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Acute Leukemia of Ambiguous LineagePhase 2NCT06013423

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

A Phase 2 study testing Biospecimen Collection, Bone Marrow Aspirate and Cyclophosphamide in people with Acute Leukemia of Ambiguous Lineage and Acute Lymphoblastic Leukemia, run by Fred Hutchinson Cancer Center at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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. Read more about this study

Who is it for?
Ages 6 months to 65 years
What is being tested?
Biospecimen Collection, Bone Marrow Aspirate and Cyclophosphamide
Could I receive a placebo?
No, everyone receives an active treatment
How many people
54 participants
Where
1 siteUnited States
Study dates
23 Jul 2024to 31 Oct 2032
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 months to 65 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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:
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  • 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 > 16 years old, Karnofsky score ≥ 70%. For patients =< 16 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.

Who cannot join

  • 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.
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  • 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.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Ann Dahlberg contact

206-667-1959

adahlber@fredhutch.org

United States1 site, 1 recruiting

  • Fred Hutch/University of Washington Cancer Consortium

    Seattle, Washington, 98109

    Ann Dahlberg, 206-667-1959, adahlber@fredhutch.org

    Ann Dahlberg

    Recruiting

Other trials in United States

About this study

Official title: Optimized Cord Blood Transplantation for the Treatment of High-Risk Hematologic Malignancies in Adults and Pediatrics

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 then undergo UCBT on day 0. 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 14 and then PO (if tolerated) three times daily (TID) on days 15-30. 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.

Being tested
Biospecimen Collection, Bone Marrow Aspirate, Cyclophosphamide, Cyclosporine, Diagnostic Imaging Testing
Medical subject headings
Leukemia, Biphenotypic, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute, Blastic Plasmacytoid Dendritic Cell Neoplasm, Myelodysplastic Syndromes, Myeloproliferative Disorders, Lymphoma, Non-Hodgkin, Leukemia, Myelogenous, Chronic, BCR-ABL Positive

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Biospecimen CollectionProcedure or surgery

    Also called Biological Sample Collection, Biospecimen Collected, Specimen Collection

    Undergo blood sample collection

  • Bone Marrow AspirateProcedure or surgery

    Also called BONE MARROW, LIQUID, Human Bone Marrow Aspirate

    Undergo bone marrow aspirate

  • CyclophosphamideDrug

    Also called (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Carloxan, Ciclofosfamida

    Given IV

  • CyclosporineDrug

    Also called 27-400, Ciclosporin, CsA, Cyclosporin

    Given IV or PO

  • Diagnostic Imaging TestingProcedure or surgery

    Also called Diagnostic Imaging, Medical Imaging

    Undergo diagnostic imaging

  • Echocardiography TestProcedure or surgery

    Also called EC, Echocardiography

    Undergo ECHO

  • Fludarabine PhosphateDrug

    Also called 2-F-ara-AMP, 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-, Beneflur, Fludara

    Given IV

  • Multigated Acquisition ScanProcedure or surgery

    Also called Blood Pool Scan, Equilibrium Radionuclide Angiography, Gated Blood Pool Imaging, Gated Heart Pool Scan

    Undergo MUGA

  • Mycophenolate MofetilDrug

    Also called CellCept, MMF

    Given IV

  • Survey AdministrationOther

    Ancillary studies

  • ThiotepaDrug

    Also called 1,1',1''-Phosphinothioylidynetrisaziridine, Girostan, N,N', N''-Triethylenethiophosphoramide, Oncotiotepa

    Given IV

  • Total-Body IrradiationRadiation

    Also called SCT_TBI, TBI, Total Body Irradiation, Whole Body

    Undergo high-dose or middle-intensity TBI

  • Umbilical Cord Blood TransplantationProcedure or surgery

    Also called Cord Blood, Cord Blood Transplantation, UCB transplantation, UMBILICAL CORD BLOOD TRANSPLANT

    Undergo UCBT

Study groups in NCT06013423
Study groupRoleWhat they receive
Arm I (myeloablative UCBT)ExperimentalSee detailed description.
Receives: Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspirate; Drug: Cyclophosphamide; Drug: Cyclosporine; Procedure: Diagnostic Imaging Testing; Procedure: Echocardiography Test; Drug: Fludarabine Phosphate; Procedure: Multigated Acquisition Scan; Drug: Mycophenolate Mofetil; Other: Survey Administration; Radiation: Total-Body Irradiation; Procedure: Umbilical Cord Blood Transplantation
Arm II (myeloablative UCBT)ExperimentalSee detailed description.
Receives: Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspirate; Drug: Cyclophosphamide; Drug: Cyclosporine; Procedure: Diagnostic Imaging Testing; Procedure: Echocardiography Test; Drug: Fludarabine Phosphate; Procedure: Multigated Acquisition Scan; Drug: Mycophenolate Mofetil; Other: Survey Administration; Drug: Thiotepa; Radiation: Total-Body Irradiation; Procedure: Umbilical Cord Blood Transplantation

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: At 1 year

Secondary outcomes (12)

  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

    Measured: 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.

    Measured: 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.

    Measured: 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.

    Measured: 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.

    Measured: 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

    Measured: 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).

    Measured: Up to 1 year

  8. Time to immunosuppression cessation

    Will be assessed using CTCAE v 5.0.

    Measured: Up to 1 year

  9. Pattern of donor chimerism

    Will be assessed using CTCAE v 5.0.

    Measured: Up to 1 year

  10. Incidence of pre-engraftment syndrome (PES)

    Will be assessed using CTCAE v 5.0.

    Measured: Up to 1 year

  11. Incidence of transplant related mortality (TRM)

    Measured: At 100 days, 6 months, 1 and 2 years

  12. Incidence of relapse

    Measured: At 1, and 2 years after CBT

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 Aug 2023
  2. Study start23 Jul 2024
  3. Primary completion31 Oct 2031 (estimated)
  4. Study completion31 Oct 2032 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
54 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06013423
Sponsor's protocol ID
RG1123652
Other IDs
NCI-2023-05598, FHIRB0020219
Status last verified
Oct 2026
First submitted
22 Aug 2023
Last updated
26 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Biospecimen Collection, Bone Marrow Aspirate and Cyclophosphamide in people with Acute Leukemia of Ambiguous Lineage and Acute Lymphoblastic Leukemia, run by Fred Hutchinson Cancer Center at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 33 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 26 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Ann Dahlberg (206-667-1959, adahlber@fredhutch.org), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall survival (time frame: At 1 year). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06013423.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Oct 2026.

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