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RecruitingNCT06013423Updated Jul 22, 2026

Cord Blood Transplant, Cyclophosphamide, Fludarabine, and Total-Body Irradiation in Treating Patients With High-Risk Hematologic Diseases

A Phase 2 interventional study of Biospecimen Collection and Bone Marrow Aspirate in Acute Leukemia of Ambiguous Lineage, Acute Lymphoblastic Leukemia and Acute Myeloid Leukemia, sponsored by Fred Hutchinson Cancer Center. Recruiting at 1 site in United States. Open to participants aged 6 Months to 65 Years. Per ClinicalTrials.gov, last updated 2026-07-22.

Sponsored by Fred Hutchinson Cancer Center · Phase 2, Interventional, and Treatment

From the registry’s dates

  • Started Jul 2024; still recruiting 2 years 2 months later.
Phase
Phase 2
Study type
Interventional
Enrollment
54
Allocation
Non-randomized
Ages
6 Months to 65 Years
Sex
All
01

Study summary

This phase II trial studies how well giving an umbilical cord blood transplant together with cyclophosphamide, fludarabine, and total-body irradiation (TBI) works in treating patients with hematologic diseases. Giving chemotherapy, such as cyclophosphamide, fludarabine and thiotepa, and TBI before a donor cord blood transplant (CBT) helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil after transplant may stop this from happening in patients with high-risk hematologic diseases.

Read the detailed description

OUTLINE: Patients are assigned to 1 of 2 arms.

ARM I: Patients aged 6 months through 30 years old receive myeloablative conditioning comprising fludarabine intravenously (IV) over 30 minutes on days -8 to -6, cyclophosphamide IV on days -7 and -6, and undergo high-dose TBI twice daily (BID) on days -4 to -1. Patients then undergo UCBT on day 0. Patients undergo blood sample collection throughout the study. Patients undergo echocardiography (ECHO) or multigated acquisition scan (MUGA) and diagnostic imaging during screening and as clinically indicated on study. Patients also undergo blood sample collection throughout the study and bone marrow aspirate during screening and on study.

ARM II: Patients aged 6 months through 65 years old receive myeloablative conditioning comprising fludarabine IV over 30-60 minutes on days -6 to -2, cyclophosphamide IV on day -6, thiotepa IV over 2-4 hours on days -5 and -4, and middle-intensity TBI once daily (QD) on days -2 and -1. Patients undergo ECHO or MUGA and diagnostic imaging during screening and as clinically indicated on study. Patients also undergo blood sample collection throughout the study and bone marrow aspirate during screening and on study.

All patients receive GVHD prophylaxis comprising cyclosporine IV over 1 hour every 8 or 12 hours, then cyclosporine orally (PO) (if tolerated), on days -3 to 100 with taper on day 101. Patients also receive mycophenolate mofetil IV every 8 hours on days 0 to 7 and then PO (if tolerated) three times daily (TID) on days 8-30. Mycophenolate mofetil is tapered to BID on day 30 or 7 days after engraftment if there is no acute GVHD, and then tapered over 2-3 weeks beginning on day 45 (or 15 days after engraftment if engraftment occurred > day 30) after engraftment if there continues to be no evidence of acute GVHD.

After completion of study treatment, patients are followed up at day 180, 1 year, and 2 years.

02

Conditions studied

  • Acute Leukemia of Ambiguous Lineage
  • Acute Lymphoblastic Leukemia
  • Acute Myeloid Leukemia
  • Blastic Plasmacytoid Dendritic Cell Neoplasm
  • Hematopoietic and Lymphatic System Neoplasm
  • Mixed Phenotype Acute Leukemia
  • Myelodysplastic Syndrome
  • Myeloproliferative Neoplasm
  • Non-Hodgkin Lymphoma
  • Chronic Myeloid Leukemia, BCR-ABL1 Positive
03

In context

Leukemia, Biphenotypic, Acute

110 studies on the registry are indexed under Leukemia, Biphenotypic, Acute; 51 are open to participants now.

This study's planned enrollment of 54 is close to the median of 50 across 98 interventional studies indexed under Leukemia, Biphenotypic, Acute.

Browse Leukemia, Biphenotypic, Acute studies →

Lead sponsor

Fred Hutchinson Cancer Center is the lead sponsor of 537 studies on the registry; 79 are open to participants now.

