Post-transplantation Maintenance Therapy With Cidabenamide in Patients With Intermediate/High-risk AML (CM-AML-001)

Multicentre, Phase II Clinical Study of Post-transplantation Maintenance Therapy With Cidabenamide in Patients With Intermediate/High-risk AML

This study is a Phase II clinical trial designed to evaluate the efficacy and safety of Chidamide as maintenance therapy in high-risk acute myeloid leukemia (AML) patients following stem cell transplantation.

Trial Design: The trial is a single-arm, open-label study. The experimental group plans to enroll 67 patients, while the control group (observation only) also plans to enroll approximately 67 patients, with randomization. All patients must have received induction chemotherapy prior to enrollment and may or may not have received consolidation therapy. The chemotherapy regimen was determined by the treating physician. Patients had received induction and/or consolidation therapy, achieved remission, and underwent stem cell transplantation.

Study Objectives: The study aims to assess the impact of Chidamide maintenance therapy on recurrence-free survival (RFS), overall survival (OS), and the duration of complete remission. The study will also evaluate the tolerability and toxicity profile of this regimen, as well as the effect of maintenance therapy on the dynamics of minimal residual disease (MRD).

Study Overview

• Status: Recruiting
• Conditions: Allogeneic Hematopoietic Cell Transplantation (HCT); AML (Acute Myeloid Leukemia)
• Intervention / Treatment: Drug: Chidamide

Detailed Description

Chidamide : 10 mg/day, orally once daily (QD) on days 1-5 per week. During the study, the dose of chidamide may be adjusted at the physician's discretion to 5 mg/day. Each treatment cycle consists of 28 days. Treatment will continue indefinitely, with interruptions and dose adjustments implemented as needed to manage toxicity. Subjects will continue receiving the assigned treatment per investigator assessment for a maximum of 24 months, until documented disease progression, intolerable toxicity, withdrawal of consent, or meeting other protocol-specified criteria for treatment discontinuation (whichever occurs first). Patients who continue to derive clinical benefit, as discussed and agreed upon with the principal investigator, may remain in the study even in the event of relapse (if deemed clinically non-significant).

• Study Type: Interventional
• Enrollment (Estimated): 134
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Jiang Erlie, doctor; Phone Number: +86-15122538106; Email: jiangerlie@ihcams.ac.cn
• Study Contact Backup: Name: Liang Chen; Phone Number: +86-13612043271; Email: liangchen@ihcams.ac.cn

Study Locations

• China
  • Hebei
    • Shijiazhuang, Hebei, China, 050000
      • Not yet recruiting
      • The Second Hospital of Hebei Medical University
      • Contact: Fuxu Wang
  • Heilongjiang
    • Harbin, Heilongjiang, China, 150001
      • Not yet recruiting
      • The First Affiliated Hospital of Harbin Medical University
      • Contact: Shengjin Fan
  • Tianjin Municipality
    • Tianjin, Tianjin Municipality, China, 300020
      • Recruiting
      • Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences
    • Tianjin, Tianjin Municipality, China, 300020
      • Not yet recruiting
      • Tianjin Medical University General Hospital
      • Contact: Lijuan Li
    • Tianjin, Tianjin Municipality, China, 300020
      • Not yet recruiting
      • Tianjin People's Hospital
      • Contact: Xingli Zhao; Email: insectzhao@163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. AML patients meeting the following conditions (diagnosed per WHO 2022 AML criteria) who achieved first complete remission (CR) with intermediate-/high-risk cytogenetic abnormalities at the time of allogeneic transplantation.
2. Patients must achieve complete remission (CR) post-transplantation.
3. Enrollment must occur between 60 and 100 days after transplantation.
4. Age 18 to 75 years.
5. ECOG performance status 0-1.
6. Serum creatinine < 1.5 × ULN (upper limit of normal).
7. Serum direct bilirubin < 1.5 mg/dL (except in Gilbert's syndrome).
8. ALT and AST < 2.5 × ULN.
9. Ability to understand and provide written informed consent.

Exclusion Criteria:

1. Receipt of any other investigational drugs post-transplantation.
2. FLT3 mutation-positive status.
3. Central nervous system (CNS) involvement.
4. Uncontrolled grade 2-4 graft-versus-host disease (GVHD).
5. Uncontrolled active infection.
6. Known or suspected hypersensitivity to Chidamide or its excipients.
7. Uncontrolled congestive heart failure (CHF) or other concomitant systemic diseases or severe complications that, in the investigator's judgment, would make the patient unsuitable for participation in this study or would significantly compromise the proper assessment of the safety and toxicity of the prescribed regimen.
8. Pregnancy or breastfeeding.
9. Any other condition that, in the investigator's judgment, would make the patient unsuitable for participation in this study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Chidamide treatment group; Patients in the experimental group receive Chidamide at a dose of 10 mg/day, administered orally for the first 5 days of each week, followed by a 2-day treatment-free interval.	Drug: Chidamide; Patients in the experimental group receive Chidamide at a dose of 10 mg/day, administered orally for the first 5 days of each week, followed by a 2-day treatment-free interval.
No Intervention: Observation group

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Recurrence-Free Survival(RFS)	the rate from first dose administration until the first achievement of complete remission (CR) or complete remission with incomplete recovery (CRi), followed by either confirmed relapse or death from any cause, whichever occurs earlier.	2years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival (OS)	the 2-year survival rate since receiving maintenance treatment with Chidamide to death from any causes	2years
Event-Free Survival (EFS)	the 2-year event-free survival since receiving maintenance treatment with Chidamide to death from any causes	2years
Duration of Response (CRd)	the duration of remission in AML patients receiving maintenance treatment with Chidamide	2years
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0	Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.The frequency and severity of adverse events were evaluated based on changes in various vital sign indicators and laboratory tests.	2years
Minor residual lesions (MRD) Response Rate	Defined as the presence of less than 0.1% residual blast cells per white blood cell, measured via bone marrow examination. Other thresholds may also be explored and correlated with efficacy outcomes. Subjects who are randomized but do not undergo MRD assessment will be considered non-responders for the MRD response rate analysis.	2years

Collaborators and Investigators

• Sponsor: Institute of Hematology & Blood Diseases Hospital, China

Study record dates

Study Major Dates

• Study Start (Actual): September 1, 2025
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2028

Study Registration Dates

• First Submitted: September 5, 2025
• First Submitted That Met QC Criteria: December 11, 2025
• First Posted (Actual): December 26, 2025

Study Record Updates

• Last Update Posted (Actual): December 26, 2025
• Last Update Submitted That Met QC Criteria: December 11, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Hematologic Diseases; Leukemia, Myeloid; Leukemia; Hemic and Lymphatic Diseases; Leukemia, Myeloid, Acute; N-(2-amino-5-fluorobenzyl)-4-(N-(pyridine-3-acrylyl)aminomethyl)benzamide
• Other Study ID Numbers: IIT2025057

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No