ThrombocytopeniaPhase 1/2NCT03838354
A Clinical Trial of Chidamide in the Management of Refractory ITP
A Phase 1/2 study testing Chidamide in people with Thrombocytopenia, run by Shandong University at 1 site in China.
Trial summary
Primary immune thrombocytopenia (ITP) is an autoimmune bleeding disorder. Increased macrophage phagocytosis of antibody-coated platelet as well as decreased number and/or impaired function of CD4+CD25+Foxp3+ regulatory T (Treg) cells have been shown to participate in the pathogenesis of ITP. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thrombocytopenia
- What is being tested?
- Chidamide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 61 participants
- Where
- 1 siteChina
- Study dates
- 1 Jun 2021to 1 Sep 2023
- Phase
- Phase 1/2
- 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
Who may be able to join
- Participant must be at least 18 years of age at the time of the screening.
- Participant may be male or female.
- Participant has a confirmed diagnosis of ITP according to the 2019 International Working Group assessment for more than 6 months at screening.
- Participant who didn't respond or relapsed after previous first-line treatment, and lack of response to rituximab, TPO agents, or splenectomy.
- Bone marrow biopsy is performed in participants over 60 years to exclude hematological malignancies.
Who cannot join
- Participant has evidence of a secondary cause of immune thrombocytopenia or to drug treatments or participant has a multiple immune cytopenia, e.g. Evan's syndrome.
- Participant with the following conditions:severe dysfunction of the heart, kidney, liver, or lung; severe immunodeficiency; malignancy; HIV; hepatitis B or C virus infection; pregnancy or lactation.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Prospective, Multicenter Clinical Trial of Chidamide in the Management of Refractory Immune Thrombocytopenia
Primary immune thrombocytopenia (ITP) is an autoimmune bleeding disorder. Increased macrophage phagocytosis of antibody-coated platelet as well as decreased number and/or impaired function of CD4+CD25+Foxp3+ regulatory T (Treg) cells have been shown to participate in the pathogenesis of ITP. Our preclinical data revealed that chidamide, a histone deacetylase inhibitor (HDACi), could attenuate macrophage phagocytosis of antibody-coated platelets, stimulate production of natural Foxp3+ Tregs, and upregulate CTLA4 expression through modulation of histone H3K27 acetylation. The project was undertaking by Qilu Hospital of Shandong University in China. In order to evaluate the efficacy and safety of chidamide at two different dosage regimens in adult patients with refractory ITP.
Read the detailed description
In this prospective, open-label, multicenter, randomized clinical trial, refractory ITP adult patients will be enrolled from five medical centers in China. Eligible participants will be randomly assigned 1:1 to receive chidamide at either 2.5 or 5 mg twice per week. The primary endpoint is the overall response at week 12. Complete response was defined as a platelet count at or above 100×10^9/L and an absence of bleeding. Partial response was defined as a platelet count at or above 30×10^9/L but less than 100×10^9/L and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30×10^9 cells per L, or less than two-times increase from baseline platelet count, or bleeding. The secondary enpoints included 6-month sustained response, time to response (TTR), duration of response, bleeding scores, health-related quality of life assessment and adverse events (AEs). This study will compare the efficacy and safety of chidamide in two different dosage regimens in adult patients with refractory.
- Conditions
- Thrombocytopenia
- Being tested
- Chidamide
- Keywords
- ITP, Chidamide
- Medical subject headings
- Thrombocytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ChidamideDrug
Also called HBI-8000
In the 2.5 mg group, chidamide will be administered orally at an initial dose of 2.5 mg twice per week for 12 weeks. If an initial response was achieved by week 12, the allocated treatment could continue. Patients in 2.5 mg group were allowed to increase dose to 5 mg if platelet counts were less than 30×10^9 cells per L or less than two-times increase from baseline platelet count at week 12 according to investigators' advice and the patients' decision.
ChidamideDrug
Also called HBI-8000
In the 5 mg group, chidamide will be administered orally at an initial dose of 5 mg twice per week for 12 weeks. If an initial response was achieved by week 12, the allocated treatment could continue. Patients who did not respond to chidamide at 5 mg for 12 weeks would stop the allocated treatment and were continuously followed up.
| Study group | Role | What they receive |
|---|---|---|
| A | Experimental | Chidamide 2.5 mg po twice per week Receives: Drug: Chidamide |
| B | Experimental | Chidamide 5 mg po twice per week Receives: Drug: Chidamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response at week 12
The primary endpoint was the overall response at week 12.Complete response was defined as a platelet count at or above 100×10^9/L and an absence of bleeding. Partial response was defined as a platelet count at or above 30×109/L but less than 100×10^9/L and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30×10^9 cells per L, or less than two-times increase from baseline platelet count, or bleeding.
Measured: week 12
Secondary outcomes (6)
Therapy associated adverse events
The number and frequency of therapy associated adverse events
Measured: up to 1 year per subject
Sustained response
A response lasting for at least 6 months was defined as a sustained response.
Measured: 6 months
Time to response
Time to response was defined as the time from treatment initiation to achieve a complete response or a partial response, whichever came first, assessed up to week 12.
Measured: 12 weeks
Duration of response
Duration of response was defined as the time from achievement of a complete response or a partial response to the loss of response.
Measured: 12 months
Bleeding scores
Bleeding symptoms were graded according a standardized bleeding scale specific to primary immune thrombocytopenia on the basis of site and severity of bleeding by Khellaf et al (PMID: 15951296). A modification was made to exclude age from the original scale so that only bleeding symptoms were described. At each visit, we recorded bleeding scores. Routine visits were scheduled once a week for the first 4 weeks and once a month thereafter. Scores ranged from 0 to 59, with higher values indicating higher bleeding risk.
