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Trial record 1 of 7 for:    "Thrombasthenia"
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Observational Registry of the Treatment of Glanzmann's Thrombasthenia

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT01476423
First received: October 7, 2011
Last updated: April 29, 2014
Last verified: April 2014
  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:


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: May 2007
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.

  Eligibility

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:
No publications provided

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: April 29, 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
Blood Coagulation Disorders, Inherited
Blood Platelet Disorders
Cardiovascular Diseases
Genetic Diseases, Inborn
Hematologic Diseases
Hemorrhagic Disorders
Vascular Diseases

ClinicalTrials.gov processed this record on November 25, 2014