Myelodysplastic SyndromesPhase 2NCT07616336
Chidamide Monotherapy for Intermediate-to-High-Risk Myelodysplastic Syndromes
A Phase 2 study testing Chidamide in people with Myelodysplastic Syndromes, run by Zhongshan Hospital (Xiamen), Fudan University at 1 site in China.
Trial summary
Myelodysplastic syndromes (MDS) are a group of bone marrow disorders that can cause low blood cell counts and may progress to acute leukemia. Treatment options for patients with intermediate-to-high-risk MDS are limited, especially for older patients or those who are not suitable for intensive chemotherapy or… Read more about this study
- Who is it for?
- Ages 60 years and older
- Conditions the study is for
- Myelodysplastic Syndromes
- 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
- 15 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2026to 30 Jun 2027
- Phase
- Phase 2
- 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 60 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Aged 60 years or older at the time of informed consent.
- Diagnosis of myelodysplastic syndromes (MDS) according to the World Health Organization (WHO) classification.
- Intermediate-to-high-risk MDS, defined as at least one of the following:
- International Prognostic Scoring System (IPSS) risk category of Intermediate-2 or High, with bone marrow blasts <15%.
- Revised International Prognostic Scoring System (IPSS-R) risk category of Intermediate, High, or Very High, with bone marrow blasts <15%.
- Intermediate-1 risk MDS with grade 1 to 3 anemia and not suitable for hypomethylating agent therapy.
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- Evidence of persistent cytopenia affecting one or more hematopoietic lineages for at least 4 months, unless MDS-associated cytogenetic abnormalities or increased blasts are present.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.
- Adequate organ function as assessed by laboratory tests within 7 days before enrollment, including liver, renal, and cardiac function, in the opinion of the investigator.
- Ability to understand and willingness to sign a written informed consent form.
Who cannot join
- Bone marrow blasts >=15% at screening.
- Prior treatment with chidamide.
- Concurrent diagnosis of acute myeloid leukemia (AML) or other active hematologic malignancy.
- Receipt of intensive chemotherapy, hypomethylating agents, or other investigational agents within 4 weeks before enrollment.
- Uncontrolled active infection or severe concurrent medical condition that, in the investigator's judgment, would interfere with study participation.
- Clinically significant cardiac disease, including uncontrolled arrhythmia or clinically relevant QT interval prolongation.
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- Known hypersensitivity to chidamide or any of its excipients.
- Participation in another interventional clinical trial at the time of enrollment.
- Any condition that, in the investigator's judgment, would make the participant unsuitable for participation in this study.
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
Zhongshan Hospital (Xiamen), Fudan University
Xiamen, Fujian, 361015
About this study
Official title: A Single-Arm, Open-Label Phase II Clinical Trial Evaluating the Efficacy and Safety of Chidamide Monotherapy in Participants With Intermediate-to-High-Risk Myelodysplastic Syndromes
Myelodysplastic syndromes (MDS) are a group of bone marrow disorders that can cause low blood cell counts and may progress to acute leukemia. Treatment options for patients with intermediate-to-high-risk MDS are limited, especially for older patients or those who are not suitable for intensive chemotherapy or hypomethylating agents.
Chidamide is an oral histone deacetylase inhibitor that has shown antitumor activity in several hematologic malignancies. This study aims to evaluate the effectiveness and safety of chidamide used alone in patients with intermediate-to-high-risk MDS.
This is a prospective, single-arm, open-label phase II study conducted at a single center. Eligible participants will receive oral chidamide twice weekly and will be followed for treatment response and side effects. The results of this study may help determine whether chidamide could be a potential treatment option for patients with intermediate-to-high-risk MDS who have limited therapeutic choices.
Read the detailed description
This is a prospective, open-label, single-arm phase II study designed to evaluate the efficacy and safety of chidamide monotherapy in participants with intermediate-to-high-risk myelodysplastic syndromes (MDS) who have limited therapeutic options or are not suitable for intensive chemotherapy or hypomethylating agent therapy.
Eligible participants will receive oral chidamide at an initial dose of 20 mg twice weekly. Each dose consists of four 5-mg tablets taken approximately 30 minutes after meals. Treatment may continue for up to 3 months, with a planned follow-up period of 6 months.