Of its 57 completed or terminated interventional studies of FDA-regulated products, 45 (79%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
6 Months to 65 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Patients aged 6 months to =\< 65 years at time of consent.

  • Acute myelogenous leukemia (AML):

    • Complete first remission (CR1), complete second remission (CR2) or greater (CR2+), must have \< 5% marrow blasts at the time of transplant.
    • Patients in morphologic remission with persistent cytogenetic, flow cytometric, or molecular aberrations are eligible.

  • Acute lymphoblastic leukemia (ALL):

    • Complete first remission (CR1) at high risk for relapse such as any of the following:

      • Presence of any high-risk cytogenetic abnormalities such as t(9;22), t(1;19), t(4;11) or other MLL rearrangements (11q23) or other high-risk molecular abnormality.
      • Failure to achieve MRD- complete remission after induction therapy.
      • Persistence or recurrence of minimal residual disease on therapy.
      • Any patient unable to tolerate consolidation and/or maintenance chemotherapy as would have been deemed appropriate by the treating physician.
      • Other high-risk features not defined above.

    • Complete second remission (CR2) or greater (CR2+).

      • Note: ALL with less than 5% blasts at time of transplant but persistent cytogenetic, flow cytometric or molecular aberrations are eligible.
  • Other acute leukemias: Acute leukemias of ambiguous lineage or mixed phenotype with less than 5% blasts. Leukemias in morphologic remission with persistent cytogenetic, flow cytometric or molecular aberrations are eligible.
  • Chronic Myeloid Leukemia (CML): Excluding refractory blast crisis. To be eligible in first chronic phase (CP1) patient must have failed or be intolerant to tyrosine kinase inhibitor therapy.

  • Myelodysplastic syndromes (MDS) and myeloproliferative disorders (MPD) other than myelofibrosis:

    • MDS/MPD overlap syndromes without myelofibrosis.
    • MDS/ MPD patients must have less than 10% bone marrow myeloblasts and absolute neutrophil count (ANC) > 0.2 (growth factor supported if necessary) at transplant work-up.

  • Non-Hodgkin lymphoma (NHL) at high-risk of relapse or progression if not in remission:

    • Eligible patients with aggressive histology (such as, but not limited to, diffuse large B-cell NHL, mantle cell NHL, and T-cell histology) in CR by PET/CT imaging.
    • Eligible patients with indolent B-cell NHL (such as, but not limited to, follicular, small cell or marginal zone NHL) will have 2nd or subsequent progression with PR or CR by PET/CT imaging.
  • Blastic plasmacytoid dendritic cell neoplasm (BPDCN) in morphologic remission.
  • Only for adult patients, to prevent graft rejection, patients who received only non-lymphodepleting agents for their malignancy (hypomethylating agents, venetoclax, hydroxyurea, TKIs etc.), or patients who received lymphodepleting chemotherapy > 3 months prior to scheduled admission, may receive fludarabine 25 mg/m\^2 daily x 3 days for lymphodepletion 14-42 days (aiming for 2-4 weeks) at the discretion of the principal investigator (PI).
  • For patients > 18 years old, Karnofsky score ≥ 70%. For patients =\< 18 years old, Lansky score ≥ 50%.
  • Calculated creatinine clearance > 70 ml/min.
  • Bilirubin \< 1.5 mg/dL (unless benign congenital hyperbilirubinemia or hemolysis).
  • Alanine transaminase (ALT) \< 3 x upper limit of normal (ULN).
  • For patients > 18 years old, pulmonary function (spirometry and corrected diffusing capacity for carbon monoxide [DLCO]) > 60% predicted. For patients =\< 18 years old, or any patient unable to perform pulmonary function tests, O2 saturation > 92% on room air.
  • Left ventricular ejection fraction > 50%.
  • Albumin > 3.0 g/dL.
  • For patients > 18 years old, Hematopoietic Cell Transplantation Comorbidity index (HCT-CI) =\< 5.
  • UCB units will be selected according to current umbilical cord blood graft selection algorithm. One or two UCB units may be used to achieve the required cell dose.
  • The UCB graft is matched at 4-6 HLA-A, B, DRB1 antigens with the recipient. This may include 0-2 antigen mismatches at the A or B or DRB1 loci. Unit selection based on cryopreserved nucleated cell dose and HLA-A, B, DRB1 using intermediate resolution A, B antigen and DRB1 allele typing.

