RecruitingPhase 2

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

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
2 Years to 22 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Procedures mentioned: Blood draws, CT scans

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "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."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests whether ruxolitinib, tacrolimus, and methotrexate can prevent graft-versus-host disease, when donated cells attack the body. It is for children and young adults ages 2–22 getting a donor stem cell transplant for certain blood cancers or myelodysplastic syndrome, a disease of the bone marrow.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Be ages 2–22 and weigh at least 25 kg (about 55 pounds).
  • Have acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL) in complete remission, meaning no signs of cancer, or have myelodysplastic syndrome (MDS).
  • Be able to get a donor transplant, with a matched relative or fully matched unrelated donor willing to give bone marrow or blood stem cells.
  • Meet the study's daily activity scores: ECOG 2 or less, and Karnofsky or Lansky 60% or higher, based on age.
  • Have recovered from past cancer treatment side effects to mild or less, except hair loss.
  • You or your legal representative must give written consent. Children may also need to agree.
  • Allow use of stored tissue from earlier tumor biopsies. The study doctor may allow an exception if none is available.
  • If able to become pregnant, have a negative pregnancy test within 30 days before treatment. Participants who can have children must use effective birth control or avoid sex that could cause pregnancy during the study and for at least 6 months after treatment.

Who can't join

  • Have had any previous donor stem cell transplant, or a transplant using your own cells within the past year.
  • Have had chemotherapy, radiation, biological therapy, or immune therapy within 14 days before study treatment. Transplant preparation within 21 days and certain maintenance chemotherapy are allowed.
  • Use herbal medicines, or have had an allergic reaction to drugs similar to the study drugs.
  • Have a history of active tuberculosis, HIV, hepatitis B, or hepatitis C, or an uncontrolled infection throughout the body needing antibiotics.
  • Have had a blood clot, including a heart attack, stroke, or clot in the lungs or a deep vein, within 6 months of joining.
  • Have ongoing diarrhea from inflammatory bowel disease or trouble absorbing nutrients, a serious uncontrolled illness, or another active cancer.
  • Are pregnant or breastfeeding.
  • Have another condition the study doctor thinks makes participation unsafe, or may be unable to follow all study steps.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

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 sites (1)

CA