Ruxolitinib and Azacitidine for AML After Stem Cell Transplant
This study is looking at the safety and best dose of two medications, ruxolitinib and azacitidine, for patients with acute myeloid leukemia (AML) who have had a reduced intensity allogeneic stem cell transplant (alloHSCT). An alloHSCT is a procedure where you receive blood-forming cells from a donor, often a family member or unrelated donor. The study will first test ruxolitinib alone, then ruxolitinib with azacitidine. Researchers want to see if these treatments can help prevent the leukemia from coming back and manage side effects. This study is for adults aged 18 and older with any type of AML. The main goals are to find out how safe these drugs are and what side effects they might cause.
- Study design
- This interventional study plans to enroll 40 participants. It will first test ruxolitinib alone, then ruxolitinib with azacitidine.
- What's involved
- You would undergo an allogeneic hematopoietic stem cell transplant, receive azacitidine intravenously, and have blood and bone marrow samples collected.
- Compensation
- Not stated in the trial record.
- Follow-up
- Side effects will be monitored up to 30 days after the last dose of the study drug.
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NCT07548983
Ruxolitinib With Azacitidine Maintenance for the Treatment of Patients With Acute Myeloid Leukemia Undergoing Reduced Intensity Allogeneic Stem Cell Transplantation
Recruiting
PHASE1Ages 18+InterventionalTreatmentOHSU Knight Cancer InstituteInvestigator-initiated
~40 participants
Updated 2026-07-06 on ClinicalTrials.gov
What's tested:Allogeneic Hematopoietic Stem Cell TransplantationAzacitidineBiospecimen CollectionBone Marrow AspirationBone Marrow BiopsyCyclophosphamide
At a glance
Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of dose limiting toxicities (DLTs)
Measured over From cycle 1 day 1 (day +5 post-transplant) to end of cycle 2 (Cycle length = 28 days)
+1 more outcome measured
Conditions
Where it's being run
1 sites across 1 statesOregon1
Study leadership
- Jennifer N Saultz · PRINCIPAL_INVESTIGATOR · OHSU Knight Cancer Institute
Who to contact
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Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
PART A: Willingness to provide written informed consent before any study-specific procedures or interventions are performed. For participants unable to independently provide consent, a legally authorized representative (LAR) must provide consent
PART A: Age ≥ 18 years, at the time of consent
PART A: All types and categories of AML, as defined by World Health Organization (WHO) 2022, excluding acute promyelocytic leukemia (APL)
PART A: In complete remission (CR) or complete remission with incomplete blood count recovery (CRi) after induction of remission for transition to transplant by European LeukemiaNet 2022 Risk Stratification (ELN 2022)
PART A: Planned alloHSCT with granulocyte colony-stimulating factor mobilized peripheral blood stem cells (PBSCs) and pre transplant conditioning disease status assessment of CR or CRi, as defined by ELN 2022 criteria
PART A: Patient is at high risk for relapse based on cytogenetics, MRD by next generation sequencing (NGS), and/or ELN 2022 definition of adverse risk disease per the opinion of the treating physician
PART A: Patients must have an unrelated PBSC donor meeting study donor selection requirements
PART A: Only RIC or NMA conditioning must be plan and patient is not a candidate for myeloablative conditioning (MAC). Post-transplant cyclophosphamide / tacrolimus / mycophenolate mofetil (PTCy/Tac/MMF) GVHD prophylaxis is planned with PTCy at 25 mg/kg/day on Day +3 and Day +4 post-HSCT
Permitted conditioning regimens (per institutional protocol)
Reduced-intensity conditioning:
Fludarabine/ melphalan
Dose reduction of melphalan to 100 rather than 140 are permitted at discretion of treating physician
Non-Myeloablative Conditioning:
Fludarabine/ busulfan/ total-body irradiation (TBI)
Fludarabine/ TBI
PART A: Backup graft donor (meeting study donor selection requirements) identified and ready for cases of graft failure
PART A: Karnofsky performance status (KPS) ≥ 50%
PART A: Direct bilirubin ≤ 3 × upper limit of normal (ULN) within 7 days prior to start of conditioning. Patients with leukemic involvement or Gilbert's Syndrome must have a direct bilirubin of ≤ 2 x ULN within 7 days of start of conditioning
PART A: Creatinine clearance \> 30 mL/min, calculated by the Cockcroft-Gault formula or measured by 24-hour urine collection
PART A: Willingness to receive infusion of SOC blood products
PART A: Evidence of chronic hepatitis B virus (HBV) infection (i.e., hepatitis B virus surface antigen \[HBsAg\]-positive with undetectable or low HBV deoxyribonucleic acid \[DNA\]) in the absence of HBV therapy, or serologic evidence of a resolved prior HBV infection (i.e., HBsAg-negative and hepatitis B virus core antibody \[anti-HBc-positive\]) is permitted but patient must agree to appropriate institutional guideline prophylaxis
