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

December 17, 2015 updated by: Stowarzyszenie Pomocy Chorym na Zakrzepicę i Skazy Krwotoczne Thrombus

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

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.

Study Overview

Status

Status

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Unknown

Conditions

Conditions

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Detailed Description

This section is Not applicable

Study Type

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Observational

Enrollment (Anticipated)

Enrollment

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80

Contacts and Locations

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Study Contact

Study Contact

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Study Locations

  • Poland
      • Gdansk, Poland, 80-952
        • Recruiting
        • Katedra i Klinika Hematologii i Transplantologii Gdanski Uniwersytet Medyczny
        • Contact:
          • Andrzej Mital, MD, PhD
      • Krakow, Poland, 31-501
        • Recruiting
        • Szpital Uniwersytecki w Krakowie, Oddzial Kliniczny Alergii i Immunologii
        • Contact:
          • Jacek Musial, MD, PhD
      • Lodz, Poland, 93-510
        • Recruiting
        • Klinika Hematologii Uniwersytetu Medycznego w Lodzi Wojewodzki Szpital Specjalistyczny im. M. Kopernika
        • Contact:
          • Krzysztof Chojnowski, MD, PhD
      • Poznan, Poland, 61-505
        • Recruiting
        • Centrum Diagnostyczno - Lecznicze INTERLAB
        • Contact:
          • Krystyna Zawilska, MD,PhD
      • Warszawa, Poland, 02-776
        • Recruiting
        • Instytut Hematologii i Transfuzjologii w Warszawie
        • Contact:
          • Jerzy Windyga, MD, PhD
      • Wroclaw, Poland, 50-367
        • Recruiting
        • Katedra i Klinika Hematologii, Nowotworow Krwi i Transplantacji Szpiku, Akademia Medyczna
        • Contact:
          • Maria Podolak-Dawidziak, MD, PhD

Participation Criteria

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Eligibility Criteria

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Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

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

Description

Inclusion Criteria:

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

Exclusion Criteria:

Study Plan

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How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

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haemophilia
Patients with acquired haemophilia. Patients with haemophilia A with inhibitor.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

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Outcome Measure
Measure Description
Time Frame
Assessment of patient's individual response to therapy with by-passing agents by simultaneous use of TGA and TEM methods.
Time Frame: 48 hours
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.
48 hours

Collaborators and Investigators

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Sponsor

Sponsor

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Stowarzyszenie Pomocy Chorym na Zakrzepicę i Skazy Krwotoczne Thrombus

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Krystyna Zawilska, MD, PhD, Centrum Diagnostyczno - Lecznicze INTERLAB
  • Principal Investigator: Maria Podolak-Dawidziak, MD,PhD, Katedra i Klinika Hematologii, Nowotworow Krwi i Transplantacji Szpiku, Akademia Medyczna
  • Principal Investigator: Andrzej Mital, MD, PhD, Katedra i Klinika Hematologii i Transplantologii Gdanski Uniwersytet Medyczny
  • Principal Investigator: Jerzy Windyga, MD, PhD, Instytut Hematologii i Transfuzjologii w Warszawie
  • Principal Investigator: Krzysztof Chojnowski, MD, PhD, Klinika Hematologii Uniwersytetu Medycznego w Lodzi Wojewodzki Szpital Specjalistyczny im. M. Kopernika
  • Principal Investigator: Jacek Musial, MD, PhD, Szpital Uniwersytecki w Krakowie, Oddzial Kliniczny Alergii i Immunologii

Publications and helpful links

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General Publications

Study record dates

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Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 1, 2014

Primary Completion (Anticipated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 1, 2016

Study Completion (Anticipated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 1, 2016

Study Registration Dates

First Submitted

First Submitted

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May 14, 2013

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 16, 2013

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
May 17, 2013

Study Record Updates

Last Update Posted (Estimate)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
December 18, 2015

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 17, 2015

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
December 1, 2015

More Information

Terms related to this study

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Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • TGA-TEM

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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