Primary Immune ThrombocytopeniaNCT06107582
Longitudinal Cohort of Pediatric Primary Immune Thrombocytopenia (ITP)
An observational study of Primary Immune Thrombocytopenia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
Immune thrombocytopenic purpura (ITP) is a kind of rare childhood disease that involve autoimmune destruction of platelets.The current Pediatric ITP cohorts are mostly based on single-center or multi-center cases, or cohorts with limited sample size in China. Read more about this study
- Who is it for?
- Ages 6 to 17 years
- Conditions the study is for
- Primary Immune Thrombocytopenia
- What is being tested?
- data collection
- How many people
- 500 participants
- Where
- 1 siteChina
- Study dates
- 1 Nov 2023to Dec 2036
- 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 6 to 17 years
- All sexes
- Child
Study population: Pediatric patients who were diagnosed with ITP in the investigating hospitals.
Who may be able to join
- Age 6-17 years old (including both ends), male and female;
- Diagnosis of ITP.
Who cannot join
- Secondary thrombocytopenia caused by various reasons, such as connective tissue disorders, bone marrow hematopoietic failure disease, myelodysplastic syndrome, malignancy, drugs, inherited thrombocytopenia, common variable immune deficiency, lymphoma, etc.;
- The expected follow-up period is less than 3 months.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Chinese Academy of Medical Science and Blood Disease Hospital
Tianjin, 300020
Ting Sun, MD, 02223909009, sunting@ihcams.ac.cn
About this study
Official title: Clinical Characteristics, Prognosis and Prognostic Model of Pediatric Immune Thrombocytopenia: a Prospective, Multicenter, Observational Cohort Study
Immune thrombocytopenic purpura (ITP) is a kind of rare childhood disease that involve autoimmune destruction of platelets.The current Pediatric ITP cohorts are mostly based on single-center or multi-center cases, or cohorts with limited sample size in China. There is a lack of comprehensive and large-scale prospective cohort studies in pediatric ITP. The purpose of this study is to analyze the clinical characteristics of Pediatric ITP, the treatment methods, prognosis and prognostic model of these patients in China.
Read the detailed description
Immune thrombocytopenia (ITP) is an organ-specific autoimmune disease, which is characterized by decreased platelet count and skin and mucosal bleeding. ITP is a kind of disease with increased platelet destruction and impaired platelet production caused by autoimmunity. Conventional treatment of adult ITP includes first-line glucocorticoid and immunoglobulin therapy, second line TPO and TPO receptor agonist, splenectomy and other immunosuppressive treatments (such as rituximab, vincristine, azathioprine, etc.). ITP is one of the most common hemorrhagic diseases. At present, the treatment response of ITP is not good, and a considerable number of patients need drug maintenance treatment, which seriously affects the quality of life of patients and increases the economic burden of patients. Longitudinal Cohort allows to describe the long-term clinical characteristics of pediatric ITP patients, to study the benefit-risk balance of treatments, including the growing development of targeted therapies and to analyze the prognostic factors and attempts to establish prognostic models.
The study will include pediatric patients diagnosed with primary immune thrombocytopenia in the investigating hospitals, and collect basic information, diagnostic and treatment information from medical records. The study will use questionnaire to measure the exposure of patients, and prospectively follow-up to collect the prognosis information.
- Conditions
- Primary Immune Thrombocytopenia
- Being tested
- data collection
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
data collectionOther
The study will collect basic information, diagnostic and treatment information from medical records and use questionnaire to measure the exposure of patients, and prospectively follow-up to collect the prognosis information.
| Study group | Role | What they receive |
|---|---|---|
| Pediatric Primary Immune Thrombocytopenia | Immune thrombocytopenia (ITP) : defined according to the international working group criteria Receives: Other: data collection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate
Overall response rate defined as proportion of subjects with a platelet count ≥ 30 × 10^9/L and at least 2-fold from baseline without bleeding
Measured: 3 years
Time to onset response
Time to onset response defined as the time needed for subjects to have a platelet count ≥ 30 × 10^9/L and at least 2-fold from baseline without bleeding
Measured: 3 years
Duration of response
Duration of response defined as the longest duration for which the subject sustained a platelet count ≥ 30 × 10^9/L and at least 2-fold from baseline without bleeding
Measured: 3 years
Sustained response rate
Sustained response rate defined as proportion of subjects who keep a platelet count ≥ 30 × 10^9/L and at least 2-fold from baseline without bleeding at 6, 12, 24, 36 months after initial administration of certain treatment in absence of rescue therapy
Measured: 3 years
Emergency treatment
Percentage of subjects who received emergency treatment after initial administration of certain treatment
Measured: 3 years
Number of subjects with clinically significant bleeding as assessed using the bleeding scale for pediatric patients with ITP after initial administration of certain treatment
Changes of the subjects' numbers in bleeding score after administration of certain treatment according to the reported bleeding scale for pediatric patients with ITP. The bleeding scale for pediatric patients with ITP is a measure of bleeding severity with the following grades: Grade 1 (minor) Minor bleeding, few petechiae (≤100 total) and/or ≤5 small bruises (≤3 cm in diameter), no mucosal bleeding；Grade 2 (mild) Mild bleeding, many petechiae (>100 total) and/or >5 large bruises (>3 cm in diameter), no mucosal bleeding；Grade 3 (moderate) Moderate bleeding, overt mucosal bleeding, troublesome lifestyle；Grade 4 (severe) Severe bleeding, mucosal bleeding leading to decrease in Hb>2 g/dL or suspected internal hemorrhage；
Measured: 3 years
Number of subjects with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale after initial administration of certain treatment
Changes of the subjects' numbers in WHO bleeding score after administration of certain treatment according to the reported World Health Organization's Bleeding Scale. The WHO Bleeding Scale is a measure of bleeding severity with the following grades: grade 0 = no bleeding, grade 1= petechiae, grade 2= mild blood loss, grade 3 = gross blood loss, and grade 4 = debilitating blood loss.
