Observational Registry of the Treatment of Glanzmann's Thrombasthenia - Full Text View

Purpose

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.

Condition	Intervention
Congenital Bleeding Disorder Glanzmann's Disease	Drug: activated recombinant human factor VII


Study Type:	Observational
Study Design:	Observational Model: Cohort Time Perspective: Prospective
Official Title:	Treatment of Glanzmann's Thrombasthenia: A Prospective Observational Registry

Resource links provided by NLM:

Genetics Home Reference related topics: Glanzmann thrombasthenia

MedlinePlus related topics: Bleeding

Genetic and Rare Diseases Information Center resources: Glanzmann Thrombasthenia Thrombasthenia

U.S. FDA Resources

Further study details as provided by Novo Nordisk A/S:

Primary Outcome Measures:

For bleeding episodes: Overall efficacy evaluated by the caregiver/patient [ Time Frame: within 30 days of end of treatment ] [ Designated as safety issue: No ]
For surgery including invasive and dental procedures: Haemoglobin level [ Time Frame: prior to surgery and 24 hours after surgery ] [ Designated as safety issue: No ]
For surgery including invasive and dental procedures: Overall haemostatic evaluation by the surgeon [ Time Frame: 24 hours after surgery ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

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 ] [ Designated as safety issue: No ]
Adverse Events (AEs) [ Time Frame: during treatment episodes ] [ Designated as safety issue: No ]
Serious Adverse Events (SAEs) [ Time Frame: during treatment episodes ] [ Designated as safety issue: No ]


Enrollment:	218
Study Start Date:	January 2004
Study Completion Date:	October 2011
Primary Completion Date:	October 2011 (Final data collection date for primary outcome measure)

Groups/Cohorts	Assigned Interventions
A	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.

Groups/Cohorts

Assigned Interventions

A

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.

Eligibility


Ages Eligible for Study:	Child, Adult, Senior
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No
Sampling Method:	Non-Probability Sample

Study Population

Patients with GT. There is no limit to the number of bleeding episodes or preventions of bleeding during invasive procedures/surgery that can be entered for each patient within the lifetime of the registry. Patients will receive standard care according to local practice, thus any systemic haemostatic treatment (with or without antifibrinolytic drugs or other agents) considered useful by the centres for treatment of GT, are included in the registry.

Criteria

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

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01476423

Locations


United States, New Jersey
Novo Nordisk Clinical Trial Call Center
Princeton, New Jersey, United States, 08540
Algeria

Algiers, Algeria, 16035
Austria

Vienna, Austria, A-1010
Belgium

Brussels, Belgium, 1070
Bulgaria

Sofia, Bulgaria, 1407
France

Paris La défense cedex, France, 92932
Germany

Mainz, Germany, 55127
Hungary

Budapest, Hungary, 1025
Italy

Rome, Italy, 00144
Netherlands

Alphen a/d Rijn, Netherlands
Pakistan

Karachi, Pakistan
Spain

Madrid, Spain, 28033
Sweden

Malmö, Sweden, 202 15
Switzerland

Zurich, Switzerland, CH-8050
United Kingdom

Crawley, United Kingdom, RH11 9RT

Sponsors and Collaborators

Novo Nordisk A/S

Investigators


Study Director:	Global Clinical Registry (GCR, 1452)	Novo Nordisk A/S

More Information

Additional Information:

Clinical Trials at Novo Nordisk This link exits the ClinicalTrials.gov site

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Di Minno G, Zotz RB, d'Oiron R, Bindslev N, Di Minno MN, Poon MC; Glanzmann Thrombasthenia Registry Investigators. The international, prospective Glanzmann Thrombasthenia Registry: treatment modalities and outcomes of non-surgical bleeding episodes in patients with Glanzmann thrombasthenia. Haematologica. 2015 Aug;100(8):1031-7. doi: 10.3324/haematol.2014.121475. Epub 2015 May 22.

Poon MC, d'Oiron R, Zotz RB, Bindslev N, Di Minno MN, Di Minno G; Glanzmann Thrombasthenia Registry Investigators. The international, prospective Glanzmann Thrombasthenia Registry: treatment and outcomes in surgical intervention. Haematologica. 2015 Aug;100(8):1038-44. doi: 10.3324/haematol.2014.121384. Epub 2015 May 22.


Responsible Party:	Novo Nordisk A/S
ClinicalTrials.gov Identifier:	NCT01476423 History of Changes
Other Study ID Numbers:	F7HAEM-3521 U1111-1122-5019
Study First Received:	October 7, 2011
Last Updated:	December 19, 2014
Health Authority:	Algeria: Ministry of Health Austria: Not required for observational study in Austria Belgium: Not required for observational study in Belgium Bulgaria: Ministry of Health France: Not required for observational study Germany: Not required for observational study Hungary: Not required for observational study Italy: Not required for observational study Netherlands: Dutch Health Care Inspectorate Pakistan: Not required for observational study Spain: Ministry of Health Sweden: Not required for observational study Switzerland: Swissmedic United Kingdom: Not required for observational study United States: Food and Drug Administration

Keywords provided by Novo Nordisk A/S:


Glanzmann's Thrombasthenia

Additional relevant MeSH terms:


Blood Coagulation Disorders Hemostatic Disorders Thrombasthenia Hematologic Diseases Vascular Diseases	Cardiovascular Diseases Hemorrhagic Disorders Blood Coagulation Disorders, Inherited Blood Platelet Disorders Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on September 27, 2016