Use of a TGA and TEM in the Assessment of the Efficacy of Treatment With APCC or rFVIIa (Thrombus)

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2013 by Stowarzyszenie Pomocy Chorym na Zakrzepicę i Skazy Krwotoczne Thrombus
Sponsor:
Information provided by (Responsible Party):
Stowarzyszenie Pomocy Chorym na Zakrzepicę i Skazy Krwotoczne Thrombus
ClinicalTrials.gov Identifier:
NCT01856751
First received: May 14, 2013
Last updated: June 24, 2014
Last verified: May 2013
  Purpose

Occurrence of inhibitors to coagulation factor VIII is diagnosed in ~30% patients with haemophilia A. Presence of inhibitor with a titre >5 BU/ml requires the use of by-passing agents: recombinant activated Factor VIIa concentrate (rFVIIa) and/or activated prothrombin complex concentrate (APCC). Similarly, haemorrhagic complications in patients with acquired haemophilia and inhibitor titre >5 BU/ml should be treated with by-passing agents.

Response to treatment with by-passing agents is patient-specific, and can vary in the same patient during subsequent bleedings. Some patients have good response to both products, however in other patients a better bleeding control is provided by one of the mentioned above agents (APCC or rFVIIa). There are clinical situations when severe bleedings requires an alternate use of both these agents.

Traditional methods of laboratory tests used post-treatment in patients with haemophilia without inhibitors are useless in the presence of inhibitor. Laboratory monitoring of therapy with by-passing agents is possible with the use of global tests for the coagulation process assessment, which are as follows: thrombin generation assay (TGA) and thromboelastometry (TEM).

Several studies revealed that TGA allows a monitoring of therapy with by-passing agents in patients with haemophilia A and inhibitor - the choice of the most effective treatment option - agent type and its dose, as well as laboratory assessment of treatment efficacy.

Up to date, laboratory tests assessing the efficacy of by-passing agents in patients with acquired haemophilia were not conducted.

In Factor VIII or IX deficiency conditions, fibrin's fibres generated by thrombin are morphologically thicker, and blood clots have increased susceptibility to fibrinolytic enzymes. Blood clot stability may be assessed with the use of thromboelastometry (TEM). We can hypothesize that simultaneous use of TGA and TEM methods may allow for an assessment of patient's individual response to therapy with by-passing agents. Clinical significance of the minimal dose of APCC and rFVIIa, needed to TGA and TEM normalization, requires further studies.

Tests' purpose: Examination of the hypothesis that simultaneous use of thrombin generation assay (TGA) and thromboelastometry (TEM) may facilitate the choice of optimal therapy with by-passing agents and laboratory monitoring of efficacy of those agents in patients with acquired haemophilia or haemophilia A with inhibitor.


Condition
Haemophilia

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Use of a TGA and TEM in the Assessment of the Efficacy of Treatment With APCC or rFVIIa Concentrate in Patients With Acquired Haemophilia and in Patients With Haemophilia A With Inhibitors

Resource links provided by NLM:


Further study details as provided by Stowarzyszenie Pomocy Chorym na Zakrzepicę i Skazy Krwotoczne Thrombus:

Primary Outcome Measures:
  • Assessment of patient's individual response to therapy with by-passing agents by simultaneous use of TGA and TEM methods. [ Time Frame: 48 hours ] [ Designated as safety issue: No ]
    This is non-inverventional study as the protocol will not assign specific treatment to the particular subjects of the study. Patients will be treated with APCC or rFVIIa based on the experience of the study site. Patients are prescribed a treatment according to their physician's judgement or local clinical practice. This is observation of the everyday clinical practise on site.


Biospecimen Retention:   Samples Without DNA

this data is not applicable


Estimated Enrollment: 80
Study Start Date: April 2014
Estimated Study Completion Date: June 2016
Estimated Primary Completion Date: April 2016 (Final data collection date for primary outcome measure)
Groups/Cohorts
haemophilia
Patients with acquired haemophilia. Patients with haemophilia A with inhibitor.

Detailed Description:

This section is Not applicable

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

60 patients with acquired haemophilia 20 patients with congenital haemophilia A with inhibitor

Criteria

Inclusion Criteria:

  • patients with acquired haemophilia
  • patients with congenital haemophilia A with inhibitor

Exclusion Criteria:

  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: NCT01856751

Contacts
Contact: Krystyna M Zawilska, MD, PhD 48618333949 k.zawilska@interia.pl

Locations
Poland
Interlab, ul. 28 Czerwca 1956 nr 161 Recruiting
Poznan, Poland, 61-505
Sponsors and Collaborators
Stowarzyszenie Pomocy Chorym na Zakrzepicę i Skazy Krwotoczne Thrombus
Investigators
Principal Investigator: Maria Podolak-Dawidziak, MD, PhD Blood Neoplasms and Bone Marrow Transplantation of the Wroclaw Medical University
Principal Investigator: Andrzej Mital, MD Hematology Clinic of Gdansk Medical University
Principal Investigator: Jerzy Windyga, MD, PhD, Institute of Heamatology and Transfusiology in Warsaw
Principal Investigator: Krystyna Zawilska, MD, PhD Centrum Diagnostyczno Lecznicze Interlab Poznan
Principal Investigator: Krzysztof Chojnowski, MD PhD Klinika Hematologii Uniwersytetu Medycznego w Łodzi Wojewódzki Szpital Specjalistyczny im. M. Kopernika ul. Ciołkowskiego 2, 93-510 Łódź
Principal Investigator: Jacek Musiał, MD PhD Oddział Kliniczny Hematologii SP ZOZ Szpital Uniwersytecki w Krakowie ul. Mikołaja Kopernika 36 31-501 Kraków
  More Information

No publications provided

Responsible Party: Stowarzyszenie Pomocy Chorym na Zakrzepicę i Skazy Krwotoczne Thrombus
ClinicalTrials.gov Identifier: NCT01856751     History of Changes
Other Study ID Numbers: TGA-TEM
Study First Received: May 14, 2013
Last Updated: June 24, 2014
Health Authority: Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products

Keywords provided by Stowarzyszenie Pomocy Chorym na Zakrzepicę i Skazy Krwotoczne Thrombus:
Haemophilia
TGA
TEM

Additional relevant MeSH terms:
Hemophilia A
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