Chidamide Monotherapy for Intermediate-to-High-Risk Myelodysplastic Syndromes

NCT07616336 | Not Yet Recruiting Phase 2

• 1 other identifier: B2025-040R
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief 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 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.

Conditions

Myelodysplastic Syndromes

Outcome Measures

Primary Outcomes (1)

• 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.

  Up to 6 months after initiation of study treatment

Secondary Outcomes (3)

• Incidence of Treatment-Emergent Adverse Events

  Up to 6 months after initiation of study treatment

• Proportion of Participants With Dose Interruption, Dose Reduction, or Treatment Discontinuation Due to Adverse Events

  Up to 6 months after initiation of study treatment

• Proportion of Participants Achieving Hematologic Improvement

  Up to 6 months after initiation of study treatment

Study Arms (1)

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.

Drug: Chidamide

Interventions

Chidamide DRUG

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.

Also known as: Tucidinostat

Chidamide Monotherapy

Eligibility Criteria

• Age: 60 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Zhongshan Hospital (Xiamen), Fudan University: lead

Study Sites (1)

Zhongshan Hospital (Xiamen), Fudan University

Xiamen, Fujian, 361015, China

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

N-(2-amino-5-fluorobenzyl)-4-(N-(pyridine-3-acrylyl)aminomethyl)benzamide

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Zheng Wei

  Zhongshan Hospital (Xiamen), Fudan University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Zheng Wei

CONTACT

+86-0592-3569860; wei.zheng@zs-hospital.sh.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Chief Physician

Study Record Dates

• First Submitted: January 17, 2026
• First Posted: June 1, 2026
• Study Start (Estimated): July 1, 2026
• Primary Completion (Estimated): March 31, 2027
• Study Completion (Estimated): June 30, 2027
• Last Updated: June 1, 2026

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)