Multinational Glanzmann Study (Glanzmann-NHS)

February 26, 2024 updated by: Roger Schutgens, UMC Utrecht

Glanzmann Thrombasthenia Natural History Study+

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.

Study Overview

Status

Not yet recruiting

Conditions

Study Type

Observational

Enrollment (Estimated)

200

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Adult patients with biochemically or genetically diagnosed Glanzmann thrombasthenia.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Genetic analysis for Glanzmann thrombasthenia
Time Frame: Single measurement at Baseline
Description of mutation analysis in the ITGA2B and ITGB3 genes.
Single measurement at Baseline

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of anti-Human Leucocyte Antigen (HLA) antibodies
Time Frame: Single measurement at Baseline
Cross-sectional evaluation of existing antibodies against Human Leucocyte Antigen (HLA) type I.
Single measurement at Baseline
Incidence of anti-Human Platelet Antigen (HPA) antibodies
Time Frame: Single measurement at Baseline
Cross-sectional evaluation of existing antibodies against Human Platelet Antigen (HPA)
Single measurement at Baseline

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

UMC Utrecht

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

March 1, 2024

Primary Completion (Estimated)

March 1, 2028

Study Completion (Estimated)

March 1, 2029

Study Registration Dates

First Submitted

December 5, 2023

First Submitted That Met QC Criteria

January 2, 2024

First Posted (Actual)

January 12, 2024

Study Record Updates

Last Update Posted (Actual)

February 28, 2024

Last Update Submitted That Met QC Criteria

February 26, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NL85068.041.24

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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