Observational Registry of the Treatment of Glanzmann's Thrombasthenia

• ClinicalTrials.gov Identifier: NCT01476423
• Recruitment Status: Completed
• First Posted: November 22, 2011
• Last Update Posted: December 23, 2014
• Sponsor: Novo Nordisk A/S
• Information provided by (Responsible Party): Novo Nordisk A/S

Study Description

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.

Condition or disease	Intervention/treatment
Congenital Bleeding Disorder; Glanzmann's Disease	Drug: activated recombinant human factor VII

Study Design

• Study Type: Observational
• Actual Enrollment: 218 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Treatment of Glanzmann's Thrombasthenia: A Prospective Observational Registry
• Study Start Date: January 2004
• Actual Primary Completion Date: October 2011
• Actual Study Completion Date: October 2011

Groups and Cohorts

Group/Cohort

Intervention/treatment

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.

Outcome Measures

Primary Outcome Measures :

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 Outcome Measures :

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 ]

Eligibility Criteria

• Ages Eligible for Study: Child, Adult, Older Adult
• Sexes Eligible for Study: All
• 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

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 

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

Zotz RB, Poon MC, Di Minno G, D'Oiron R; Glanzmann Thrombasthenia Registry Investigators . The International Prospective Glanzmann Thrombasthenia Registry: Pediatric Treatment and Outcomes. TH Open. 2019 Sep 12;3(3):e286-e294. doi: 10.1055/s-0039-1696657. eCollection 2019 Jul.

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
• Other Study ID Numbers: F7HAEM-3521; U1111-1122-5019 ( Other Identifier: WHO )
• First Posted: November 22, 2011
• Last Update Posted: December 23, 2014
• Last Verified: December 2014

Keywords provided by Novo Nordisk A/S:

Glanzmann's Thrombasthenia

Additional relevant MeSH terms:

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