Exclusion criteria

Exclusion Criteria:

  • Diagnosis of myelofibrosis or other malignancy with moderate-severe bone marrow fibrosis.
  • Patients persistent with central nervous system (CNS) involvement in cerebrospinal fluid (CSF) or CNS imaging at time of screening0
  • Prior checkpoint inhibitors/ blockade in the last 12 months.
  • Two prior stem cell transplants of any kind.
  • One prior autologous stem cell transplant within the preceding 12 months.
  • Prior allogeneic transplantation.
  • Prior involved field radiation therapy that would preclude safe delivery of 400cGy total body irradiation (TBI) in the opinion of radiation oncology.
  • Active and uncontrolled infection at time of transplantation.
  • HIV infection.
  • Inadequate performance status/ organ function.
  • Pregnancy or breast feeding.
  • Patient or guardian unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, long-term follow-up, and research tests.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Single group
Masking
None (open label)
Enrollment
54 participants (estimated)

Study arms

  • Experimental
    Arm I (myeloablative UCBT)

    See detailed description.

    Procedure: Biospecimen Collection · Procedure: Bone Marrow Aspirate · Drug: Cyclophosphamide · Drug: Cyclosporine · Procedure: Diagnostic Imaging · Procedure: Echocardiography · Drug: Fludarabine Phosphate · Procedure: Multigated Acquisition Scan · Drug: Mycophenolate Mofetil · Other: Survey Administration · Radiation: Total-Body Irradiation · Procedure: Umbilical Cord Blood Transplantation

  • Experimental
    Arm II (myeloablative UCBT)

    See detailed description.

    Procedure: Biospecimen Collection · Procedure: Bone Marrow Aspirate · Drug: Cyclophosphamide · Drug: Cyclosporine · Procedure: Diagnostic Imaging · Procedure: Echocardiography · Drug: Fludarabine Phosphate · Procedure: Multigated Acquisition Scan · Drug: Mycophenolate Mofetil · Other: Survey Administration · Drug: Thiotepa · Radiation: Total-Body Irradiation · Procedure: Umbilical Cord Blood Transplantation

Interventions

  • ProcedureBiospecimen Collection

    Undergo blood sample collection

    Also known as: Biological Sample Collection, Biospecimen Collected, Specimen Collection

  • ProcedureBone Marrow Aspirate

    Undergo bone marrow aspirate

    Also known as: BONE MARROW, LIQUID, Human Bone Marrow Aspirate

  • DrugCyclophosphamide

    Receive IV

    Also known as: (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Carloxan, Ciclofosfamida, Ciclofosfamide, Cicloxal, Clafen, Claphene, CP monohydrate, CTX, CYCLO-cell, Cycloblastin, Cycloblastine, Cyclophospham, Cyclophosphamid monohydrate, Cyclophosphamide Monohydrate, Cyclophosphamidum, Cyclophosphan, Cyclophosphane, Cyclophosphanum, Cyclostin, Cyclostine, Cytophosphan, Cytophosphane, Cytoxan, Fosfaseron, Genoxal, Genuxal, Ledoxina, Mitoxan, Neosar, Revimmune, Syklofosfamid, WR- 138719, Asta B 518, B-518, WR-138719

  • DrugCyclosporine

    Receive IV or PO

    Also known as: 27-400, Ciclosporin, CsA, Cyclosporin, Cyclosporin A, Cyclosporine Modified, Gengraf, Neoral, OL 27-400, Sandimmune, SangCya

  • ProcedureDiagnostic Imaging

    Undergo diagnostic imaging

    Also known as: Medical Imaging

  • ProcedureEchocardiography

    Undergo ECHO

    Also known as: EC

  • DrugFludarabine Phosphate

    Receive IV

    Also known as: 2-F-ara-AMP, 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-, Beneflur, Fludara, SH T 586

  • ProcedureMultigated Acquisition Scan

    Undergo MUGA

    Also known as: Blood Pool Scan, Equilibrium Radionuclide Angiography, Gated Blood Pool Imaging, Gated Heart Pool Scan, MUGA, MUGA Scan, Multi-Gated Acquisition Scan, Radionuclide Ventriculogram Scan, Radionuclide Ventriculography, RNVG, SYMA Scanning, Synchronized Multigated Acquisition Scanning

