Observational Registry of the Treatment of Glanzmann's Thrombasthenia

December 19, 2014 updated by: Novo Nordisk A/S

Treatment of Glanzmann's Thrombasthenia: A Prospective Observational Registry

This observational registry is conducted in Europe, Asia, Africa and the United States of America (USA).

The purpose of the registry is to evaluate the efficacy and safety of activated recombinant human factor VII (rFVIIa) during bleeding episodes and for the prevention of bleeding during invasive procedures/surgery in patients with Glanzmann's thrombasthenia (GT) with past or present refractoriness to platelet transfusions. Attention will be directed towards complications related to thrombo-embolic events and concomitant medications especially antifibrinolytics.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

218

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients with GT. There is no limit to the number of bleeding episodes or preventions of bleeding during invasive procedures/surgery that can be entered for each patient within the lifetime of the registry. Patients will receive standard care according to local practice, thus any systemic haemostatic treatment (with or without antifibrinolytic drugs or other agents) considered useful by the centres for treatment of GT, are included in the registry.

Description

Inclusion Criteria:

  • Patients with congenital GT defined as patients with lifelong bleeding tendency characterised by impaired or absent platelet aggregation, impaired clot retraction and prolonged bleeding time or prolonged platelet function analyser closure time. The patient has normal platelet counts and platelet morphology. Optional diagnosis criteria are quantitative or qualitative evaluation of GP (Glycoprotein) IIb/IIIa receptor including flow cytometry and identification of gene defects
  • Signed informed consent by the patient or next of kin or legally acceptable representative to collect data on treatment of a given bleeding episode or surgical event as specified in the protocol. If informed consent is provided by the next of kin or legally acceptable representative, consent must also be obtained from the patient as soon as he/she is able to do so. Informed consent must be obtained before entry of data into the registry

Exclusion Criteria:

  • Patients with acquired thrombasthenic states caused by autoimmune disorders (acute or chronic) or drugs

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

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
A
Drug: activated recombinant human factor VII
A prospective, observational multi-national registry collecting data and evaluating the efficacy and safety of rFVIIa in patients with GT with past or present refractoriness to platelet transfusions. The registry will also collect data from a broader range of GT patients treated with systemic haemostatic treatment (with or without antifibrinolytic drugs or other agents) used in the clinics. Data collection will continue for a maximum of six years. Baseline data as well as data obtained during either bleeding episodes or invasive procedures/surgeries will be recorded in the registry.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
For bleeding episodes: Overall efficacy evaluated by the caregiver/patient
Time Frame: within 30 days of end of treatment
within 30 days of end of treatment
For surgery including invasive and dental procedures: Haemoglobin level
Time Frame: prior to surgery and 24 hours after surgery
prior to surgery and 24 hours after surgery
For surgery including invasive and dental procedures: Overall haemostatic evaluation by the surgeon
Time Frame: 24 hours after surgery
24 hours after surgery

Secondary Outcome Measures

Outcome Measure
Time Frame
Changes in laboratory parameters (prothrombin time, platelet count, fibrinogen), if available
Time Frame: at the time of administration and two hours after the administration of rFVIIa
at the time of administration and two hours after the administration of rFVIIa
Adverse Events (AEs)
Time Frame: during treatment episodes
during treatment episodes
Serious Adverse Events (SAEs)
Time Frame: during treatment episodes
during treatment episodes

Collaborators and Investigators

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

Sponsor

Novo Nordisk A/S

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

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

January 1, 2004

Primary Completion (Actual)

October 1, 2011

Study Completion (Actual)

October 1, 2011

Study Registration Dates

First Submitted

October 7, 2011

First Submitted That Met QC Criteria

November 17, 2011

First Posted (Estimate)

November 22, 2011

Study Record Updates

Last Update Posted (Estimate)

December 23, 2014

Last Update Submitted That Met QC Criteria

December 19, 2014

Last Verified

December 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • F7HAEM-3521
  • U1111-1122-5019 (Other Identifier: WHO)

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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