NCT02094430

Brief Summary

The aim of the study is to evaluate clinical pharmacology, efficacy and safety of FGTW in pediatric patients with congenital fibrinogen deficiency.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
16

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jan 2014

Geographic Reach
4 countries

4 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2014

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

February 26, 2014

Completed
23 days until next milestone

First Posted

Study publicly available on registry

March 21, 2014

Completed
1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2015

Completed
Last Updated

February 12, 2016

Status Verified

February 1, 2016

Enrollment Period

1.9 years

First QC Date

February 26, 2014

Last Update Submit

February 11, 2016

Conditions

Hypofibrinogenemia, CongenitalAfibrinogenemia, Congenital

Outcome Measures

Primary Outcomes (2)

  • Investigator's overall assessment of efficacy of FGTW on hemostasis using a 4-point scale at the end of each bleeding or surgical episode.

    6 hours or up to 5 days

  • Terminal half life for Fibrinogen antigen and activity

    5 days

Secondary Outcomes (1)

  • Adverse Events

    Participants will be followed for the duration of their participation in the study, an expected average of 1 year

Study Arms (1)

FGTW

EXPERIMENTAL
Drug: biological: human fibrinogen concentrate

Interventions

biological: human fibrinogen concentrateDRUG
FGTW

Eligibility Criteria

AgeUp to 12 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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)

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Hopital Necker enfants malades

Paris, 75015, France

Location

Hôpital Hôtel Dieu

Beirut, Lebanon

Location

Hôpital d'enfants - CHU Avicenne

Rabat, Morocco

Location

Faculty Ihsan Dogramaci Children's Hospital

Ankara, Turkey (Türkiye)

Location

MeSH Terms

Conditions

Afibrinogenemia

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 26, 2014

First Posted

March 21, 2014

Study Start

January 1, 2014

Primary Completion

December 1, 2015

Study Completion

December 1, 2015

Last Updated

February 12, 2016

Record last verified: 2016-02

Locations

LE(1)MO(1)TU(1)FR(1)