Observational Registry of the Treatment of Glanzmann's Thrombasthenia

Study Overview
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Brief Summary
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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.

Official Title
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Treatment of Glanzmann's Thrombasthenia: A Prospective Observational Registry

Conditions
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Congenital Bleeding Disorder, Glanzmann's Disease

Intervention / Treatment
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* Drug: activated recombinant human factor VII


Participation Criteria
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Eligibility Criteria
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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
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| A<br> | Drug: activated recombinant human factor VII<br>* 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.<br>|

What is the study measuring?
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Primary Outcome Measures

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

Secondary Outcome Measures

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

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Glanzmann's Thrombasthenia