Itacitinib plus standard therapy in acute leukemia or myelofibrosis patients undergoing reduced-intensity conditioning donor stem cell transplantation

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ClinicalTrials.gov Published April 13, 2026 Medically reviewed April 19, 2026 Study authors: City of Hope Medical Center NCT04339101 ↗ By Dr. Julia Lee, PhD · Oncology, Genomics & Drug Development

Key Takeaway

Note that main results and safety data were not reported for this Phase IIa trial.

This Phase IIa trial investigated the efficacy and safety of itacitinib administered alongside standard treatment consisting of tacrolimus and sirolimus. The study population comprised 59 patients diagnosed with acute leukemia, myelodysplastic syndrome, or myelofibrosis who were undergoing reduced-intensity conditioning donor stem cell transplantation. The primary outcome measured was graft-versus-host disease free relapse-free (GRFS) survival at one year post-allogeneic stem cell transplantation. Secondary outcomes included cumulative incidence of acute and chronic graft-versus-host disease, non-relapse mortality, overall and progression-free survival, infection rates, second malignancies, and quality of life at specified intervals.

The provided input data does not contain specific numerical results for the primary or secondary outcomes, nor does it report details on adverse events, serious adverse events, discontinuations, or tolerability. Safety information was not reported in the source material.

Key limitations regarding the study design, specific results, and funding or conflicts of interest were not reported in the available information. Because the main results and safety data are absent from the input, the practice relevance and certainty of the findings cannot be determined from this record. Clinicians should interpret these data with caution due to the lack of reported efficacy metrics and safety profiles.

Given the absence of reported main results and safety data, the restrained practice relevance of this trial cannot be established from the current evidence. Further studies with complete reporting are needed to inform clinical decisions regarding itacitinib in this setting.

Study Details

Study typePhase2

Sample sizen = 59

EvidenceLevel 3

Follow-up30.3 mo

PublishedMar 2026

TrialNCT04339101

View Original Abstract ↓

Status: COMPLETED | Phase: PHASE2 Condition(s): Acute Leukemia, Hematologic and Lymphocytic Disorder, Myelodysplastic Syndrome, Primary Myelofibrosis, Secondary Myelofibrosis Intervention(s): Fludarabine (DRUG), Itacitinib Adipate (DRUG), Melphalan (DRUG), Quality-of-Life Assessment (OTHER), Questionnaire Administration (OTHER) This phase IIa trial studies the side effects of itacitinib when given together with standard treatment (tacrolimus and sirolimus), and to see how well it works in preventing graft-versus-host-disease (GVHD) in patients with acute leukemia, myelodysplastic syndrome or myelofibrosis who are undergoing reduced intensity conditioning donor stem cell transplantation. GVHD is a common complication after donor stem cell transplantation, resulting from donor immune cells recognizing recipients' cells and attacking them. Adding itacitinib to tacrolimus and sirolimus may reduce the risk GVHD and ultimately improve overall outcome and survival after donor stem cell transplantation. Detailed: PRIMARY OBJECTIVE: I. Following a patient safety lead-in, estimate graft-versus-host disease free relapse free (GRFS) survival at 1- year post allogeneic stem cell transplantation (alloHCT). SECONDARY OBJECTIVES: I. Estimate the cumulative incidence of acute graft-versus-host disease (aGVHD) and non-relapse mortality (NRM) at 100-days post-transplant. II. Estimate the cumulative incidence of chronic GVHD at 1- and 2-years post-transplant. III. Estimate the probabilities of overall and progression-free survival (OS/PFS) at 1- and 2-years post-transplant. IV. Estimate rate of infection and development of second malignancies including lymphoproliferative disorders at 1- and 2-years post-transplant. V. Assess patients' quality of life (QOL) at day 100 and 1 year post alloHCT. EXPLORATO Primary Outcome(s): Graft-versus-host Disease Free Relapse Free (GRFS) at 1 Year Enrollment: 59 (ACTUAL) Lead Sponsor: City of Hope Medical Center Start: 2020-11-11 | Primary Completion: 2023-05-22 Results posted: 2023-09-13

Itacitinib plus standard therapy in acute leukemia or myelofibrosis patients undergoing reduced-intensity conditioning donor stem cell transplantation

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