RecruitingPhase 2

Total Marrow and Lymphoid Irradiation in Combination With Fludarabine and Melphalan as Conditioning for Allogeneic Peripheral Blood Stem Cell Hematopoietic Cell Transplant in Older Patients With Refractory and Relapsed Acute Myeloid Leukemia and High-risk Myelodysplastic Syndrome

Phase 2 Trial of Total Marrow and Lymphoid Irradiation (TMLI) as Conditioning for Allogeneic Peripheral Blood Stem Cell Hematopoietic Cell Transplantation (PBSC-HCT) From a Match Donor With Fludarabine and Melphalan in Older Patients With Refractory Acute Myeloid Leukemia and MDS

Who can join

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

Age
18 Years and older
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, Biopsies

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
  • "Collect longitudinal blood samples for immune analysis II."
  • "Collect longitudinal blood samples to assess presence and levels of GVHD biomarkers and inflammatory cytokines III."
  • "Collect longitudinal blood samples for circulating tumor DNA (ctDNA) profiling."
  • "Additionally, patients undergo echocardiography or multigated acquisition scan (MUGA), computed tomography (CT), urine and blood sample collection, and bone marrow biopsy throughout the study."
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 targeted radiation with two cancer drugs, fludarabine and melphalan, before a blood stem cell transplant from a matched donor. It is mainly for older adults with hard-to-treat acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS), diseases that affect blood-forming cells. The study looks at how safe this treatment is and how well it works.

This phase II trial tests the effect of total marrow and lymphoid irradiation (TMLI) in combination with fludarabine and melphalan as conditioning regimen in older patients with acute myeloid leukemia or high-risk myelodysplastic syndrome that has not responded to previous treatment (refractory) and that has come back after a period of improvement (relapsed) and are undergoing a donor (allogeneic) peripheral blood stem cell (PBSC) hematopoietic cell transplant (HCT) from a matched related or unrelated donor. HCT is the only curative treatment for high-risk patients, but the side effects related to the current conditioning treatments limit the use to younger and more fit patients. TMLI is a targeted form of total body radiation that uses intensity-modulated radiation therapy to target marrow, lymph node chains, and the spleen. It is designed to reduce radiation-associated side effects and maximize the radiation therapeutic effect. Fludarabine blocks cells from making deoxyribonucleic acid (DNA) and may kill cancer cells. It is a type of purine antagonist and a type of ribonucleotide reductase inhibitor. Melphalan is in a class of medications called alkylating agents. It may kill cancer cells by damaging their DNA and stopping them from dividing. Giving chemotherapy, such as fludarabine and melphalan, and TMLI before an allogeneic transplant helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells to grow. When healthy stem cells from a related or unrelated donor, such as PBSC HCT, that closely match the patient's blood, are infused into a patient, they may help the patient's bone marrow make more healthy cells and platelets, an may help destroy any remaining cancer cells. Giving TMLI in combination with fludarabine and melphalan as conditioning treatment for an allogeneic PBSC HCT from a matched related or unrelated donor may be safe, tolerable, and/or effective in treating high-risk older patients with relapsed and refractory acute myeloid leukemia or high-risk myelodysplastic syndrome.

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

Eligibility

Who can join

  • Be age 50 or older. Adults ages 18–49 may join if other health problems or active disease make stronger transplant preparation unsafe.
  • Have confirmed AML with intermediate- or poor-risk features, or active AML, including small amounts found by special tests; or have MDS or chronic myelomonocytic leukemia (CMML) with at least 10% immature blood cells called blasts.
  • Have a daily activity score of at least 70 on the study's scale.
  • Have a fully matched sibling or unrelated donor willing to give blood stem cells.
  • Meet liver and kidney test limits. Heart pumping strength must be at least 50%. Lung tests must be at least 50% of expected, or oxygen levels must be above 92% without added oxygen if lung tests cannot be done.
  • Meet infection testing rules. Tests for HIV and hepatitis B and C must be negative, or the amount of virus must be too low to detect. People with HIV must be on effective treatment.
  • Give written consent, personally or through a legal representative. Allow use of stored tumor biopsy tissue, unless the study leader approves an exception.
  • If able to become pregnant, have a negative pregnancy test. Anyone able to have children must use effective birth control or avoid sex that could cause pregnancy during treatment and for at least 6 months afterward.

