Skip to main content
OpenTrials
Completed

NCT Number: NCT01476423

Observational Registry of the Treatment of Glanzmann's Thrombasthenia

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Congenital Bleeding Disorder Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Blood Platelet Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Glanzmann's Disease Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders Thrombasthenia

Sex eligibility

All sexes

Study type

Observational

Primary location

Algiers, Algeria

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

activated recombinant human factor VII

Drug

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.

Primary outcomes

  1. For bleeding episodes: Overall efficacy evaluated by the caregiver/patient

    Time frame: within 30 days of end of treatment

  2. For surgery including invasive and dental procedures: Haemoglobin level

    Time frame: prior to surgery and 24 hours after surgery

  3. For surgery including invasive and dental procedures: Overall haemostatic evaluation by the surgeon

    Time frame: 24 hours after surgery

Secondary outcomes

  1. 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

  2. Adverse Events (AEs)

    Time frame: during treatment episodes

  3. Serious Adverse Events (SAEs)

    Time frame: during treatment episodes

Sponsors and collaborators

Lead sponsor

Novo Nordisk A/S

Industry

Registry information

Official study title

Treatment of Glanzmann's Thrombasthenia: A Prospective Observational Registry

Important dates

Study start
2004
Primary completion
2011
Study completion
2011
First posted
Nov 22, 2011
Registry last updated
Dec 23, 2014

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.