  • DrugMycophenolate Mofetil

    Receive IV

    Also known as: CellCept, MMF

  • OtherSurvey Administration

    Ancillary studies

  • DrugThiotepa

    Receive IV

    Also known as: 1,1',1''-Phosphinothioylidynetrisaziridine, Girostan, N,N', N''-Triethylenethiophosphoramide, Oncotiotepa, STEPA, Tepadina, TESPA, Tespamin, Tespamine, Thio-Tepa, Thiofosfamide, Thiofozil, Thiophosphamide, Thiophosphoramide, Thiotef, Tifosyl, TIO TEF, Tio-tef, Triethylene Thiophosphoramide, Triethylenethiophosphoramide, Tris(1-aziridinyl)phosphine sulfide, TSPA, WR 45312, SH105

  • RadiationTotal-Body Irradiation

    Undergo high-dose or middle-intensity TBI

    Also known as: SCT_TBI, TBI, Total Body Irradiation, Whole Body, Whole Body Irradiation, Whole-Body Irradiation

  • ProcedureUmbilical Cord Blood Transplantation

    Undergo UCBT

    Also known as: Cord Blood, Cord Blood Transplantation, UCB transplantation, UMBILICAL CORD BLOOD TRANSPLANT

06

What researchers measure

Primary outcomes

  1. Overall survival

    Will be assessed after optimized cord blood transplant (CBT) in adults and children with hematologic malignancies. Will be calculated using the Kaplan-Meier method.

    Time frame: At 1 year

Secondary outcomes

  1. Cumulative incidence of neutrophil and platelet engraftment

    Will be calculated within the competing risks framework considering death without neutrophil or platelet recovery, respectively, as completing events

    Time frame: Up to 1 year

  2. Incidences of graft failure

    Will be calculated within the competing risks framework considering death without engraftment before day 21 as a competing event.

    Time frame: Up to 1 year

  3. Incidence of grade II-IV and III-IV acute graft-versus-host disease (aGVHD)

    Will be calculated within the competing risks framework considering relapse/ death without developing GVHD, death in the absence of relapse, and relapse as competing events, respectively.

    Time frame: At day 100

  4. Incidence of grade II-IV and III-IV aGVHD

    Will be calculated within the competing risks framework considering relapse/ death without developing GVHD, death in the absence of relapse, and relapse as competing events, respectively.

    Time frame: At day 180

  5. Incidence of chronic graft-versus-host disease (cGVHD)

    Will be calculated within the competing risks framework considering relapse/ death without developing GVHD, death in the absence of relapse, and relapse as competing events, respectively.

    Time frame: At 1, 2 and 3 years

  6. Organ distribution of GVHD

    Will be calculated within the competing risks framework considering death without neutrophil or platelet recovery, respectively, as completing events

    Time frame: Up to 1 year

  7. Incidence of adverse events

    Will be assessed using Common Terminology Criteria for Adverse Events version 5.0 (CTCAE v 5.0).

    Time frame: Up to 1 year

  8. Time to immunosuppression cessation

    Will be assessed using CTCAE v 5.0.

    Time frame: Up to 1 year

  9. Pattern of donor chimerism

    Will be assessed using CTCAE v 5.0.

    Time frame: Up to 1 year

  10. Incidence of pre-engraftment syndrome (PES)

    Will be assessed using CTCAE v 5.0.

    Time frame: Up to 1 year

  11. Incidence of transplant related mortality (TRM)

    Time frame: At 100 days, 6 months, 1 and 2 years

  12. Incidence of relapse

    Time frame: At 1, and 2 years after CBT

07

Study locations

1 of 1 sites recruiting
  • Fred Hutch/University of Washington Cancer Consortium
    Seattle, Washington 98109, United States
    Recruiting
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jul 22, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT06013423
Lead sponsor
Fred Hutchinson Cancer Center
Collaborators
National Cord Blood Network
Responsible party
Sponsor
First posted
Aug 28, 2023
Start date
Jul 23, 2024
Primary completion
Oct 31, 2031 (estimated)
Completion
Oct 31, 2032 (estimated)
Last update
Jul 22, 2026

Study contacts

Ann Dahlberg
Contact
adahlber@fredhutch.org
206-667-1959
Ann Dahlberg
principal investigator · Fred Hutch/University of Washington Cancer Consortium

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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