Observational Registry of the Treatment of Glanzmann's Thrombasthenia

Novo Nordisk

Status: Completed

Conditions: Glanzmann's Disease; Congenital Bleeding Disorder

Treatments: Drug: activated recombinant human factor VII

Study type: Observational

Funder types: Industry

Identifiers

NCT01476423

U1111-1122-5019 (Other Identifier)

F7HAEM-3521

Details and patient eligibility

About

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.

Enrollment: 218 patients

Sex: All

Volunteers: No Healthy Volunteers

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

Trial design

218 participants in 1 patient group

A

Treatment:

Drug: activated recombinant human factor VII