Measured: 3 years
Recurrence-free survival rate
Time from the start of treatment to the occurrence of a relapse or death event
Measured: 3 years
Secondary outcomes (7)
Incidence
The incidence of pediatric primary immune thrombocytopenia in China will be described
Measured: 3 years
Distribution
The population characteristics of pediatric primary immune thrombocytopenia in China will be described
Measured: 3 years
Prognosis related factors selected from transcriptome data
The prognosis related factors will be selected from transcriptome data and be used to establish prognosis prediction model
Measured: 3 years
Prognosis related factors selected from proteomics data
The prognosis related factors will be selected from proteomics data and be used to establish prognosis prediction model
Measured: 3 years
Prognosis related factors selected from metabolomics data
The prognosis related factors will be selected from metabolomics data and be used to establish prognosis prediction model
Measured: 3 years
Prognosis related factors selected from microbiome data
The prognosis related factors will be selected from proteomics data and be used to establish prognosis prediction model
Measured: 3 years
Incidence, severity, and relationship of treatment emergent adverse events after treatment
Incidence, severity, and relationship of treatment emergent adverse events after treatment will be analyzed
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Oct 2023
- Study start1 Nov 2023
- Primary completionDec 2026 (estimated)
- Study completionDec 2036 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe sponsor's other registered studies.
- 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
- 500 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Beijing Children's Hospital, Tianjin People's Hospital, Henan Cancer Hospital, Tianjin Medical University Second Hospital, The First Affiliated Hospital of Xiamen University, The Second Affiliated Hospital of Kunming Medical University
- Responsible party
- Sponsor
- Investigators
- Lei Zhang, MD, Principal investigator, Chinese Academy of Medical Science and Blood Disease Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06107582
- Sponsor's protocol ID
- IIT2023044
- Status last verified
- Dec 2025
- First submitted
- 26 Aug 2023
- Last updated
- 11 Feb 2026
- 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 11 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Characteristics, Prognosis and Prognostic Model of Pediatric Immune Thrombocytopenia: a Prospective, Multicenter, Observational Cohort Study
- ClinicalTrials.gov ID
- NCT06107582
- Acronym / sponsor ID
- IIT2023044
- Conditions
- Primary Immune Thrombocytopenia
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 500 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Collaborators
- Beijing Children's Hospital, Tianjin People's Hospital, Henan Cancer Hospital, Tianjin Medical University Second Hospital, The First Affiliated Hospital of Xiamen University, The Second Affiliated Hospital of Kunming Medical University
- Study start
- 1 Nov 2023 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2036 (estimated)
- First posted
- 30 Oct 2023 (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.
Immune thrombocytopenic purpura (ITP) is a kind of rare childhood disease that involve autoimmune destruction of platelets.The current Pediatric ITP cohorts are mostly based on single-center or multi-center cases, or cohorts with limited sample size in China. There is a lack of comprehensive and large-scale prospective cohort studies in pediatric ITP. The purpose of this study is to analyze the clinical characteristics of Pediatric ITP, the treatment methods, prognosis and prognostic model of these patients in China.
Detailed description
Immune thrombocytopenia (ITP) is an organ-specific autoimmune disease, which is characterized by decreased platelet count and skin and mucosal bleeding. ITP is a kind of disease with increased platelet destruction and impaired platelet production caused by autoimmunity. Conventional treatment of adult ITP includes first-line glucocorticoid and immunoglobulin therapy, second line TPO and TPO receptor agonist, splenectomy and other immunosuppressive treatments (such as rituximab, vincristine, azathioprine, etc.). ITP is one of the most common hemorrhagic diseases. At present, the treatment response of ITP is not good, and a considerable number of patients need drug maintenance treatment, which seriously affects the quality of life of patients and increases the economic burden of patients. Longitudinal Cohort allows to describe the long-term clinical characteristics of pediatric ITP patients, to study the benefit-risk balance of treatments, including the growing development of targeted therapies and to analyze the prognostic factors and attempts to establish prognostic models.
