Fibrinogen Concentrate (Human) - Efficacy and Safety Study

February 2, 2023 updated by: CSL Behring

Efficacy and Safety of Fibrinogen Concentrate (Human) (FCH) for On-demand Treatment of Acute Bleeding in Subjects With Congenital Fibrinogen Deficiency

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.

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

Study Type

Interventional

Phase

  • Phase 3

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • ADULT
  • OLDER_ADULT
  • CHILD

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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:
  • RIASTAP
  • Haemocomplettan P
OTHER: 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Clinical assessment of hemostatic efficacy
Time Frame: 24 hours after last infusion or at Day 14 (whichever occurs first)
24 hours after last infusion or at Day 14 (whichever occurs first)

Secondary Outcome Measures

Outcome Measure
Time Frame
Maximum clot firmness (MCF)
Time Frame: Prior to and 60 minutes after the end of each infusion
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
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
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
Day 1 to Day 45

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

CSL Behring

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

October 1, 2009

Primary Completion (ACTUAL)

March 1, 2014

Study Completion (ACTUAL)

March 1, 2014

Study Registration Dates

First Submitted

June 8, 2009

First Submitted That Met QC Criteria

June 8, 2009

First Posted (ESTIMATE)

June 9, 2009

Study Record Updates

Last Update Posted (ACTUAL)

February 6, 2023

Last Update Submitted That Met QC Criteria

February 2, 2023

Last Verified

February 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BI3023_3001
  • 1475 (CSL Behring)
  • 2007-004088-22 (EUDRACT_NUMBER)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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