PART A: History of hepatitis C virus (HCV) infection is permitted given prior curative treatment or undetectable HCV viral load by serology or polymerase chain reaction (PCR) testing
Patients who are HCV antibody (Ab) seropositive but HCV ribonucleic acid (RNA) negative due to prior treatment or natural resolution are eligible
PART A: For persons of child-bearing potential (PCBP), a negative pregnancy test within ≤ 7 days of start of conditioning
PART A: Ruxolitinib may have adverse effects on a fetus in utero. Furthermore, it is not known if either drug agent has transient adverse effects on the composition of sperm. PCBP and participants who produce viable sperm must be willing to comply with study requirements for contraception starting at start of conditioning through 120 days after discontinuation of study treatment. Should a participant or participant's sexual partner become pregnant or suspect a pregnancy while participating in this study, the individual should inform their treating physician immediately
PART A: It is unknown whether ruxolitinib, or its metabolites, are excreted in human milk. Since many drugs are excreted in human milk, and because of the potential for serious adverse reactions in the nursing infant, patients must agree to not breast-feed for the entire 2 years of the study to be eligible for enrollment
PART B: Absolute neutrophil count (ANC) ≥ 1,000/μL, measured twice approximately 48 hours apart, within 7 days prior to C2D1, in the absence of granulocyte colony-stimulating factor (G-CSF) treatment during this 7-day period
PART B: Platelet count ≥ 50,000/μL, measured twice approximately 48 hours apart, within 7 days prior to C2D1, in the absence of platelet transfusions or growth factor therapies that increase platelet counts during this 7 day period
PART B: Hemoglobin \> 8 g/L within the 7 days prior to C2D1, regardless of transfusion support
PART B: Day +30 (+/- 5 days) chimerism of ≥ 70% donor CD3+ cells and 100% donor CD33+ cells
PART B: For PCBP, a negative pregnancy test within 72 hours prior to C2D1
DONOR: Donor is willing and able to donate
DONOR: Unrelated donor with a 7/8 or 8/8 match at human leukocyte antigen (HLA)-A, -B, -C and -DRB1 at high resolution using DNA-based typing. Unrelated donor must be willing to donate peripheral blood stem cells
DONOR: Age 18-35 at the time of workup request for PBSC donation
DONOR: Meet the donor registries' medical suitability requirements for PBSC donation
DONOR: Must undergo screening, testing, and determination of eligibility according to current Food and Drug Administration (FDA) requirements as defined in 21 Code of Federal Regulations (CFR) 1271 Subpart C and applicable guidance documents. Donors will be tested using kits that are FDA licensed, approved, or cleared for human cell and tissue/tissue-product donor screening. Sample testing will be performed at Clinical Laboratory Improvement Amendments (CLIA) certified laboratories
DONOR: Must agree to donate PBSC
DONOR: Must have the ability to give informed consent according to standard (non study) informed consent according to applicable donor regulatory requirements
Exclusion
PART A: Patients with active central nervous system (CNS) involvement with AML. Prior diagnosis of CNS involvement of AML will be allowed if curatively treated
PART A: Patients with malabsorption syndrome or other condition that precludes oral route of drug administration
PART A: Patients with prior intolerance to any of the interventional study drugs or component of the formulations
PART A: Patients with prior failure of treatment with ruxolitinib
PART A: Patients with history of any other malignancy within the 5 years prior to screening, with the exception of the following:
Adequately treated in situ carcinoma of the cervix uteri or carcinoma in situ of breast;
Basal cell carcinoma of the skin or localized squamous cell carcinoma of the skin;
Previous non-hematologic malignancy that has been successfully treated with curative intent (i.e., confined and surgically resected or treated with other modalities);
Myelodysplastic syndrome (MDS) or myeloproliferative neoplasm (only allowed if it transformed to AML and AML should be the indication for marrow transplantation)
PART A: Patients with ejection fraction (EF) \< 40% and patients with New York Heart Association (NYHA) grade III or IV heart failure
PART A: Patients with history of pulmonary embolism (PE) or myocardial infarction (MI) within the 6 months prior to cycle 1 day 1 (C1D1). Treated deep vein thromboses (DVTs) are allowed
PART A: Patients with known history of stroke (including intracranial hemorrhage) within 60 days prior to start of conditioning
PART A: Patients administered therapy with an investigational agent, a known JAK1/2 inhibitor (with the exception of ruxolitinib), or a SOC anti-cancer therapy, including chemotherapy and radiotherapy, within 5 half lives prior to C1D1, or per institutional practice
PART A: Patients administered therapy with a biologic agent (e.g., a monoclonal antibody) for anti-neoplastic intent within 30 days prior to C1D1, or per the institutional practice
