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Acute Lymphoblastic LeukemiaPhase 2NCT06128070

Ruxolitinib With Tacrolimus and Methotrexate for the Prevention 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

A Phase 2 study testing Biospecimen Collection, Chest Computed Tomography and Echocardiography in people with Acute Lymphoblastic Leukemia and Acute Myeloid Leukemia, run by City of Hope Medical Center at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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. Read more about this study

Who is it for?
Ages 2 to 22 years
What is being tested?
Biospecimen Collection, Chest Computed Tomography and Echocardiography
Could I receive a placebo?
No, everyone receives an active treatment
How many people
40 participants
Where
1 siteUnited States
Study dates
1 Aug 2024to 22 Nov 2030
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 to 22 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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
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  • 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)

Who cannot join

  • 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
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  • 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)

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

United States1 site, 1 recruiting

  • City of Hope Medical Center

    Duarte, California, 91010

    Haris Ali, 626-218-2405, harisali@coh.org

    Haris Ali

    Recruiting

Other trials in United States

About this study

Official title: Phase 2a Study of Adding Ruxolitinib With Tacrolimus/Methotrexate Regimen for Graft-versus-Host Disease Prophylaxis in Myeloablative Conditioning Hematopoietic Cell Transplantation in Pediatric and Young Adult Patients

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.

Read the detailed description

PRIMARY OBJECTIVES:

  1. Determine if the addition of ruxolitinib phosphate (ruxolitinib) to tacrolimus and methotrexate as graft-versus-host disease (GVHD) prophylaxis, is safe in pediatric and young adult patients with hematologic malignancies who are eligible to undergo allogeneic hematopoietic cell transplantation (HCT) from a matched donor. (Safety lead-in segment) II. Following a patient safety lead-in, evaluate the efficacy of ruxolitinib, when given as part of reduced intensity HCT from a matched related/unrelated donor, as assessed by 1 year graft-versus-host disease-free and relapse-free (GRFS) rates in pediatric and young adult patients. (Phase II segment)

SECONDARY OBJECTIVES:

  1. Estimate the cumulative incidence of acute GVHD (aGVHD) and non-relapse mortality (NRM) at 100-days after transplant.

II. Estimate the cumulative incidence of chronic GVHD (cGVHD) at 1- and 2-years after transplant.

III. Estimate the probabilities of overall and progression-free survival (OS/PFS) at 1- and 2-years after transplant.

IV. Estimate the relapse/progression rate. V. Estimate rate of infection and development of second malignancies including lymphoproliferative disorders at 1- and 2-years post-transplant.

VI. Further evaluate the safety of this regimen by assessing:

VIa. Adverse event type, frequency, severity, attribution, time-course, and duration; VIb. Complications including: infection, and delayed engraftment.

EXPLORATORY OBJECTIVES:

  1. Characterize and evaluate hematologic recovery, donor cell engraftment and immune reconstitution by cell count and flow cytometry of lymphocyte subsets.

II. Characterize changes in aGVHD biomarkers (Reg-3alpha, sTNF RI, IL2Ralpha), JAK-regulated pro-inflammatory cytokines (i.e. IL-6, TNFalpha, C-reactive protein [CRP], beta2Microglubuolin) and STAT3 phosphorylation (downstream of JAK signaling) over time and by aGVHD status/grade.

III. Evaluate the pharmacokinetics of ruxolitinib in pediatric and young adult patients.

OUTLINE:

Patients receive ruxolitinib orally (PO) twice daily (BID) from day -1 to day +100, tacrolimus intravenously (IV) on day -1, and methotrexate IV on days +1, +3, +6, and +11, and undergo HCT on day 0. Patients also undergo chest computed tomography (CT) and echocardiography (ECHO)/multigated acquisition scan (MUGA) at screening and undergo collection of blood samples throughout the trial.

After completion of study treatment, patients are followed up at 30 days after the last dose of ruxolitinib and at 1 and 2 years post transplant.

Being tested
Biospecimen Collection, Chest Computed Tomography, Echocardiography, Hematopoietic Cell Transplantation, Methotrexate
Medical subject headings
Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute, Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Biospecimen CollectionProcedure or surgery

    Also called Biological Sample Collection, Biospecimen Collected, Specimen Collection

    Undergo blood sample collection

  • Chest Computed TomographyProcedure or surgery

    Also called Chest CT, Computed Tomography of the Chest

    Undergo chest CT

  • EchocardiographyProcedure or surgery

    Also called EC

    Undergo ECHO

  • Hematopoietic Cell TransplantationProcedure or surgery

    Also called HCT, Hematopoietic Stem Cell Infusion, Hematopoietic Stem Cell Transplantation, HSCT

