate
    Intervention Description
    Patients that received on-demand treatment with Cryoprecipitate for a classified bleeding event (minor or major) with a documented hemostatic efficacy assessment.
    Primary Outcome Measure Information:
    Title
    Clinical assessment of hemostatic efficacy
    Time Frame
    24 hours after last infusion or at Day 14 (whichever occurs first)
    Secondary Outcome Measure Information:
    Title
    Maximum clot firmness (MCF)
    Time Frame
    Prior to and 60 minutes after the end of each infusion
    Title
    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
    Title
    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
    Title
    Virus safety markers
    Time Frame
    Day 1 to Day 45

    10. Eligibility

    Sex
    All
    Accepts Healthy Volunteers
    No
    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
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Program Director, Clinical R&D
    Organizational Affiliation
    CSL Behring
    Official's Role
    Study Director

    12. IPD Sharing Statement

    Learn more about this trial

    Fibrinogen Concentrate (Human) - Efficacy and Safety Study

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