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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

6. května 2026 aktualizováno: City of Hope Medical Center

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

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.

Přehled studie

Detailní popis

PRIMARY OBJECTIVE:

I. Assess the efficacy of the total marrow and lymphoid irradiation (TMLI)-based therapy at the recommended phase 2 dose (RP2D) of 1600 cGy prior to hematopoietic cell transplant (HCT) for older patients (≥ 50 years of age) with refractory and relapsed acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) undergoing peripheral blood stem cell (PBSC) HCT from matched related/unrelated donor, as measured by 2-years leukemia-free survival (LFS).

SECONDARY OBJECTIVES:

I. Further evaluation of safety of the TMLI-based conditioning regimen, by assessing the following: type, frequency, severity, attribution, time course and duration of adverse events, including acute/chronic GVHD, infection and delayed engraftment.

II. Estimate overall survival (OS: at 1 and 2 years post-HCT), cumulative incidence (CI) of relapse/progression (at 1 and 2 years post-HCT), and non-relapse mortality (NRM) at 100 days, 1 year and 2 years post-HCT.

III. Estimate the cumulative incidence and severity of acute graft-versus-host disease (GVHD) by day 180 using Malignant Germ Cell International Consortium (MAGIC) grading and chronic GVHD by 1 and 2 years post-HCT using National Institutes of Health (NIH) consensus criteria.

IV. Estimate the cumulative incidence of GVHD-free and relapse-free survival (GRFS) at 1-year post-HCT.

EXPLORATORY OBJECTIVES:

I. 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 bone marrow samples to assess changes in the bone marrow environment after TMLI.

IV. Collect longitudinal blood samples for circulating tumor DNA (ctDNA) profiling.

V. Collect longitudinal stool samples to explore the potential effects of lower gastrointestinal (GI) tract radiation exposure on microbiome composition and HCT outcomes.

VI. In patients ≥ 50 years old, evaluate physical function and quality of life and cognitive impairment using Cancer Health Assessments Reaching Many (CHARM) assessments at baseline then frailty assessments, Patient Reported Outcomes Measurement Information Systems (PROMIS) Physical Function and Montreal cognitive assessment on day 100 and 180-, and 1-year post-HCT.

OUTLINE:

Patients receive palifermin intravenously (IV) on days -11, -10, -9, 0, 1 and 2, fludarabine IV on days -4 to -2 and melphalan IV on day -2 and undergo TMLI twice daily (BID) for 8 fractions on days -8 to -5. Patients receive allogeneic PBSC-HCT on day 0. Starting on day -1, patients also receive tacrolimus IV or orally (PO) once daily (QD) and sirolimus QD per standard of care.

Additionally, patients undergo echocardiography or multigated acquisition scan (MUGA), computed tomography (CT), urine and blood sample collection, and bone marrow biopsy throughout the study.

After completion of study treatment, patients are followed up at 30, 60, 100 and 180 days and at 1 and 2 years.

Typ studie

Intervenční

Zápis (Odhadovaný)

35

Fáze

  • Fáze 2

Kontakty a umístění

Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.

Studijní místa

    • California
      • Duarte, California, Spojené státy, 91010
        • City of Hope Medical Center
        • Kontakt:
        • Vrchní vyšetřovatel:
          • Monzr M. Al Malki

Kritéria účasti

Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.

Kritéria způsobilosti

Věk způsobilý ke studiu

  • Dospělý
  • Starší dospělý

Přijímá zdravé dobrovolníky

Ne

Popis

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)

Studijní plán

Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.

Jak je studie koncipována?

Detaily designu

  • Primární účel: Léčba
  • Přidělení: N/A
  • Intervenční model: Přiřazení jedné skupiny
  • Maskování: Žádné (otevřený štítek)

Zbraně a zásahy

Skupina účastníků / Arm
Intervence / Léčba
Experimentální: Treatment (TMLI, fludarabine, melphalan, allogeneic PBSC-HCT)

Patients receive palifermin IV on days -11, -10, -9, 0, 1 and 2, fludarabine IV on days -4 to -2 and melphalan IV on day -2 and undergo TMLI BID for 8 fractions on days -8 to -5. Patients receive allogeneic PBSC-HCT on day 0. Starting on day -1, patients also receive tacrolimus IV or PO QD and sirolimus QD per standard of care.

