Immune ThrombocytopeniaNCT03469661
Differential Diagnostic of Immune ThrombocytoPenia (ITP) and Myelodysplastic Syndrome (MDS)
An observational study of Immune Thrombocytopenia and Myelodysplastic Syndromes, run by Fundacion CRIS de Investigación para Vencer el Cáncer at 6 sites in Spain.
Trial summary
Current diagnostic criteria for Immune ThrombocytoPenia (ITP) are mainly based on the presence of low numbers of platelets, excluding other multiple causes of thrombocytopenia, including immunodeficiencies, constitutional or acquired thrombocytopenia, hypersplenism and clonal hematological disorders such as MDS… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenia, Myelodysplastic Syndromes
- How many people
- 63 participants
- Where
- 6 sitesSpain
- Study dates
- 25 Apr 2018to 19 Apr 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: It is estimated that approximately 60 patients will be included in the study, 30 newly diagnosed ITP patients and 30 newly diagnosed MDS cases.
Who may be able to join
- Patients aged ≥ 18 years old at diagnosis
- Informed consent in writing
- Newly diagnosed primary ITP patients, or
- Newly diagnosed MDS patients
Who cannot join
- Patients who participated in a interventional thrombopoietin receptor agonists (TPO-RA) clinical trial since TPO-RA treatment initiation
- Patients with secondary immune thrombopenia
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
Spain6 sites
Complejo Hospitalario de A Coruña
A Coruña
Hospital de Burgos
Burgos
Hospital Universitario Insular de Gran Canaria
Las Palmas de Gran Canaria
Hospital Ramon y Cajal
Madrid
Hospital Regional de Málaga
Málaga
Hospital Virgen de la Victoria
Málaga
About this study
Official title: Differential Diagnostic of ITP and MDS: a Prospective Study by Next-Generation Flow Cytometry and Cytomorphological Approaches
Current diagnostic criteria for Immune ThrombocytoPenia (ITP) are mainly based on the presence of low numbers of platelets, excluding other multiple causes of thrombocytopenia, including immunodeficiencies, constitutional or acquired thrombocytopenia, hypersplenism and clonal hematological disorders such as MDS, disorders lymphoproliferative and acute myeloid leukemia (AML), among others. The analysis complementary tests for the diagnosis of ITP include studies basic systematic hematology, together with autoimmune assays and microbiological tests, while the evaluation of bone marrow is limited to elderly patients and/or patients resistant to treatment. Previous research has described the development of Myelodysplastic Syndrome (MDS) in patients with a previous diagnosis of ITP, and even the presence of MDS associated with genetic background. Therefore, it is conceivable fact that a percentage of cases with clinical signs of ITP in the moment of appearance may actually correspond to the first stages of MDS development in which bone marrow cells are not systematically evaluated in the initial presentation.
The anomalous immunophenotypic patterns between multiple compartments of bone marrow cells and peripherally blood (PB) platelets have been characterized through flow cytometry. The flow cytometry currently represents an important complementary tool for diagnosis of MDS that has shown great effectiveness and applicability in the differential diagnosis of non-clonal cytopenias against early MDS and for the detection of stages prior to MDS. Besides, the flow cytometry has made it possible to detect the presence of coexisting features related to MDS in patients with other malignancies hematologic conditions such as multiple myeloma, AML, and lymphocytic leukemia chronic. Therefore, the immunophenotypic analysis of the cells of the bone marrow of patients with ITP at the time of appearance would help to identify the cases that underlie clonal hematopoiesis MDS type. In the present study it is planned a broad characterization immunophenotyping of multiple compartments of bone marrow cells and PB platelets from patients with recently diagnosed ITP and investigate their morphological antecedents, in order to identify those patients who show compatible clonal hematopoietic patterns with MDS evident (or at risk of development), as candidates to receive most appropriate therapeutic methods.
- Conditions
- Immune Thrombocytopenia, Myelodysplastic Syndromes
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Immune Thrombocytopenia Diagnosis | Not described | |
| Myelodysplastic Syndrome Diagnosis | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Morphological profile
Bone marrow cell compartment profiles
Measured: Baseline
Morphological profile
Peripheral blood platelets profiles
Measured: Baseline
Immunological profile
Bone marrow cell compartment profiles
Measured: Baseline
Immunological profile
Peripheral blood platelets profiles
Measured: Baseline
Secondary outcomes (2)
Immunophenotypic abnormalities
Evaluation of abnormal immunophenotypic profiles
Measured: Baseline
Morphological abnormalities
Evaluation of abnormal morphological profiles.