The study will include pediatric patients diagnosed with primary immune thrombocytopenia in the investigating hospitals, and collect basic information, diagnostic and treatment information from medical records. The study will use questionnaire to measure the exposure of patients, and prospectively follow-up to collect the prognosis information.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pediatric Primary Immune Thrombocytopenia | – | Other: data collectionImmune thrombocytopenia (ITP) : defined according to the international working group criteria |
Who may qualify
- Ages
- 6 to 17 years
- Sex
- All
- Age groups
- Child
- Study population
- Pediatric patients who were diagnosed with ITP in the investigating hospitals.
Key inclusion criteria (2 listed)
- Age 6-17 years old (including both ends), male and female;
- Diagnosis of ITP.
Key exclusion criteria (2 listed)
- Secondary thrombocytopenia caused by various reasons, such as connective tissue disorders, bone marrow hematopoietic failure disease, myelodysplastic syndrome, malignancy, drugs, inherited thrombocytopenia, common variable immune deficiency, lymphoma, etc.;
- The expected follow-up period is less than 3 months.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| data collectionThe study will collect basic information, diagnostic and treatment information from medical records and use questionnaire to measure the exposure of patients, and prospectively follow-up to collect the prognosis information. | Other | Pediatric Primary Immune Thrombocytopenia |
Primary outcome
Overall response rate
Time frame: 3 years
Overall response rate defined as proportion of subjects with a platelet count ≥ 30 × 10^9/L and at least 2-fold from baseline without bleeding
Time to onset response
Time frame: 3 years
Time to onset response defined as the time needed for subjects to have a platelet count ≥ 30 × 10^9/L and at least 2-fold from baseline without bleeding
Duration of response
Time frame: 3 years
Duration of response defined as the longest duration for which the subject sustained a platelet count ≥ 30 × 10^9/L and at least 2-fold from baseline without bleeding
Sustained response rate
Time frame: 3 years
Sustained response rate defined as proportion of subjects who keep a platelet count ≥ 30 × 10^9/L and at least 2-fold from baseline without bleeding at 6, 12, 24, 36 months after initial administration of certain treatment in absence of rescue therapy
Emergency treatment
Time frame: 3 years
Percentage of subjects who received emergency treatment after initial administration of certain treatment
Number of subjects with clinically significant bleeding as assessed using the bleeding scale for pediatric patients with ITP after initial administration of certain treatment
Time frame: 3 years
Changes of the subjects' numbers in bleeding score after administration of certain treatment according to the reported bleeding scale for pediatric patients with ITP. The bleeding scale for pediatric patients with ITP is a measure of bleeding severity with the following grades: Grade 1 (minor) Minor bleeding, few petechiae (≤100 total) and/or ≤5 small bruises (≤3 cm in diameter), no mucosal bleeding；Grade 2 (mild) Mild bleeding, many petechiae (>100 total) and/or >5 large bruises (>3 cm in diameter), no mucosal bleeding；Grade 3 (moderate) Moderate bleeding, overt mucosal bleeding, troublesome lifestyle；Grade 4 (severe) Severe bleeding, mucosal bleeding leading to decrease in Hb>2 g/dL or suspected internal hemorrhage；
Number of subjects with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale after initial administration of certain treatment
Time frame: 3 years
Changes of the subjects' numbers in WHO bleeding score after administration of certain treatment according to the reported World Health Organization's Bleeding Scale. The WHO Bleeding Scale is a measure of bleeding severity with the following grades: grade 0 = no bleeding, grade 1= petechiae, grade 2= mild blood loss, grade 3 = gross blood loss, and grade 4 = debilitating blood loss.
Recurrence-free survival rate
Time frame: 3 years
Time from the start of treatment to the occurrence of a relapse or death event
7 secondary outcomes
Incidence
Time frame: 3 years
Distribution
Time frame: 3 years
Prognosis related factors selected from transcriptome data
Time frame: 3 years
Prognosis related factors selected from proteomics data
Time frame: 3 years
Prognosis related factors selected from metabolomics data
Time frame: 3 years
Prognosis related factors selected from microbiome data
Time frame: 3 years
Incidence, severity, and relationship of treatment emergent adverse events after treatment
Time frame: 3 years
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 11 Feb 2026.
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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.
Lei Zhang, MD principal investigator
Chinese Academy of Medical Science and Blood Disease Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Primary Immune Thrombocytopenia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
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 2 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: looking for participants now, as of the sponsor's last update on 11 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. 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?
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?
Contact Ting Sun, MD (02223909009, sunting@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall response rate (time frame: 3 years). The study lists 8 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 11 Feb 2026.