Observational Registry of the Treatment of Glanzmann's Thrombasthenia

Congenital Bleeding Disorder Clinical Trial

Official title:

Treatment of Glanzmann's Thrombasthenia: A Prospective Observational Registry

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01476423
• Other study ID #: F7HAEM-3521
• Secondary ID: U1111-1122-5019
• Status: Completed
• Phase: N/A
• First received: October 7, 2011
• Last updated: December 19, 2014
• Start date: January 2004
• Est. completion date: October 2011

Study information

• Verified date: December 2014
• Source: Novo Nordisk A/S
• Contact: n/a
• Is FDA regulated: No
• Health authority: Algeria: Ministry of HealthAustria: Not required for observational study in AustriaBelgium: Not required for observational study in BelgiumBulgaria: Ministry of HealthFrance: Not required for observational studyGermany: Not required for observational studyHungary: Not required for observational studyItaly: Not required for observational studyNetherlands: Dutch Health Care InspectoratePakistan: Not required for observational studySpain: Ministry of HealthSweden: Not required for observational studySwitzerland: SwissmedicUnited Kingdom: Not required for observational studyUnited States: Food and Drug Administration
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 218
• Est. completion date: October 2011
• Est. primary completion date: October 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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 Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• Blood Coagulation Disorders
• Congenital Bleeding Disorder
• Glanzmann's Disease
• Hemostatic Disorders

Intervention

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.

Locations

Country	Name	City	State
United States	Novo Nordisk Clinical Trial Call Center	Princeton	New Jersey

Sponsors (1)

Lead Sponsor	Collaborator
Novo Nordisk A/S

Countries where clinical trial is conducted

United States,  Algeria,  Austria,  Belgium,  Bulgaria,  France,  Germany,  Hungary,  Italy,  Netherlands,  Pakistan,  Spain,  Sweden,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	For bleeding episodes: Overall efficacy evaluated by the caregiver/patient		within 30 days of end of treatment	No
Primary	For surgery including invasive and dental procedures: Haemoglobin level		prior to surgery and 24 hours after surgery	No
Primary	For surgery including invasive and dental procedures: Overall haemostatic evaluation by the surgeon		24 hours after surgery	No
Secondary	Changes in laboratory parameters (prothrombin time, platelet count, fibrinogen), if available		at the time of administration and two hours after the administration of rFVIIa	No
Secondary	Adverse Events (AEs)		during treatment episodes	No
Secondary	Serious Adverse Events (SAEs)		during treatment episodes	No

External Links

•   Clinical Trials at Novo Nordisk