Observational Registry of the Treatment of Glanzmann's Thrombasthenia

NCT ID: NCT01476423

Last Updated: 2014-12-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

218 participants

Study Classification

OBSERVATIONAL

Study Start Date

2004-01-31

Study Completion Date

2011-10-31

Brief Summary

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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.

Detailed Description

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Conditions

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Congenital Bleeding Disorder Glanzmann's Disease

Keywords

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Glanzmann's Thrombasthenia

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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A

activated recombinant human factor VII

Intervention Type 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.

Interventions

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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.

Intervention Type DRUG

Eligibility Criteria

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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
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novo Nordisk A/S

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Global Clinical Registry (GCR, 1452)

Role: STUDY_DIRECTOR

Novo Nordisk A/S

Locations

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Novo Nordisk Clinical Trial Call Center

Princeton, New Jersey, United States

Site Status

Algiers, , Algeria

Site Status

Vienna, , Austria

Site Status

Brussels, , Belgium

Site Status

Sofia, , Bulgaria

Site Status

Paris La Défense Cedex, , France

Site Status

Mainz, , Germany

Site Status

Budapest, , Hungary

Site Status

Rome, , Italy

Site Status

Alphen aan den Rijn, , Netherlands

Site Status

Karachi, , Pakistan

Site Status

Madrid, , Spain

Site Status

Malmo, , Sweden

Site Status

Zurich, , Switzerland

Site Status

Crawley, , United Kingdom

Site Status

Countries

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

References

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Poon MC, D'Oiron R, Baby S, Zotz RB, Di Minno G. The Glanzmann Thrombasthenia Registry: safety of platelet therapy in patients with Glanzmann thrombasthenia and changes in alloimmunization status. Haematologica. 2023 Oct 1;108(10):2855-2858. doi: 10.3324/haematol.2022.281973. No abstract available.

Reference Type DERIVED
PMID: 36924249 (View on PubMed)

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.

Reference Type DERIVED
PMID: 31523745 (View on PubMed)

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.

Reference Type DERIVED
PMID: 26001793 (View on PubMed)

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.

Reference Type DERIVED
PMID: 26001792 (View on PubMed)

Related Links

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http://novonordisk-trials.com

Clinical Trials at Novo Nordisk

Other Identifiers

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U1111-1122-5019

Identifier Type: OTHER

Identifier Source: secondary_id

F7HAEM-3521

Identifier Type: -

Identifier Source: org_study_id