PART A: Patients with known clinically significant liver disease defined as ongoing drug-induced liver injury, alcoholic liver disease, non alcoholic steatohepatitis, primary biliary cirrhosis, extrahepatic obstruction caused by cholelithiasis, cirrhosis of the liver, portal hypertension, or history of autoimmune hepatitis
PART A: Patients with known human immunodeficiency virus (HIV) infection. HIV infection, even when controlled with combination antiretroviral therapy, is not allowed due to increased risk of lethal infections associated with marrow-suppressive therapy. Studies in participants under combination antiretroviral therapy may be undertaken at a later date
PART A: Patients with history of active tuberculosis (TB)
PART A: Patients with known active severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection
Subjects with confirmed SARS-CoV-2 infection must be screen failed and may only rescreen after they meet institutional SARS-CoV-2 infection viral clearance criteria
PART A: Patients with evidence of clinically significant systemic infection
PART A: Patients with clinically significant coagulation abnormality defined by international normalized ratio (INR) \> 1.5
PART A: Patients with recent significant medical interventions, such as major surgery within 28 days of C1D1. Standard of care procedures for patients with hematologic malignancies, such as biopsies and lumbar punctures, are allowed
PART A: Patients with psychiatric illness/social situation that would limit compliance with the study
PART A: Patients with history of clinically significant medical condition(s) (including comorbidities) and no other reason, per the determination of the investigator, which could interfere with the patient's adherence to the study protocol or would make the patient an unsuitable candidate to receive study drugs
PART B: Patients with evidence of primary graft failure (PGF) or secondary graft failure (SGF) while enrolled on Part A or SGF during screening for Part B
PART B: Patients who experienced a dose limiting toxicity (DLT) in Part A
PART B: Patients administered an investigational agent, a known JAK1/2 inhibitor (with the exception of ruxolitinib), or SOC chemotherapy or radiotherapy within 5 half-lives of C2D1, or per institutional practice
PART B: Patients administered a biologic of anti-neoplastic intent within 30 days of C2D1, or per institutional practice
PART B: Patients with a plan for other maintenance therapy outside of this trial, including a FLT3-ITD inhibitor
PART B: Patients with evidence of clinically significant, uncontrolled systemic infection
PART B: Patients with Mount Sinai Acute Graft Versus Host Disease International Consortium (MAGIC) grade II or greater active aGVHD
PART B: Patients with ongoing or planned therapy with prednisone equivalent of ≥ 10 mg daily for GVHD treatment or other illness
PART B: Patients with newly diagnosed psychiatric illness or new social situation that would limit compliance with the study
PART B: Patients with new or recent history of clinically significant medical condition(s) (including comorbidities) or any other reason, per the determination of the investigator, which could interfere with the patient's adherence to the study protocol or would make the patient an unsuitable candidate to receive study drugs
DONOR: Recipient has evidence of donor-specific HLA antibody (DSA). If recipient is positive for HLA antibodies against a mismatched HLA in the selected donor, defined as the presence of DSA to any mismatched HLA allele/antigen at any of the following loci (HLA-A, -B, -C, -DRB1, DRB3, DRB4, DRB5, -DQA1, - DQB1, -DPA1, -DPB1) with mean fluorescence intensity (MFI) \> 3000 by microarray-based single antigen bead testing, the donor will be excluded. In patients receiving red blood cell or platelet transfusions, DSA evaluation should be performed prior to donor mobilization and initiation of recipient preparative regimen whenever possible
DONOR: Donors with positive coronavirus disease 2019 (COVID-19) testing results (confirmed by PCR) within 72 hours (hr) of stem cell collection
What this trial measures
- Incidence of dose limiting toxicities (DLTs)From cycle 1 day 1 (day +5 post-transplant) to end of cycle 2 (Cycle length = 28 days)
Will determine the safety and tolerability of ruxolitinib monotherapy (part A) and ruxolitinib plus azacitidine therapy (part B). DLTs will be used to identify a maximum tolerated dose. Dose levels will either be de-escalated, escalated, or retained-according to pre calculated Bayesian Optimal Interval decision rules.
- Incidence of grade 3+ treatment-related adverse events (TRAEs) (Part B)Up to 30 days after last dose of study drug
TRAEs of grade 3 and higher experienced during treatment with the doublet of ruxolitinib + azacitidine will be reported (with frequencies and percentages) as a co-primary endpoint. TRAEs are defined as adverse events that are definitely or possibly attributable to study treatment. Will be assessed using Common Terminology Criteria for Adverse Events version 5.0, where adverse events are assessed on a scale of 1-5, with higher scores indicating worse outcomes.