Multinational Glanzmann Study

Glanzmann Thrombasthenia Clinical Trial

— Glanzmann-NHS  

Official title:

Glanzmann Thrombasthenia Natural History Study+

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06204042
• Other study ID #: NL85068.041.24
• Status: Not yet recruiting
• Start date: March 1, 2024
• Est. completion date: March 1, 2029

Study information

• Verified date: February 2024
• Source: UMC Utrecht
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

Glanzmann thrombasthenia is a rare autosomal recessive platelet disorder characterized by a lack of functional integrins alfaIIb or beta3 (glycoproteins IIb/IIIa). The prevalence is variously reported to be between 1:200,000 to 1:1,000,000, with substantial geographic variation. The clinical phenotype is dominated by an increased mucocutaneous bleeding tendency. In absence of a primary bleeding prophylaxis, the current treatment of Glanzmann thrombasthenia is mainly focused on prevention or management of bleeding. However, as potential new therapies emerge, clinicians require unbiased, long-term safety and efficacy data for both current treatment and new therapies. We have designed this study to investigate genetic phenotype (ITGA2B and ITGB3 genes) and the prevalence of antibodies against human leucocyte antigen (HLA) and human platelet antigen (HPA), the latter two being a potential consequence of the current golden standard treatment: platelet transfusion. The results of this study will be merged with a longitudinal registry with retrospective and prospective data collection of clinical phenotype, haemorrhagic burden and bleeding management. Analysis of the data from the Glanzmann-NHS+ study and the registry will help us to get a better understanding of the clinical variation among participants with Glanzmann thrombasthenia. The ultimate goal is to accelerate improvement in the care of patients with Glanzmann thrombasthenia.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 200
• Est. completion date: March 1, 2029
• Est. primary completion date: March 1, 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: 16 Years and older

Eligibility

Inclusion Criteria:

• Adult patients (=16 years);
• Biochemically or genetically diagnosed Glanzmann thrombasthenia.
• Willing and able to give written informed consent.

Exclusion Criteria:

• Patients with acquired thrombasthenic states caused by auto-immune disorders or drugs.

Related Conditions & MeSH terms

• Glanzmann Thrombasthenia

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
UMC Utrecht

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Genetic analysis for Glanzmann thrombasthenia	Description of mutation analysis in the ITGA2B and ITGB3 genes.	Single measurement at Baseline
Secondary	Incidence of anti-Human Leucocyte Antigen (HLA) antibodies	Cross-sectional evaluation of existing antibodies against Human Leucocyte Antigen (HLA) type I.	Single measurement at Baseline
Secondary	Incidence of anti-Human Platelet Antigen (HPA) antibodies	Cross-sectional evaluation of existing antibodies against Human Platelet Antigen (HPA)	Single measurement at Baseline