The primary objective is to evaluate the overall response rate (ORR) after chidamide treatment. Treatment response will be assessed according to hematologic response and bone marrow evaluation as specified in the study protocol. Secondary objectives include assessment of safety, tolerability, dose modification, treatment discontinuation, and changes in blood cell counts.
Participants will be followed for treatment response, adverse events, laboratory abnormalities, and overall clinical status for up to 6 months after initiation of study treatment.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- Chidamide
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ChidamideDrug
Also called Tucidinostat
Chidamide will be administered orally at an initial dose of 20 mg twice weekly. Each dose consists of four 5-mg tablets taken approximately 30 minutes after meals. Treatment may continue for up to 3 months, with a planned follow-up period of 6 months.
Dose modifications are permitted based on hematologic and non-hematologic toxicities. In the event of grade 3 or higher hematologic toxicity or clinically significant non-hematologic toxicity, chidamide will be temporarily withheld and may be resumed at a reduced dose after recovery, according to the investigator's judgment. Treatment will be discontinued if unacceptable toxicity persists despite dose interruption or dose reduction.
| Study group | Role | What they receive |
|---|---|---|
| Chidamide Monotherapy | Experimental | Participants in this arm will receive oral chidamide monotherapy for the treatment of intermediate-to-high-risk myelodysplastic syndromes. Chidamide will be administered twice weekly, with dose modification based on safety, tolerability, and hematologic response. Receives: Drug: Chidamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate Assessed According to Study Protocol-Defined Response Criteria
Overall response rate is defined as the proportion of participants who achieve a protocol-defined treatment response after chidamide treatment. Response assessment will be based on hematologic parameters and bone marrow evaluation, as specified in the study protocol.
Measured: Up to 6 months after initiation of study treatment
Secondary outcomes (3)
Incidence of Treatment-Emergent Adverse Events
Treatment-emergent adverse events will be assessed based on clinical evaluation, laboratory testing, and investigator assessment during the study period.
Measured: Up to 6 months after initiation of study treatment
Proportion of Participants With Dose Interruption, Dose Reduction, or Treatment Discontinuation Due to Adverse Events
This outcome will assess the proportion of participants who require dose interruption, dose reduction, or permanent discontinuation of chidamide due to adverse events.
Measured: Up to 6 months after initiation of study treatment
Proportion of Participants Achieving Hematologic Improvement
Hematologic improvement will be assessed based on changes in peripheral blood counts and transfusion requirements according to the study protocol.
Measured: Up to 6 months after initiation of study treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jun 2026
- Study start1 Jul 2026 (estimated)
- Primary completion31 Mar 2027 (estimated)
- Study completion30 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Zhongshan Hospital (Xiamen), Fudan University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Zheng Wei, MD, Zhongshan Hospital (Xiamen), Fudan University
- Investigators
- Zheng Wei, Principal investigator, Zhongshan Hospital (Xiamen), Fudan University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07616336
- Sponsor's protocol ID
- B2025-040R
- Status last verified
- Jul 2025
- First submitted
- 17 Jan 2026
- Last updated
- 1 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-Arm, Open-Label Phase II Clinical Trial Evaluating the Efficacy and Safety of Chidamide Monotherapy in Participants With Intermediate-to-High-Risk Myelodysplastic Syndromes
- ClinicalTrials.gov ID
- NCT07616336
- Acronym / sponsor ID
- B2025-040R
- Conditions
- Myelodysplastic Syndromes
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Zhongshan Hospital (Xiamen), Fudan UniversityUniversity, hospital or other
- Study start
- 1 Jul 2026 (estimated)
- Primary completion
- 31 Mar 2027 (estimated)
- Study completion
- 30 Jun 2027 (estimated)
- First posted
- 1 Jun 2026 (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.
Myelodysplastic syndromes (MDS) are a group of bone marrow disorders that can cause low blood cell counts and may progress to acute leukemia. Treatment options for patients with intermediate-to-high-risk MDS are limited, especially for older patients or those who are not suitable for intensive chemotherapy or hypomethylating agents.
Chidamide is an oral histone deacetylase inhibitor that has shown antitumor activity in several hematologic malignancies. This study aims to evaluate the effectiveness and safety of chidamide used alone in patients with intermediate-to-high-risk MDS.
This is a prospective, single-arm, open-label phase II study conducted at a single center. Eligible participants will receive oral chidamide twice weekly and will be followed for treatment response and side effects. The results of this study may help determine whether chidamide could be a potential treatment option for patients with intermediate-to-high-risk MDS who have limited therapeutic choices.
