Glanzmann ThrombastheniaGlanzmann-NHSNCT06204042
Multinational Glanzmann Study
An observational study of Glanzmann Thrombasthenia, run by UMC Utrecht.
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. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Glanzmann Thrombasthenia
- How many people
- 200 participants
- Where
- No sites listed yet
- Study dates
- 1 Mar 2024to 1 Mar 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Adult patients with biochemically or genetically diagnosed Glanzmann thrombasthenia.
Who may be able to join
- Adult patients (≥16 years);
- Biochemically or genetically diagnosed Glanzmann thrombasthenia.
- Willing and able to give written informed consent.
Who cannot join
- Patients with acquired thrombasthenic states caused by auto-immune disorders or drugs.
About this study
Official title: 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.
- Conditions
- Glanzmann Thrombasthenia
- Medical subject headings
- Thrombasthenia
What is being measured
The primary outcome is the main question the study is designed to answer.
Genetic analysis for Glanzmann thrombasthenia
Description of mutation analysis in the ITGA2B and ITGB3 genes.
Measured: Single measurement at Baseline
Secondary outcomes (2)
Incidence of anti-Human Leucocyte Antigen (HLA) antibodies
Cross-sectional evaluation of existing antibodies against Human Leucocyte Antigen (HLA) type I.
Measured: Single measurement at Baseline
Incidence of anti-Human Platelet Antigen (HPA) antibodies
Cross-sectional evaluation of existing antibodies against Human Platelet Antigen (HPA)
Measured: Single measurement at Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jan 2024
- Study start1 Mar 2024 (estimated)
- Primary completion1 Mar 2028 (estimated)
- Study completion1 Mar 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Glanzmann ThrombastheniaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- UMC Utrecht
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Roger Schutgens, UMC Utrecht
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06204042
- Sponsor's protocol ID
- NL85068.041.24
- Acronym
- Glanzmann-NHS
- Status last verified
- Feb 2024
- First submitted
- 5 Dec 2023
- Last updated
- 28 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Glanzmann Thrombasthenia Natural History Study+
- ClinicalTrials.gov ID
- NCT06204042
- Acronym / sponsor ID
- Glanzmann-NHS / NL85068.041.24
- Conditions
- Glanzmann Thrombasthenia
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- UMC UtrechtUniversity, hospital or other
- Study start
- 1 Mar 2024 (estimated)
- Primary completion
- 1 Mar 2028 (estimated)
- Study completion
- 1 Mar 2029 (estimated)
- First posted
- 12 Jan 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Adult patients with biochemically or genetically diagnosed Glanzmann thrombasthenia.
Key inclusion criteria (3 listed)
- Adult patients (≥16 years);
- Biochemically or genetically diagnosed Glanzmann thrombasthenia.
- Willing and able to give written informed consent.
Key exclusion criteria (1 listed)
- Patients with acquired thrombasthenic states caused by auto-immune disorders or drugs.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Genetic analysis for Glanzmann thrombasthenia
Time frame: Single measurement at Baseline
Description of mutation analysis in the ITGA2B and ITGB3 genes.
2 secondary outcomes
Incidence of anti-Human Leucocyte Antigen (HLA) antibodies
Time frame: Single measurement at Baseline
Incidence of anti-Human Platelet Antigen (HPA) antibodies
Time frame: Single measurement at Baseline
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Feb 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
An observational study of Glanzmann Thrombasthenia, run by UMC Utrecht.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 28 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Genetic analysis for Glanzmann thrombasthenia (time frame: Single measurement at Baseline). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Feb 2024; AzTrials last refreshed its copy on 28 Oct 2026.