Now Available: Final Rule for FDAAA 801 and NIH Policy on Clinical Trial Reporting

Human Fibrinogen Concentrate (FGTW) in Pediatric Patients With Congenital Fibrinogen Deficiency

This study has been completed.
Information provided by (Responsible Party):
Laboratoire français de Fractionnement et de Biotechnologies Identifier:
First received: February 26, 2014
Last updated: February 11, 2016
Last verified: February 2016
The aim of the study is to evaluate clinical pharmacology, efficacy and safety of FGTW in pediatric patients with congenital fibrinogen deficiency.

Condition Intervention Phase
Hypofibrinogenemia, Congenital
Afibrinogenemia, Congenital
Drug: biological: human fibrinogen concentrate
Phase 2
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Clinical Pharmacology, Efficacy and Safety Study of FGTW in Paediatric Patients With Severe Congenital Fibrinogen Deficiency

Resource links provided by NLM:

Further study details as provided by Laboratoire français de Fractionnement et de Biotechnologies:

Primary Outcome Measures:
  • Investigator's overall assessment of efficacy of FGTW on hemostasis using a 4-point scale at the end of each bleeding or surgical episode. [ Time Frame: 6 hours or up to 5 days ] [ Designated as safety issue: No ]
  • Terminal half life for Fibrinogen antigen and activity [ Time Frame: 5 days ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Adverse Events [ Time Frame: Participants will be followed for the duration of their participation in the study, an expected average of 1 year ] [ Designated as safety issue: Yes ]

Enrollment: 16
Study Start Date: January 2014
Study Completion Date: December 2015
Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: FGTW Drug: biological: human fibrinogen concentrate


Ages Eligible for Study:   up to 12 Years   (Child)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Signed and dated informed consent form by parents or a legal representative
  • Age less or equal to 12 years old
  • Patients with inherited afibrinogenemia or severe inherited hypofibrinogenemia
  • Negative results on HCG-based pregnancy test for females of childbearing potential (presence of menstruation)

Exclusion Criteria:

  • Dysfibrinogenemia
  • Acquired fibrinogen deficiency
  • Suspected present or past anticoagulation inhibitor
  • Personal history of venous or arterial thrombosis or thromboembolic event
  • Co-morbidity with other/unrelated coagulopathies
  • Administration of any fibrinogen concentrate or fibrinogen containing blood product during the last 15 days
  • Permanent treatment with antithrombotic or anti-platelet agents such as heparins, anti-IIa or anti-Xa agents, aspirin, clopidogrel and NSAIDs.
  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 identifier: NCT02094430

Hopital Necker enfants malades
Paris, France, 75015
Hôpital Hôtel Dieu
Beirut, Lebanon
Hôpital d'enfants - CHU Avicenne
Rabat, Morocco
Faculty Ihsan Dogramaci Children's Hospital
Ankara, Turkey
Sponsors and Collaborators
Laboratoire français de Fractionnement et de Biotechnologies
  More Information

Responsible Party: Laboratoire français de Fractionnement et de Biotechnologies Identifier: NCT02094430     History of Changes
Other Study ID Numbers: FGTW-1004 
Study First Received: February 26, 2014
Last Updated: February 11, 2016
Health Authority: France: Agence Nationale de Sécurité du Médicament et des produits de santé
France: Committee for the Protection of Personnes
Lebanon: Institutional Review Board
Morocco: Ministry of Public Health
Morocco: Ethics Committee
Turkey: Ethics Committee
Turkey: Ministry of Health

Additional relevant MeSH terms:
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn processed this record on October 21, 2016