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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

18. august 2026 oppdatert av: 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.

Studieoversikt

Detaljert beskrivelse

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.

Studietype

Intervensjonell

Registrering (Antatt)

35

Fase

  • Fase 2

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

    • California
      • Duarte, California, Forente stater, 91010
        • Rekruttering
        • City of Hope Medical Center
        • Ta kontakt med:
        • Hovedetterforsker:
          • Monzr M. Al Malki

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Voksen
  • Eldre voksen

Tar imot friske frivillige

Nei

Beskrivelse

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)

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Intervensjonsmodell: Enkeltgruppeoppdrag
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: 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.

Hjelpestudier
Gjennomgå CT
Andre navn:
  • CT
  • KATT
  • CAT-skanning
  • Beregnet aksial tomografi
  • Datastyrt aksialtomografi
  • Datastyrt tomografi
  • CT skann
  • tomografi
  • Datastyrt aksial tomografi (prosedyre)
  • Datastyrt tomografi (CT) skanning
  • Diagnostisk CAT -skanning
  • Diagnostisk CAT -skannertype
Gitt IV
Andre navn:
  • CB-3025
  • L-PAM
  • L-Sarcolysin
  • Alanin nitrogen sennep
  • L-fenylalanin sennep
  • L-Sarcolysin Fenylalanin sennep
  • L-sarkolysin
  • Melphalanum
  • Fenylalanin sennep
  • Fenylalanin nitrogen sennep
  • Sarkoklorin
  • Sarkolysin
  • WR-19813
  • Melphalan for injeksjon-leverleveringssystem
Gjennomgå MUGA
Andre navn:
  • Skanning av blodbasseng
  • Likevektsradionuklidangiografi
  • Gated Blood Pool Imaging
  • MUGA
  • Radionuklid ventrikulografi
  • RNVG
  • SYMA-skanning
  • Synchronized Multigated Acquisition Scanning
  • MUGA Scan
  • Multi-Gated Acquisition Scan
  • Radionuklid ventrikulogramskanning
  • Gated Heart Pool Scan
  • RNV Scan
Gitt IV
Andre navn:
  • 2-F-ara-AMP
  • Beneflur
  • Fludara
  • 9H-Purin-6-amin, 2-fluor-9-(5-O-fosfono-P-D-arabinofuranosyl)-
  • SH T 586
Gitt IV eller PO
Andre navn:
  • Prograf
  • FK506
  • Hecoria
  • FK 506
  • Fujimycin
  • Protopic
  • FK-506
  • Tacforius
Gitt IV
Andre navn:
  • Vekstfaktor, rekombinant human keratinocytt
  • Kepivance
  • Keratinocyttvekstfaktor, rekombinant menneske
  • Rekombinant human keratinocyttvekstfaktor
  • rhKGF
  • rhu Keratinocytt-vekstfaktor
Gjennomgå benmargsbiopsi
Andre navn:
  • Biopsi av benmarg
  • Biopsi, benmarg
Gjennomgå TMLI
Andre navn:
  • TMLI
Gitt sirolimus
Andre navn:
  • Rapamycin
  • Rapamune
  • AY 22989
  • RAPA
  • SILA 9268A
  • WY-090217
  • AY-22989
  • AY22989
  • SILA-9268A
  • SILA9268A
  • WY 090217
  • WY090217
Gjennomgå urin- og blodprøvetaking
Andre navn:
  • Biologisk prøvesamling
  • Bioprøve samlet
  • Prøvesamling
Gjennomgå ekkokardiografi
Andre navn:
  • Ekkokardiografi
  • EC
Undergo allogeneic PBSC HCT
Andre navn:
  • PBPC-transplantasjon
  • Perifer blodprogenitorcelletransplantasjon
  • Perifer stamcellestøtte
  • Perifer stamcelletransplantasjon
  • PBSCT
  • TRANSPLANTERING AV PERIFER BLODSTAMCELLE
  • Perifert blod

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Leukemia-free survival
Tidsramme: 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)

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Incidence of adverse events (AEs)
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Samarbeidspartnere

Etterforskere

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

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Antatt)

5. april 2027

Primær fullføring (Antatt)

5. april 2029

Studiet fullført (Antatt)

5. april 2029

Datoer for studieregistrering

Først innsendt

6. mai 2026

Først innsendt som oppfylte QC-kriteriene

6. mai 2026

Først lagt ut (Faktiske)

12. mai 2026

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

20. august 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

18. august 2026

Sist bekreftet

1. august 2026

Mer informasjon

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

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