Additionally, patients undergo echocardiography or MUGA, CT, urine and blood sample collection, and bone marrow biopsy throughout the study.

Pomocná studia
Podstoupit ČT
Ostatní jména:
  • ČT
  • KOČKA
  • CAT skenování
  • Počítačová axiální tomografie
  • Počítačová tomografie
  • CT vyšetření
  • tomografie
  • Počítačová axiální tomografie (postup)
  • Počítačová tomografie (CT).
  • Diagnostické skenování CAT
  • Typ služby diagnostického skenování CAT
Vzhledem k tomu, IV
Ostatní jména:
  • CB-3025
  • L-PAM
  • L-Sarcolysin
  • Alaninová dusíkatá hořčice
  • L-fenylalanin hořčice
  • L-Sarcolysin Fenylalanin hořčice
  • L-Sarkolysin
  • Melphalanum
  • Fenylalaninová hořčice
  • Fenylalanin dusíkatá hořčice
  • Sarcoclorin
  • Sarkolysin
  • WR-19813
  • Melfalan pro injekčně-hepatální aplikační systém
Podstoupit MUGA
Ostatní jména:
  • Skenování bazénu krve
  • Rovnovážná radionuklidová angiografie
  • Gated Blood Pool Imaging
  • MUGA
  • Radionuklidová ventrikulografie
  • RNVG
  • Skenování SYMA
  • Synchronizované multigated Acquisition Scanning
  • Skenování MUGA
  • Multi-Gated Acquisition Scan
  • Radionuklidový Ventrikulogram Scan
  • Gated Heart Pool Scan
  • Skenování RNV
Vzhledem k tomu, IV
Ostatní jména:
  • 2-F-ara-AMP
  • Beneflur
  • Fludara
  • 9H-purin-6-amin, 2-fluor-9-(5-0-fosfono-p-D-arabinofuranosyl)-
  • SH T 586
Vzhledem k IV nebo PO
Ostatní jména:
  • Prograf
  • FK506
  • Hecoria
  • FK 506
  • Fujimycin
  • Protopic
  • FK-506
  • Tacforius
Vzhledem k tomu, IV
Ostatní jména:
  • Růstový faktor, Rekombinantní lidský keratinocyt
  • Kepivance
  • Keratinocytový růstový faktor, rekombinantní člověk
  • Rekombinantní lidský keratinocytový růstový faktor
  • rhKGF
  • rhu keratinocytový růstový faktor
Podstoupit biopsii kostní dřeně
Ostatní jména:
  • Biopsie kostní dřeně
  • Biopsie, kostní dřeň
Podstoupit TMLI
Ostatní jména:
  • TMLI
Podáno sirolimus
Ostatní jména:
  • Rapamycin
  • Rapamune
  • AY 22989
  • RAPA
  • SILA 9268A
  • WY-090217
  • AY-22989
  • AY22989
  • SILA-9268A
  • SILA9268A
  • WY 090217
  • WY090217
Podstoupit odběr moči a krve
Ostatní jména:
  • Sběr biologických vzorků
  • Odebrán biovzorek
  • Sbírka vzorků
Podstoupit echokardiografii
Ostatní jména:
  • Echokardiografie
  • EC
Undergo allogeneic PBSC HCT
Ostatní jména:
  • Transplantace PBPC
  • Transplantace progenitorových buněk periferní krve
  • Podpora periferních kmenových buněk
  • Transplantace periferních kmenových buněk
  • PBSCT
  • TRANSPLANTACE KMENOVÝCH BUNĚK PERIFERNÍ KRVE
  • Periferní krev

Co je měření studie?

