Chidamide maintenance after stem cell transplant for intermediate- or high-risk AML

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

This trial tests whether taking oral chidamide after an allogeneic stem cell transplant helps adults with intermediate- or high-risk AML stay in remission and live longer.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 134 (estimated)
• Ages: 18 Years to 75 Years
• Sex: All
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China (Academic / other)
• Drugs / interventions: chemotherapy
• Locations: 5 sites (Shijiazhuang, Hebei and 4 other locations)
• Trial ID: NCT07304232 on ClinicalTrials.gov

What this trial studies

This Phase II, open-label randomized trial will compare oral chidamide maintenance to observation in patients who achieve remission after allogeneic hematopoietic cell transplantation, with about 67 patients planned per arm. Chidamide is given 10 mg once daily on days 1–5 each week in 28-day cycles, with dose reductions to 5 mg allowed and treatment continued for up to 24 months or until progression or intolerable toxicity. The study's main outcomes are recurrence-free survival and overall survival, with additional measurement of duration of complete remission and tolerability. Eligible adults (18–75) must enroll 60–100 days post-transplant and have intermediate- or high-risk cytogenetics, while patients with FLT3 mutations, CNS disease, uncontrolled GVHD, or active infection are excluded.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18–75 who achieved first complete remission after allogeneic transplant for intermediate- or high-risk AML and can enroll 60–100 days post-transplant with good organ function and ECOG 0–1.

Not a fit: Patients with FLT3‑positive disease, active CNS involvement, uncontrolled grade 2–4 GVHD, uncontrolled infection, or prior post-transplant investigational therapy are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, chidamide maintenance could reduce relapse after transplant and extend remission and survival for high-risk AML patients.

How similar studies have performed: This approach is relatively novel in AML post-transplant—HDAC inhibitors like chidamide have shown activity in other blood cancers but robust randomized evidence as transplant maintenance in AML is limited.

Eligibility criteria

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.

Where this trial is running

Shijiazhuang, Hebei and 4 other locations

• The Second Hospital of Hebei Medical University — Shijiazhuang, Hebei, China (Not_yet_recruiting)
• The First Affiliated Hospital of Harbin Medical University — Harbin, Heilongjiang, China (Not_yet_recruiting)
• Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences — Tianjin, Tianjin Municipality, China (Recruiting)
• Tianjin Medical University General Hospital — Tianjin, Tianjin Municipality, China (Not_yet_recruiting)
• Tianjin People's Hospital — Tianjin, Tianjin Municipality, China (Not_yet_recruiting)

Study contacts

• Study coordinator: Jiang Erlie, doctor

Conditions Allogeneic Hematopoietic Cell Transplantation; AML