Who can't join

  • Had a donor stem cell transplant, or a transplant using your own cells within the past year.
  • Had chemotherapy, radiation, biological therapy, or immune treatment within 14 days before study treatment. Some low-dose or maintenance cancer drugs are allowed closer to treatment.
  • Had more than three rounds of different intensive chemotherapy treatments meant to make the cancer go away.
  • Are in another trial testing maintenance treatment after transplant or a treatment that could affect how long the cancer stays away.
  • Have had an allergic reaction to a drug similar to a study drug.
  • Have a serious uncontrolled illness or an active infection that does not respond to antibiotics.
  • Have another active cancer that could affect the study's safety or results, or are pregnant or breastfeeding.
  • Have another condition the study doctor considers 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 principal investigator (PI) approval * Age: ≥ 50 years (no upper age limit) * Note: Patients ≥ 18 years and \< 50 years are also included if they are not candidates for myeloablative conditioning regimens due to comorbidities or active disease * Karnofsky or Lansky performance status ≥ 70 * Eligible patients will have a histopathological confirmed diagnosis of hematologic malignancy in one of the following categories: * Acute myeloid leukemia (AML): * Patients with de novo or secondary disease in unfavorable risk group including poor risk cytogenetics according to National Comprehensive Cancer Network (NCCN) guidelines for AML i.e., monosomal karyotype, -5,5q-,-7,7q-,11q23-non t(9;11), inv (3), t(3;3), t(6;9), t(9;22) and complex karyotypes (≥ 3 unrelated abnormalities), or all patient in intermediate risk groups * Patients with active disease: * Morphologically * Minimal residual disease (MRD) testing (MRD+ through flow cytometry, cytogenetics, or molecular assays) * Myelodysplastic syndrome/chronic myelomonocytic leukemia (CMML) (MDS) with ≥ 10% blast * Patients must have an human leukocyte antigen (HLA) (A, B, C, and DRB1) identical sibling or a 8/8 (A, B, C, and DR) allele matched unrelated donor who is willing to donate primed blood stem cells * Serum direct bilirubin ≤ 2.0 x upper limit of normal (ULN) (unless has Gilbert's disease) (within 35 days prior to day 1 of protocol therapy unless otherwise stated) * Aspartate aminotransferase (AST) ≤ 2.5 x ULN (within 35 days prior to day 1 of protocol therapy unless otherwise stated) * Alanine aminotransferase (ALT) ≤ 2.5 x ULN (within 35 days prior to day 1 of protocol therapy unless otherwise stated) * Creatinine clearance of ≥ 60 mL/min per 24 hour urine test or the Cockcroft-Gault formula (within 35 days prior to day 1 of protocol therapy unless otherwise stated) * Left ventricular ejection fraction (LVEF) ≥ 50% * Note: To be performed within 35 days prior to day 1 of protocol therapy * If able to perform pulmonary function tests: Forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC) and DLCO (diffusion capacity) ≥ 50% of predicted (corrected for hemoglobin) * Note: To be performed within 35 days prior to day 1 of protocol therapy * If unable to perform pulmonary function tests: Oxygen (O2) saturation \> 92% on room air * Note: To be performed within 35 days prior to day 1 of protocol therapy * Seronegative for HIV antigen (Ag)/antibody (Ab) combo, hepatitis C virus (HCV), active hepatitis B virus (HBV) (surface antigen negative) (within 35 days prior to day 1 of protocol therapy unless otherwise stated) * If seropositive for HIV, HCV or HBV, nucleic acid quantitation must be performed. Viral load must be undetectable * HIV-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial * Meets institutional and federal requirements for infectious disease titer requirements * Note: Infectious disease testing to be performed within 28 days prior to day 1 of protocol therapy * Women of childbearing potential (WOCBP): Negative urine or serum pregnancy test (within 35 days prior to day 1 of protocol therapy unless otherwise stated) * If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required * 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: * Allogeneic stem cell transplant or autologous HCT within 1 year prior to day 1 of protocol therapy * Chemotherapy, radiation therapy, biological therapy, immunotherapy within 14 days of day 1 of protocol therapy * Note: Low dose chemotherapy or maintenance chemotherapy given within 7 days of planned study enrollment is permitted. These include hydroxyurea, 6-meraptopurine, oral methotrexate, vincristine, oral etoposide, and tyrosine kinase inhibitors (TKIs). TKIs can also be given up to 3-5 days before conditioning regimen * More than three previous lines of intensive chemotherapy, where the regimen intent was to induce remission * Co-enrollment in other clinical trials involving post-HCT maintenance interventions or any study with potential to affect disease-free survival is not allowed * History of allergic reactions attributed to compounds of similar chemical or biologic composition to study agent * Clinically significant uncontrolled illness * Active infection not responding to antibiotics * Other active malignancy. Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial * 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