Detailed description
This is a prospective, open-label, single-arm phase II study designed to evaluate the efficacy and safety of chidamide monotherapy in participants with intermediate-to-high-risk myelodysplastic syndromes (MDS) who have limited therapeutic options or are not suitable for intensive chemotherapy or hypomethylating agent therapy.
Eligible participants will receive oral chidamide at an initial dose of 20 mg twice weekly. Each dose consists of four 5-mg tablets taken approximately 30 minutes after meals. Treatment may continue for up to 3 months, with a planned follow-up period of 6 months.
The primary objective is to evaluate the overall response rate (ORR) after chidamide treatment. Treatment response will be assessed according to hematologic response and bone marrow evaluation as specified in the study protocol. Secondary objectives include assessment of safety, tolerability, dose modification, treatment discontinuation, and changes in blood cell counts.
Participants will be followed for treatment response, adverse events, laboratory abnormalities, and overall clinical status for up to 6 months after initiation of study treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Chidamide Monotherapy | Experimental | Drug: ChidamideParticipants in this arm will receive oral chidamide monotherapy for the treatment of intermediate-to-high-risk myelodysplastic syndromes. Chidamide will be administered twice weekly, with dose modification based on safety, tolerability, and hematologic response. |
Who may qualify
- Ages
- 60 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Aged 60 years or older at the time of informed consent.
- Diagnosis of myelodysplastic syndromes (MDS) according to the World Health Organization (WHO) classification.
- Intermediate-to-high-risk MDS, defined as at least one of the following:
- International Prognostic Scoring System (IPSS) risk category of Intermediate-2 or High, with bone marrow blasts <15%.
- Revised International Prognostic Scoring System (IPSS-R) risk category of Intermediate, High, or Very High, with bone marrow blasts <15%.
Key exclusion criteria (9 listed)
- Bone marrow blasts >=15% at screening.
- Prior treatment with chidamide.
- Concurrent diagnosis of acute myeloid leukemia (AML) or other active hematologic malignancy.
- Receipt of intensive chemotherapy, hypomethylating agents, or other investigational agents within 4 weeks before enrollment.
- Uncontrolled active infection or severe concurrent medical condition that, in the investigator's judgment, would interfere with study participation.
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: TucidinostatChidamide will be administered orally at an initial dose of 20 mg twice weekly. Each dose consists of four 5-mg tablets taken approximately 30 minutes after meals. Treatment may continue for up to 3 months, with a planned follow-up period of 6 months. Dose modifications are permitted based on hematologic and non-hematologic toxicities. In the event of grade 3 or higher hematologic toxicity or clinically significant non-hematologic toxicity, chidamide will be temporarily withheld and may be resumed at a reduced dose after recovery, according to the investigator's judgment. Treatment will be discontinued if unacceptable toxicity persists despite dose interruption or dose reduction. | Drug | Chidamide Monotherapy |
Primary outcome
Overall Response Rate Assessed According to Study Protocol-Defined Response Criteria
Time frame: Up to 6 months after initiation of study treatment
Overall response rate is defined as the proportion of participants who achieve a protocol-defined treatment response after chidamide treatment. Response assessment will be based on hematologic parameters and bone marrow evaluation, as specified in the study protocol.
3 secondary outcomes
Incidence of Treatment-Emergent Adverse Events
Time frame: Up to 6 months after initiation of study treatment
Proportion of Participants With Dose Interruption, Dose Reduction, or Treatment Discontinuation Due to Adverse Events
Time frame: Up to 6 months after initiation of study treatment
Proportion of Participants Achieving Hematologic Improvement
Time frame: Up to 6 months after initiation of study treatment
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 1 Jun 2026.
- Individual participant dataSharing planned: No
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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.
Zheng Wei principal investigator
Zhongshan Hospital (Xiamen), Fudan University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Chidamide in people with Myelodysplastic Syndromes, run by Zhongshan Hospital (Xiamen), Fudan 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 10 inclusion and 9 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 1 Jun 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.
What does this phase mean?
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?
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 Zheng Wei (+86-0592-3569860, wei.zheng@zs-hospital.sh.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Response Rate Assessed According to Study Protocol-Defined Response Criteria (time frame: Up to 6 months after initiation of study treatment). 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 1 Jun 2026.