Observational Registry of the Treatment of Glanzmann's Thrombasthenia

Completed

• Conditions: Congenital Bleeding Disorder; Glanzmann's Disease
• Interventions: Drug: activated recombinant human factor VII

• Registration Number: NCT01476423
• Lead Sponsor: Novo Nordisk A/S

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2004-01
• Primary Completion: 2011-10
• Study Completion: 2011-10
• Study First Submitted: 2011-10-07
• Study First Submitted QC: 2011-11-17
• Study First Posted: 2011-11-22
• Status Verified: 2014-12
• Last Update Submitted: 2014-12-19
• Last Update Posted: 2014-12-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 218

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

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Exclusion Criteria

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

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
A	activated recombinant human factor VII	-

Primary Outcome Measures

Name	Time	Method
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
For bleeding episodes: Overall efficacy evaluated by the caregiver/patient	within 30 days of end of treatment

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Novo Nordisk Clinical Trial Call Center; 🇺🇸 Princeton, New Jersey, United States