- ICH GCP
- US Clinical Trials Registry
- Klinisk forsøg NCT07582172
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
Studieoversigt
Status
Betingelser
- Myelodysplastisk syndrom
- Tilbagevendende akut myeloid leukæmi
- Tilbagevendende myelodysplastisk syndrom
- Refraktær akut myeloid leukæmi
- Refraktært myelodysplastisk syndrom
- Tilbagevendende sekundær akut myeloide leukæmi
- Ildfast sekundær akut myeloide leukæmi
- Acute Myeloid Leukemia With Complex Karyotype
- Myelodysplastic Chronic Myelomonocytic Leukemia
- Recurrent Myelodysplastic Chronic Myelomonocytic Leukemia
- Refractory Myelodysplastic Chronic Myelomonocytic Leukemia
Intervention / Behandling
- Andet: Spørgeskemaadministration
- Procedure: Computertomografi
- Medicin: Melphalan
- Procedure: Multigated Acquisition Scan
- Medicin: Fludarabin fosfat
- Medicin: Tacrolimus
- Biologisk: Palifermin
- Procedure: Knoglemarvsbiopsi
- Stråling: Total marv og lymfoid bestråling
- Medicin: Sirolimus
- Procedure: Bioprøvesamling
- Procedure: Ekkokardiografitest
- Procedure: Peripheral Blood Stem Cell Transplantation
Detaljeret 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.
Undersøgelsestype
Tilmelding (Anslået)
Fase
- Fase 2
Kontakter og lokationer
Studiesteder
-
-
California
-
Duarte, California, Forenede Stater, 91010
- City of Hope Medical Center
-
Kontakt:
- Monzr M. Al Malki
- Telefonnummer: 626-218-2405
- E-mail: malamalki@coh.org
-
Ledende efterforsker:
- Monzr M. Al Malki
-
-
Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
- Voksen
- Ældre voksen
Tager imod sunde frivillige
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
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: N/A
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
|
Eksperimentel: 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. |
Hjælpestudier
Gennemgå CT
Andre navne:
Givet IV
Andre navne:
Gennemgå MUGA
Andre navne:
Givet IV
Andre navne:
Givet IV eller PO
Andre navne:
Givet IV
Andre navne:
Gennemgå knoglemarvsbiopsi
Andre navne:
Gennemgå TMLI
Andre navne:
Givet sirolimus
Andre navne:
Gennemgå urin- og blodprøvetagning
Andre navne:
Gennemgå ekkokardiografi
Andre navne:
Undergo allogeneic PBSC HCT
Andre navne:
|
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
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 |
Foranstaltningsbeskrivelse |
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
|
Samarbejdspartnere og efterforskere
Sponsor
Samarbejdspartnere
Efterforskere
- Ledende efterforsker: Monzr M Al Malki, City of Hope Medical Center
Datoer for undersøgelser
Studer store datoer
Studiestart (Anslået)
Primær færdiggørelse (Anslået)
Studieafslutning (Anslået)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Faktiske)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
- Neoplasmer
- Neoplasmer efter histologisk type
- Hæmatologiske sygdomme
- Leukæmi, myeloid
- Knoglemarvssygdomme
- Leukæmi
- Hemiske og lymfatiske sygdomme
- Leukæmi, Myeloid, Akut
- Myelodysplastiske syndromer
- Peptider
- Aminosyrer, peptider og proteiner
- Proteiner
- Organiske kemikalier
- Undersøgelsesteknikker
- Terapeutik
- Kliniske laboratorieteknikker
- Diagnostiske teknikker og procedurer
- Diagnose
- Kirurgiske procedurer, operative
- Cytologiske teknikker
- Cytodiagnose
- Kulbrinter
- Biologiske faktorer
- Transplantation
- Aminosyrer
- Diagnostiske teknikker, kirurgisk
- Makrolider
- Lactoner
- Nitrogen sennepsforbindelser
- Sennepsforbindelser
- Kulbrinter, halogeneret
- Intercellulære signalpeptider og proteiner
- Strålebehandling
- Celletransplantation
- Celle- og vævsbaseret terapi
- Biologisk terapi
- Phenylalanin
- Aminosyrer, aromatisk
- Aminosyrer, cykliske
- Stamcelletransplantation
- Fibroblastvækstfaktorer
- Hæmatopoietisk stamcelletransplantation
- Sirolimus
- Melphalan
- Tacrolimus
- Biopsi
- Håndtering af eksemplar
- Fludarabinphosphat
- Fibroblastvækstfaktor 7
- Perifer blodstamcelletransplantation
- Lymfeirradiation
Andre undersøgelses-id-numre
- 25663 (Anden identifikator: City of Hope Medical Center)
- P30CA033572 (U.S. NIH-bevilling/kontrakt)
- NCI-2026-03208 (Registry Identifier: CTRP (Clinical Trial Reporting Program))
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
Studerer et amerikansk FDA-reguleret lægemiddelprodukt
Studerer et amerikansk FDA-reguleret enhedsprodukt
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