Clinical trial NCT01476423: Observational Registry of the Treatment of Glanzmann's Thrombasthenia
Official title: Treatment of Glanzmann's Thrombasthenia: A Prospective Observational Registry
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Congenital Bleeding Disorder
Glanzmann's Disease
Keywords: Glanzmann's Thrombasthenia
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2011-11-22
Enrollment: 218
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.


Eligibility 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

Primary outcomes:
measure: For bleeding episodes: Overall efficacy evaluated by the caregiver/patient
timeFrame: within 30 days of end of treatment
measure: For surgery including invasive and dental procedures: Haemoglobin level
timeFrame: prior to surgery and 24 hours after surgery
measure: For surgery including invasive and dental procedures: Overall haemostatic evaluation by the surgeon
timeFrame: 24 hours after surgery
Secondary outcomes:
measure: Changes in laboratory parameters (prothrombin time, platelet count, fibrinogen), if available
timeFrame: at the time of administration and two hours after the administration of rFVIIa
measure: Adverse Events (AEs)
timeFrame: during treatment episodes
measure: Serious Adverse Events (SAEs)
timeFrame: during treatment episodes
Other outcomes:
Locations:
city: Princeton
country: United States
facility: Novo Nordisk Clinical Trial Call Center
state: New Jersey
city: Algiers
country: Algeria
city: Vienna
country: Austria
city: Brussels
country: Belgium
city: Sofia
country: Bulgaria
city: Paris La défense cedex
country: France
city: Mainz
country: Germany
city: Budapest
country: Hungary
city: Rome
country: Italy
city: Alphen a/d Rijn
country: Netherlands
city: Karachi
country: Pakistan
city: Madrid
country: Spain
city: Malmö
country: Sweden
city: Zurich
country: Switzerland
city: Crawley
country: United Kingdom
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References:
citation: 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.
citation: 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.
citation: 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.