Observational Registry of the Treatment of Glanzmann's Thrombasthenia

NCT01476423 | Completed

• 2 other identifiers: F7HAEM-3521U1111-1122-5019
• Study Type: observational
• Target: 218
• Locations: 15 countries
• Sites: 15

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.

Conditions

Congenital Bleeding Disorder; Glanzmann's Disease

Keywords

Glanzmann's Thrombasthenia

Outcome Measures

Primary Outcomes (3)

• For bleeding episodes: Overall efficacy evaluated by the caregiver/patient

  within 30 days of end of treatment

• For surgery including invasive and dental procedures: Haemoglobin level

  prior to surgery and 24 hours after surgery

• For surgery including invasive and dental procedures: Overall haemostatic evaluation by the surgeon

  24 hours after surgery

Secondary Outcomes (3)

• Changes in laboratory parameters (prothrombin time, platelet count, fibrinogen), if available

  at the time of administration and two hours after the administration of rFVIIa

• Adverse Events (AEs)

  during treatment episodes

• Serious Adverse Events (SAEs)

  during treatment episodes

Study Arms (1)

A

Drug: activated recombinant human factor VII

Interventions

activated recombinant human factor VII 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.

A

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

• Patients with acquired thrombasthenic states caused by autoimmune disorders (acute or chronic) or drugs

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (15)

Novo Nordisk Clinical Trial Call Center

Princeton, New Jersey, 08540, United States

Location

Unknown Facility

Algiers, 16035, Algeria

Location

Unknown Facility

Vienna, A-1010, Austria

Location

Unknown Facility

Brussels, 1070, Belgium

Location

Unknown Facility

Sofia, 1407, Bulgaria

Location

Unknown Facility

Paris La Défense Cedex, 92932, France

Location

Unknown Facility

Mainz, 55127, Germany

Location

Unknown Facility

Budapest, 1025, Hungary

Location

Unknown Facility

Rome, 00144, Italy

Location

Unknown Facility

Alphen aan den Rijn, Netherlands

Location

Unknown Facility

Karachi, Pakistan

Location

Unknown Facility

Madrid, 28033, Spain

Location

Unknown Facility

Malmo, 202 15, Sweden

Location

Unknown Facility

Zurich, CH-8050, Switzerland

Location

Unknown Facility

Crawley, RH11 9RT, United Kingdom

Location

Related Publications (4)

• 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.

  PMID: 36924249 DERIVED

• 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.

  PMID: 31523745 DERIVED

• 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.

  PMID: 26001793 DERIVED

• 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.

  PMID: 26001792 DERIVED

Related Links

• Clinical Trials at Novo Nordisk

MeSH Terms

Conditions

Thrombasthenia

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Global Clinical Registry (GCR, 1452)

  Novo Nordisk A/S

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 7, 2011
• First Posted: November 22, 2011
• Study Start: January 1, 2004
• Primary Completion: October 1, 2011
• Study Completion: October 1, 2011
• Last Updated: December 23, 2014

Record last verified: 2014-12

Locations

SE (1); IT (1); US (1); PA (1); NL (1); BU (1); GB (1); HU (1); FR (1); ES (1); AL (1); BE (1); AU (1); CH (1); DE (1)