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Mar 2018
- Study start25 Apr 2018
- Primary completion19 Apr 2021
- Study completion19 Apr 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immune ThrombocytopeniaStudies 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
- Prospective
- Enrolment
- 63 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fundacion CRIS de Investigación para Vencer el Cáncer
- Sponsor type
- University, hospital or other
- Collaborators
- Amgen
- Responsible party
- Sponsor
- Investigators
- Tomás González, Principal investigator, Hospital de Burgos
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03469661
- Sponsor's protocol ID
- FCR-PTI-2017-01
- Status last verified
- Aug 2021
- First submitted
- 2 Mar 2018
- Last updated
- 18 Aug 2021
- 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 18 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Differential Diagnostic of ITP and MDS: a Prospective Study by Next-Generation Flow Cytometry and Cytomorphological Approaches
- ClinicalTrials.gov ID
- NCT03469661
- Acronym / sponsor ID
- FCR-PTI-2017-01
- Conditions
- Immune Thrombocytopenia, Myelodysplastic Syndromes
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 63 participants (actual)
- Lead sponsor
- Fundacion CRIS de Investigación para Vencer el CáncerUniversity, hospital or other
- Collaborators
- Amgen
- Study start
- 25 Apr 2018 (actual)
- Primary completion
- 19 Apr 2021 (actual)
- Study completion
- 19 Apr 2021 (actual)
- First posted
- 19 Mar 2018 (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.
Current diagnostic criteria for Immune ThrombocytoPenia (ITP) are mainly based on the presence of low numbers of platelets, excluding other multiple causes of thrombocytopenia, including immunodeficiencies, constitutional or acquired thrombocytopenia, hypersplenism and clonal hematological disorders such as MDS, disorders lymphoproliferative and acute myeloid leukemia (AML), among others. The analysis complementary tests for the diagnosis of ITP include studies basic systematic hematology, together with autoimmune assays and microbiological tests, while the evaluation of bone marrow is limited to elderly patients and/or patients resistant to treatment. Previous research has described the development of Myelodysplastic Syndrome (MDS) in patients with a previous diagnosis of ITP, and even the presence of MDS associated with genetic background. Therefore, it is conceivable fact that a percentage of cases with clinical signs of ITP in the moment of appearance may actually correspond to the first stages of MDS development in which bone marrow cells are not systematically evaluated in the initial presentation.
The anomalous immunophenotypic patterns between multiple compartments of bone marrow cells and peripherally blood (PB) platelets have been characterized through flow cytometry. The flow cytometry currently represents an important complementary tool for diagnosis of MDS that has shown great effectiveness and applicability in the differential diagnosis of non-clonal cytopenias against early MDS and for the detection of stages prior to MDS. Besides, the flow cytometry has made it possible to detect the presence of coexisting features related to MDS in patients with other malignancies hematologic conditions such as multiple myeloma, AML, and lymphocytic leukemia chronic. Therefore, the immunophenotypic analysis of the cells of the bone marrow of patients with ITP at the time of appearance would help to identify the cases that underlie clonal hematopoiesis MDS type. In the present study it is planned a broad characterization immunophenotyping of multiple compartments of bone marrow cells and PB platelets from patients with recently diagnosed ITP and investigate their morphological antecedents, in order to identify those patients who show compatible clonal hematopoietic patterns with MDS evident (or at risk of development), as candidates to receive most appropriate therapeutic methods.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Immune Thrombocytopenia Diagnosis | – | – |
| Myelodysplastic Syndrome Diagnosis | – | – |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- It is estimated that approximately 60 patients will be included in the study, 30 newly diagnosed ITP patients and 30 newly diagnosed MDS cases.
Key inclusion criteria (4 listed)
- Patients aged ≥ 18 years old at diagnosis
- Informed consent in writing
- Newly diagnosed primary ITP patients, or
- Newly diagnosed MDS patients
Key exclusion criteria (2 listed)
- Patients who participated in a interventional thrombopoietin receptor agonists (TPO-RA) clinical trial since TPO-RA treatment initiation
- Patients with secondary immune thrombopenia
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
Morphological profile
Time frame: Baseline
Bone marrow cell compartment profiles
Morphological profile
Time frame: Baseline
Peripheral blood platelets profiles
Immunological profile
Time frame: Baseline
Bone marrow cell compartment profiles
Immunological profile
Time frame: Baseline
Peripheral blood platelets profiles
2 secondary outcomes
Immunophenotypic abnormalities
Time frame: Baseline
Morphological abnormalities
Time frame: Baseline
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Aug 2021.
Related trials for Immune Thrombocytopenia
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RecruitingPhase 1/2
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RecruitingPhase 3
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RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
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RecruitingPhase 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
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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. This study is not recruiting at the moment.
Tomás González principal investigator
Hospital de Burgos
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Immune Thrombocytopenia and Myelodysplastic Syndromes, run by Fundacion CRIS de Investigación para Vencer el Cáncer at 6 sites in Spain.
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 4 inclusion and 2 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: finished, as of the sponsor's last update on 18 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Morphological profile (time frame: Baseline). The study lists 4 primary outcomes in all. 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 18 Aug 2021.