Measured: 12 months
Health-related quality of life assessment
Health-related quality of life was assessed using a self-administered immune thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) at baseline and at week 12. Scores ranged from 0 to 100, with higher values indicating better quality of life.
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Feb 2019
- Study start1 Jun 2021
- Primary completion1 Sep 2022
- Study completion1 Sep 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 61 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shandong University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ming Hou, Shandong University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03838354
- Sponsor's protocol ID
- ITP-Chidamide
- Status last verified
- Aug 2025
- First submitted
- 27 Nov 2018
- Last updated
- 26 Aug 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Multicenter Clinical Trial of Chidamide in the Management of Refractory Immune Thrombocytopenia
- ClinicalTrials.gov ID
- NCT03838354
- Acronym / sponsor ID
- ITP-Chidamide
- Conditions
- Thrombocytopenia
- Keywords
- ITP, Chidamide
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 61 participants (actual)
- Lead sponsor
- Shandong UniversityUniversity, hospital or other
- Study start
- 1 Jun 2021 (actual)
- Primary completion
- 1 Sep 2022 (actual)
- Study completion
- 1 Sep 2023 (actual)
- First posted
- 12 Feb 2019 (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.
Primary immune thrombocytopenia (ITP) is an autoimmune bleeding disorder. Increased macrophage phagocytosis of antibody-coated platelet as well as decreased number and/or impaired function of CD4+CD25+Foxp3+ regulatory T (Treg) cells have been shown to participate in the pathogenesis of ITP. Our preclinical data revealed that chidamide, a histone deacetylase inhibitor (HDACi), could attenuate macrophage phagocytosis of antibody-coated platelets, stimulate production of natural Foxp3+ Tregs, and upregulate CTLA4 expression through modulation of histone H3K27 acetylation. The project was undertaking by Qilu Hospital of Shandong University in China. In order to evaluate the efficacy and safety of chidamide at two different dosage regimens in adult patients with refractory ITP.
Detailed description
In this prospective, open-label, multicenter, randomized clinical trial, refractory ITP adult patients will be enrolled from five medical centers in China. Eligible participants will be randomly assigned 1:1 to receive chidamide at either 2.5 or 5 mg twice per week. The primary endpoint is the overall response at week 12. Complete response was defined as a platelet count at or above 100×10^9/L and an absence of bleeding. Partial response was defined as a platelet count at or above 30×10^9/L but less than 100×10^9/L and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30×10^9 cells per L, or less than two-times increase from baseline platelet count, or bleeding. The secondary enpoints included 6-month sustained response, time to response (TTR), duration of response, bleeding scores, health-related quality of life assessment and adverse events (AEs). This study will compare the efficacy and safety of chidamide in two different dosage regimens in adult patients with refractory.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| A | Experimental | Drug: ChidamideChidamide 2.5 mg po twice per week |
| B | Experimental | Drug: ChidamideChidamide 5 mg po twice per week |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Participant must be at least 18 years of age at the time of the screening.
- Participant may be male or female.
- Participant has a confirmed diagnosis of ITP according to the 2019 International Working Group assessment for more than 6 months at screening.
- Participant who didn't respond or relapsed after previous first-line treatment, and lack of response to rituximab, TPO agents, or splenectomy.
- Bone marrow biopsy is performed in participants over 60 years to exclude hematological malignancies.
Key exclusion criteria (2 listed)
- Participant has evidence of a secondary cause of immune thrombocytopenia or to drug treatments or participant has a multiple immune cytopenia, e.g. Evan's syndrome.
- Participant with the following conditions:severe dysfunction of the heart, kidney, liver, or lung; severe immunodeficiency; malignancy; HIV; hepatitis B or C virus infection; pregnancy or lactation.
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 |
|---|---|---|
| ChidamideAlso: HBI-8000In the 2.5 mg group, chidamide will be administered orally at an initial dose of 2.5 mg twice per week for 12 weeks. If an initial response was achieved by week 12, the allocated treatment could continue. Patients in 2.5 mg group were allowed to increase dose to 5 mg if platelet counts were less than 30×10^9 cells per L or less than two-times increase from baseline platelet count at week 12 according to investigators' advice and the patients' decision. | Drug | A |
| ChidamideAlso: HBI-8000In the 5 mg group, chidamide will be administered orally at an initial dose of 5 mg twice per week for 12 weeks. If an initial response was achieved by week 12, the allocated treatment could continue. Patients who did not respond to chidamide at 5 mg for 12 weeks would stop the allocated treatment and were continuously followed up. | Drug | B |
Primary outcome
Overall response at week 12
Time frame: week 12
The primary endpoint was the overall response at week 12.Complete response was defined as a platelet count at or above 100×10^9/L and an absence of bleeding. Partial response was defined as a platelet count at or above 30×109/L but less than 100×10^9/L and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30×10^9 cells per L, or less than two-times increase from baseline platelet count, or bleeding.
6 secondary outcomes
Therapy associated adverse events
Time frame: up to 1 year per subject
Sustained response
Time frame: 6 months
Time to response
Time frame: 12 weeks
Duration of response
Time frame: 12 months
Bleeding scores
Time frame: 12 months
Health-related quality of life assessment
Time frame: 12 weeks
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 26 Aug 2025.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Chidamide in people with Thrombocytopenia, run by Shandong University 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 5 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 26 Aug 2025. 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Overall response at week 12 (time frame: week 12). 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 26 Aug 2025.