Primární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Leukemia-free survival
Časové okno: From the date of stem cell infusion to the date of first observation of relapse/progression, or date of death, whichever comes first, assessed at 2 years post-hematopoietic cell transplantation (HCT)
Will be calculated using the Kaplan-Meier method. Point estimates and 95% confidence intervals will be provided.
From the date of stem cell infusion to the date of first observation of relapse/progression, or date of death, whichever comes first, assessed at 2 years post-hematopoietic cell transplantation (HCT)

Sekundární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Incidence of adverse events (AEs)
Časové okno: From day 1 of protocol therapy up to day 30 post-HCT
Will be scored on both the Bearman Scale and National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version (v) 5.0. Will be summarized by type, attribution, grade and duration.
From day 1 of protocol therapy up to day 30 post-HCT
Incidence of highest grades of AEs
Časové okno: From day 31 up to day 100 post-HCT
Will be scored on both the Bearman Scale and NCI CTCAE v 5.0. Will be summarized by type, attribution, grade and duration.
From day 31 up to day 100 post-HCT
Overall survival
Časové okno: From date of stem cell infusion t the date of death, assessed at 1 and 2 years post-HCT
Will be calculated using the Kaplan-Meier method. Point estimates and 95% confidence intervals will be provided.
From date of stem cell infusion t the date of death, assessed at 1 and 2 years post-HCT
Relapse
Časové okno: From date of stem cell infusion to first observation of relapse/progression, assessed at 1 and 2 years post-HCT
The cumulative incidence of will be calculated using the competing risk method as described by Gooley et al. (1999). Point estimates and 95% confidence intervals will be provided for each outcome measure.
From date of stem cell infusion to first observation of relapse/progression, assessed at 1 and 2 years post-HCT
Non-relapse mortality
Časové okno: From date of stem cell infusion until non-disease related death, assessed at 100 days, and at 1 and 2 years post-HCT
The cumulative incidence of will be calculated using the competing risk method as described by Gooley et al. (1999). Point estimates and 95% confidence intervals will be provided for each outcome measure.
From date of stem cell infusion until non-disease related death, assessed at 100 days, and at 1 and 2 years post-HCT
Acute graft-versus-host disease (GVHD)
Časové okno: From date of stem cell infusion to document/biopsy proven acute GVHD onset date, assessed up to 180 days post-transplant
Will be graded according to the 1994 Keystone Consensus Grading. The cumulative incidence of will be calculated using the competing risk method as described by Gooley et al. (1999). Point estimates and 95% confidence intervals will be provided for each outcome measure.
From date of stem cell infusion to document/biopsy proven acute GVHD onset date, assessed up to 180 days post-transplant
Chronic GVHD
Časové okno: From approximately 80-100 days post-transplant to the documented/biopsy proven chronic GVHD onset date, assessed at 1 and 2 years
Will be scored according to Jagasia et al. The cumulative incidence of will be calculated using the competing risk method as described by Gooley et al. (1999). Point estimates and 95% confidence intervals will be provided for each outcome measure.
From approximately 80-100 days post-transplant to the documented/biopsy proven chronic GVHD onset date, assessed at 1 and 2 years
GVHD-free and relapse-free survival
Časové okno: From the date of stem cell infusion to garde 3-4 acute GVHD, chronic GVHD requiring systemic treatment, relapse/progression, whichever comes first, assessed at 1 year post-HCT
Will be calculated using the Kaplan-Meier method. Point estimates and 95% confidence intervals will be provided.
From the date of stem cell infusion to garde 3-4 acute GVHD, chronic GVHD requiring systemic treatment, relapse/progression, whichever comes first, assessed at 1 year post-HCT

Spolupracovníci a vyšetřovatelé

Zde najdete lidi a organizace zapojené do této studie.

Vyšetřovatelé

  • Vrchní vyšetřovatel: Monzr M Al Malki, City of Hope Medical Center

Termíny studijních záznamů

Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.

Hlavní termíny studia

Začátek studia (Odhadovaný)

5. dubna 2027

Primární dokončení (Odhadovaný)

5. dubna 2029

Dokončení studie (Odhadovaný)

5. dubna 2029

Termíny zápisu do studia

První předloženo

6. května 2026

První předloženo, které splnilo kritéria kontroly kvality

6. května 2026

První zveřejněno (Aktuální)

12. května 2026

Aktualizace studijních záznamů

Poslední zveřejněná aktualizace (Aktuální)

12. května 2026

Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality

6. května 2026

Naposledy ověřeno

1. května 2026

Více informací

Termíny související s touto studií

Informace o lécích a zařízeních, studijní dokumenty

Studuje lékový produkt regulovaný americkým FDA

Ano

Studuje produkt zařízení regulovaný americkým úřadem FDA

Ne

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