    Undergo HCT

  • MethotrexateDrug

    Also called Abitrexate, Alpha-Methopterin, Amethopterin, Brimexate

    Given IV

  • Multigated Acquisition ScanProcedure or surgery

    Also called Blood Pool Scan, Equilibrium Radionuclide Angiography, Gated Blood Pool Imaging, Gated Heart Pool Scan

    Undergo MUGA

  • Ruxolitinib PhosphateDrug

    Also called INCB-18424 Phosphate, Jakafi, Jakavi

    Given PO

  • TacrolimusDrug

    Also called FK 506, FK-506, Fujimycin, Hecoria

    Given IV

Study groups in NCT06128070
Study groupRoleWhat they receive
Prevention (Ruxolitinib, tacrolimus, methotrexate)ExperimentalPatients 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.
Receives: Procedure: Biospecimen Collection; Procedure: Chest Computed Tomography; Procedure: Echocardiography; Procedure: Hematopoietic Cell Transplantation; Drug: Methotrexate; Procedure: Multigated Acquisition Scan; Drug: Ruxolitinib Phosphate; Drug: Tacrolimus

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of adverse events

    Defined using the modified Bearman Scale and the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0 scale.

    Measured: Up to day +30 post hematopoietic cell transplant (HCT)

  2. Graft-versus-host disease (GVHD)-free and relapse-free (GRFS)

    Will be estimated using the product-limit method of Kaplan and Meier.

    Measured: 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

Secondary outcomes (14)

  1. Patients receiving planned doses of ruxolitinib (feasibility)

    Patients who have received at least 80% of planned doses of ruxolitinib are deemed to meet feasibility criteria.

    Measured: At completion of therapy (up to day+100)

  2. Incidence of acute GVHD

    Will be graded and staged according to Mount Sinai Acute GVHD International Consortium criteria. Will be estimated using the method described by Gooley et al (1999).

    Measured: At 100 days post HCT transplant

  3. Incidence of non-relapse mortality

    Defined as death occurring in a patient from causes other than relapse or progression. Will be estimated using the method described by Gooley et al (1999).

    Measured: At 100 days post HCT transplant

  4. Incidence of chronic GVHD

    Will be evaluated and scored according to National Institutes of Health Consensus Staging. Will be estimated using the method described by Gooley et al (1999).

    Measured: At 1 and 2 years post HCT transplant

  5. Overall survival

    Will be estimated using the product-limit method of Kaplan and Meier.

    Measured: From the day of stem cell infusion until death, up to 2 years

  6. Progression free survival

    Will be estimated using the product-limit method of Kaplan and Meier.

    Measured: From the date of stem cell infusion to the date of death, disease relapse/progression, whichever occurs first, up to 2 years

  7. incidence of relapse/progression

    Will be estimated using the method described by Gooley et al (1999).

    Measured: From day of stem cell infusion (day 0) to first observation of disease relapse/progression, up to 2 years

  8. Infection rate

    Will be reported by site of disease, date of onset, severity and resolution, if any.

    Measured: From day -1 to day 130 post HCT transplant

  9. Incidence of secondary malignancies

    Measured: From day of stem cell infusion (day 0) to first observation of event of interest, assessed at 1 and 2 years post HCT transplant

  10. Hematologic recovery, donor cell engraftment and immune reconstitution

    Assessed using: absolute neutrophil count ≥ 0.5 x 10^3/uL achieved and sustained for 3 consecutive lab values on different days with no subsequent decline; platelets ≥ 20 K/uL independent of platelet transfusion support; immune reconstitution studies done by flow cytometry.

    Measured: Up to 2 years

  11. Incidence of adverse events during phase II segment

    Defined using the modified Bearman Scale and the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0 scale.

    Measured: Up to day +30 post HCT transplant

  12. Acute GVHD biomarkers

    Measured: Up to 2 years

  13. JAK-regulated pro-inflammatory cytokines

    Measured: Up to 2 years

  14. STAT3 phosphorylation

    Measured: Up to 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted13 Nov 2023
  2. Study start1 Aug 2024
  3. Primary completion22 Nov 2030 (estimated)
  4. Study completion22 Nov 2030 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Prevention
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
40 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06128070
Sponsor's protocol ID
23010
Other IDs
NCI-2023-07401, 23010, P30CA033572
Status last verified
Oct 2026
First submitted
27 Oct 2023
Last updated
14 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Biospecimen Collection, Chest Computed Tomography and Echocardiography in people with Acute Lymphoblastic Leukemia and Acute Myeloid Leukemia, run by City of Hope Medical Center at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 15 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 14 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of adverse events (time frame: Up to day +30 post hematopoietic cell transplant (HCT)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06128070.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2026.

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