Ruxolitinib plus decitabine intensified conditioning for AML patients having allogeneic stem cell transplant

Ruxolitinib and Decitabine-Enhanced Conditioning Versus Modified Bu/Cy or BuF Conditioning for the Impact on Relapse of Acute Myeloid Leukemia in First Complete Remission (CR1)After Allogeneic Hematopoietic Stem Cell Transplantation: A Multicenter, Prospective Randomized Controlled Trial

This trial will see if adding ruxolitinib and decitabine to standard busulfan-based conditioning before allogeneic HSCT lowers relapse in high‑risk AML patients in first complete remission (CR1).

Quick facts

• Phase: PHASE4
• Study type: Interventional
• Enrollment: 200 (estimated)
• Ages: 14 Years to 75 Years
• Sex: All
• Sponsor: Chinese PLA General Hospital (other)
• Drugs / interventions: Ruxolitinib, chemotherapy
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT07101588 on ClinicalTrials.gov

What this trial studies

This randomized Phase 4 trial compares an intensified conditioning regimen that adds ruxolitinib (step-based dose) and decitabine (20 mg/m²/day) to conventional Bu/Cy or BuF conditioning versus the conventional Bu/Cy or BuF regimen alone in patients with high‑risk AML in CR1 undergoing allogeneic HSCT. Eligible participants are aged 12–64 with appropriate matched or haploidentical donors and adequate organ function. The trial will track relapse rate after transplant, safety (including hematologic toxicity and non‑relapse mortality), and survival outcomes. The approach is based on prior single‑arm data and preclinical rationale that JAK‑STAT inhibition plus hypomethylation may reduce post‑transplant recurrence while maintaining acceptable toxicity.

Who should consider this trial

Good fit: Ideal candidates are patients aged 12–64 with high‑risk AML in first complete remission who have a matched or haploidentical donor, adequate liver and kidney function, no uncontrolled infection, and who can give informed consent.

Not a fit: Patients with acute promyelocytic leukemia (M3), those without a suitable donor, those outside the age or organ‑function criteria, pregnant patients, or those with uncontrolled psychiatric illness or infections are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, the regimen could lower post‑transplant relapse and improve long‑term survival for high‑risk AML patients undergoing allo‑HSCT.

How similar studies have performed: Prior single‑arm and clinical data suggest decitabine reduces post‑transplant recurrence and ruxolitinib has activity in hematologic malignancies, and small studies of the combination showed promising safety and low short‑term relapse but randomized confirmation is lacking.

Eligibility criteria

Inclusion Criteria:

* 1\) Acute myeloid leukemia with indications for allogeneic hematopoietic stem cell transplantation, CR1 2) Have HLA-matched sibling donors or haploidentical donors or ≥8/10 HLA-matched unrelated donors 3) The patients' ages range from 12 to 64 years old 4) Liver function: ALT and AST≤2.5 times the upper limit of normal values, bilirubin ≤2 times the upper limit of normal values 5) Renal function: Creatinine ≤ the upper limit of the normal value 6) There are no uncontrollable infections or serious mental and psychological disorders 7) Sign the informed consent form.

Exclusion Criteria:

* 1\. Patients with acute promyelocytic leukemia (M3) 2. One of the donor and recipient is pregnant 3. Suffering from mental illness or other conditions that prevent one from following the plan.

Where this trial is running

Beijing, Beijing Municipality

• Chinese PLA General Hospital — Beijing, Beijing Municipality, China (RECRUITING)

Study contacts

• Study coordinator: Li-ping Dou, Dr.

Conditions: Allogeneic Hematopoietic Stem Cell Transplantation, HSCT