Recruiting
Phase 2

Ruxolitinib with Tacrolimus & Methotrexate

Sponsor:

City of Hope Medical Center

Code:

NCT06128070

Conditions

Acute Lymphoblastic Leukemia

Acute Myeloid Leukemia

Myelodysplastic Syndrome

Eligibility Criteria

Sex: All

Age: 2 - 22

Healthy Volunteers: Not accepted

Interventions

Biospecimen Collection

Chest Computed Tomography

Echocardiography

Hematopoietic Cell Transplantation

Methotrexate

Study Details

Brief summary:

This phase II trial tests how well ruxolitinib with tacrolimus and methotrexate work to prevent the development of graft versus host disease in pediatric and young adult patients undergoing allogeneic hematopoietic cell transplant for acute myeloid leukemia, acute lymphoblastic leukemia, or myelodysplastic syndrome. Ruxolitinib is a type of medication called a kinase inhibitor. It works by blocking the signals of cells that cause inflammation and cell proliferation, which may help prevent graft versus host disease (GVHD). Tacrolimus is a drug used to help reduce the risk of rejection by the body of organ and bone marrow transplants by suppressing the immune system. Methotrexate stops cells from making DNA, may kill cancer cells, and also suppress the immune system, which may reduce the risk of GVHD. Giving ruxolitinib with tacrolimus and methotrexate may prevent GVHD in pediatric and young adults undergoing allogeneic hematopoietic cell transplants.

Conditions

Acute Lymphoblastic Leukemia

Acute Myeloid Leukemia

Myelodysplastic Syndrome

Study ID

NCT06128070

Start date

Jul 16, 2024

Status verified date

Oct, 2025

Completion date

Nov 22, 2030

Anticipated

Primary completion date

Nov 22, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 22

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Documented informed consent of the participant and/or legally authorized representative

  • Assent, when appropriate, will be obtained per institutional guidelines
  • Agreement to allow the use of archival tissue from diagnostic tumor biopsies

  • If unavailable, exceptions may be granted with study primary investigator (PI) approval
  • Age: 2-22
  • Weight ≥25kg
  • Eastern Cooperative Oncology Group (ECOG) ≤ 2
  • Performance status: Karnofsky ≥ 60% for patients ≥ 16 years old OR Lansky status ≥ 60% for patients < 16 years old
  • Candidate for allogeneic bone marrow transplant with and available matched related donor (MRD) or an 8/8 matched unrelated donor (MUD) who is willing to donate bone marrow (BM) or mobilized peripheral blood stem cells

  • Note: Donor selection process will be in accordance with City of Hope (COH)-standard operating procedures (SOPs) (B.001.09 Allogeneic Cellular Therapy Product Donor Evaluation, Selection \& Consent), which follows Food and Drug Administration (FDA) guidelines for donation of hematopoietic stem/progenitor cells (HPCs) obtained from peripheral blood or bone marrow
  • Diagnosis of acute leukemia (acute myeloid leukemia \[AML\] or acute lymphoblastic leukemia \[ALL\]) in complete remission, or myelodysplastic syndrome (MDS)
  • Fully recovered from the acute toxic effects (except alopecia) to ≤ grade 1 from prior anti-cancer therapy
  • Women of childbearing potential (WOCBP): negative urine or serum pregnancy test. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required (to be performed within 30 days prior to day 1 of protocol therapy)
  • Agreement by females and males of childbearing potential to use an effective method of birth control or abstain from heterosexual activity for the course of the study through at least 6 months after the last dose of protocol therapy

  • Childbearing potential defined as not being surgically sterilized (men and women) or have not been free from menses for > 1 year (women only)

Exclusion Criteria:

  • Autologous stem cell transplant within 1 year prior to day 1 of protocol therapy
  • Prior allogeneic transplantation
  • Chemotherapy, radiation therapy, biological therapy, immunotherapy within 14 days prior to day 1 of protocol therapy

  • Note: Conditioning regimen within 21 days prior to day 1 of protocol therapy is not considered as an exclusion criterion.
  • Note: Patients on maintenance chemotherapy with agents listed are not excluded
  • Herbal medications
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to study agent
  • History of active tuberculosis
  • Patients with history of thrombosis including but not limited to myocardial infarction (MI)/stroke and pulmonary embolism (PE)/deep vein thrombosis (DVT) within 6 months of enrollment
  • Active diarrhea due to inflammatory bowel disease or malabsorption syndrome
  • Clinically significant uncontrolled illness
  • Active, uncontrolled systemic infection (viral, bacterial, or fungal) requiring antibiotics
  • Known history of immunodeficiency virus (HIV) or hepatitis B or hepatitis C infection
  • Other active malignancy
  • Females only: Pregnant or breastfeeding
  • Any other condition that would, in the investigator's judgment, contraindicate the patient's participation in the clinical study due to safety concerns with clinical study procedures
  • Prospective participants who, in the opinion of the investigator, may not be able to comply with all study procedures (including compliance issues related to feasibility/logistics)

Study Design

Enrollment

40 participants

Anticipated

Intervention Model

Single group

Primary purpose

Prevention

Interventions and Outcome Measures

Arms

experimental: Prevention (Ruxolitinib, tacrolimus, methotrexate)

Patients receive ruxolitinib PO BID from day -1 to day +100, tacrolimus IV on day -1, and methotrexate IV on days +1, +3, +6, and +11, and undergo HCT on day 0. Patients also undergo chest CT and ECHO/MUGA at screening and undergo collection of blood samples throughout the trial.

Interventions

Biospecimen Collection

Undergo blood sample collection

Chest Computed Tomography

Undergo chest CT

Echocardiography

Undergo ECHO

Hematopoietic Cell Transplantation

Undergo HCT

Methotrexate

Given IV

Multigated Acquisition Scan

Undergo MUGA

Ruxolitinib Phosphate

Given PO

Tacrolimus

Given IV

Primary outcome measure

  • Incidence of adverse events [ Time Frame: Up to day +30 post hematopoietic cell transplant (HCT) ]
  • Graft-versus-host disease (GVHD)-free and relapse-free (GRFS) [ Time Frame: From the date of transplantation to the first time of observing the following events: grade 3-4 acute GVHD, chronic GVHD requiring systemic treatment, relapse, or death, assessed at 1 year post transplantation ]

Central Contacts and Locations

Locations

City of Hope Medical Center

Recruiting

Duarte, California, United States, 91010

Contacts

Haris Ali

626-218-2405harisali@coh.org

Principal Investigator:

Haris Ali

More Information

Sponsor

City of Hope Medical Center

Last update posted

Oct 31, 2025

Last verified

Oct, 2025

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by City of Hope Medical Center on 2025-10-31.