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

October 7, 2011
April 29, 2014
May 2007
October 2011   (final data collection date for primary outcome measure)
  • 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 ]
Same as current
Complete list of historical versions of study NCT01476423 on ClinicalTrials.gov Archive Site
  • 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 ]
Same as current
Not Provided
Not Provided
 
Observational Registry of the Treatment of Glanzmann's Thrombasthenia
Treatment of Glanzmann's Thrombasthenia: A Prospective Observational Registry

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.

Not Provided
Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
Not Provided
Non-Probability Sample

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.

  • Congenital Bleeding Disorder
  • Glanzmann's Disease
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.
A
Intervention: Drug: activated recombinant human factor VII
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
218
October 2011
October 2011   (final data collection date for primary outcome measure)

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
Both
Not Provided
No
Contact information is only displayed when the study is recruiting subjects
United States,   Algeria,   Austria,   Belgium,   Bulgaria,   France,   Germany,   Hungary,   Italy,   Netherlands,   Pakistan,   Spain,   Sweden,   Switzerland,   United Kingdom
 
NCT01476423
F7HAEM-3521, U1111-1122-5019
No
Novo Nordisk A/S
Novo Nordisk A/S
Not Provided
Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
Novo Nordisk A/S
April 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP