Observational Registry of the Treatment of Glanzmann's Thrombasthenia
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| ClinicalTrials.gov Identifier: NCT01476423 |
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Recruitment Status :
Completed
First Posted : November 22, 2011
Last Update Posted : December 23, 2014
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Sponsor:
Novo Nordisk A/S
Information provided by (Responsible Party):
Novo Nordisk A/S
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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 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 |
Resource links provided by the National Library of Medicine
Genetics Home Reference related topics:
Glanzmann thrombasthenia
| 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. |
Primary Outcome Measures :
- For bleeding episodes: Overall efficacy evaluated by the caregiver/patient [ Time Frame: within 30 days of end of treatment ]
- For surgery including invasive and dental procedures: Haemoglobin level [ Time Frame: prior to surgery and 24 hours after surgery ]
- For surgery including invasive and dental procedures: Overall haemostatic evaluation by the surgeon [ Time Frame: 24 hours after surgery ]
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 ]
- Adverse Events (AEs) [ Time Frame: during treatment episodes ]
- Serious Adverse Events (SAEs) [ Time Frame: during treatment episodes ]
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| 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
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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 |
Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Novo Nordisk A/S |
| ClinicalTrials.gov Identifier: | NCT01476423 History of Changes |
| Other Study ID Numbers: |
F7HAEM-3521 U1111-1122-5019 ( Other Identifier: WHO ) |
| First Posted: | November 22, 2011 Key Record Dates |
| Last Update Posted: | December 23, 2014 |
| Last Verified: | December 2014 |
Keywords provided by Novo Nordisk A/S:
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Glanzmann's Thrombasthenia |
Additional relevant MeSH terms:
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Blood Coagulation Disorders Hemostatic Disorders Thrombasthenia Hematologic Diseases Vascular Diseases |
Cardiovascular Diseases Hemorrhagic Disorders Blood Coagulation Disorders, Inherited Blood Platelet Disorders Genetic Diseases, Inborn |