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Hematologic MalignancyPhase 2NCT05735717

MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies

A Phase 2 study testing Fludarabine, Busulfan and Melphalan in people with Hematologic Malignancy and Acute Leukemia, run by Masonic Cancer Center, University of Minnesota 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 is a phase II, open-label, prospective study of T cell receptor alpha/beta depletion (TCR α/β TCD) peripheral blood stem cell (PBSC) transplantation for children and adults with hematological malignancies. This is a safety/feasibility study of the investigational procedure/product. Read more about this study

Who is it for?
Ages Up to 60 years
Conditions the study is for
Hematologic Malignancy, Acute Leukemia, Remission
What is being tested?
Fludarabine, Busulfan and Melphalan
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
70 participants
Where
1 siteUnited States
Study dates
11 May 2023to 30 Nov 2030
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 Up to 60 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Histological confirmation of hematological malignancies
  • Acute leukemias
  • Acute Myeloid Leukemia (AML) and related precursor neoplasms
  • Favorable risk AML is defined as having one of the following:
  • Acute lymphoblastic leukemia (ALL)/lymphoma
  • Myelodysplasia (MDS) IPSS INT-2 or High Risk (i.e. RAEB, RAEBt) or Refractory Anemia with severe pancytopenia, transfusion dependence, or high risk cytogenetics or molecular features.
Show all 9Show fewer
  • Age 60 years of age or younger at the time of consent
  • Karnofsky performance status ≥ 70% or Lansky play score 50% for ≤16 years of age.
  • Adequate organ function

Who cannot join

  • Pregnant or breastfeeding.
  • Active uncontrolled infection within 1 week of starting preparative therapy
  • Known seropositive for HIV or known active Hepatitis B or C infection with detectable viral load by PCR.
  • Any prior autologous or allogeneic transplant
  • CML blast crisis
  • Active central nervous system malignancy

Trial locations and contacts

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

Margaret MacMillan contact

612-626-2961

macmi002@umn.edu

United States1 site, 1 recruiting

  • University of Minnesota Masonic Cancer Center

    Minneapolis, Minnesota, 55455

    Margaret MacMillan

    Recruiting

Other trials in United States

About this study

Official title: Phase II, Open-Label, Prospective Study of T Cell Receptor Alpha/Beta Depletion (A/B TCD) Peripheral Blood Stem Cell (PBSC) Transplantation for Children and Adults With Hematological Malignancies

This is a phase II, open-label, prospective study of T cell receptor alpha/beta depletion (TCR α/β TCD) peripheral blood stem cell (PBSC) transplantation for children and adults with hematological malignancies. This is a safety/feasibility study of the investigational procedure/product.

Being tested
Fludarabine, Busulfan, Melphalan, Rituximab, Levetiracetam
Keywords
Bu, Flu, G-CSF, GFSR, aGVHD, HCT, MAC, Mel, PBSCT, PTLD, RECIST, TCR
Medical subject headings
Hematologic Neoplasms, Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Chromosome Aberrations, Neoplasm, Residual, Anemia, Refractory, with Excess of Blasts, Leukemia, Myelomonocytic, Juvenile, Neurofibromatosis 1

What participants receive

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

  • FludarabineDrug

    Fludarabine 25mg/m2 IV on days -8 to -6 or days -4 to -2. 40mg/m2 IV on days -5 to -2.

  • BusulfanDrug

    Busulfan 82.1 mg*hr/L IV on days -5 to -2 or days -8 to -5

  • MelphalanDrug

    Melphalan 50 mg/m2 IV on days -4 to -2

  • RituximabDrug

    200 mg/m2 intravenous given once on day-1

  • LevetiracetamDrug

    Also called Keppra

    As seizures have occurred following high dose busulfan, all patients will be treated with Keppra beginning day -6 and continuing until day -1 per institutional guidelines.

  • Alpha/Beta T Cell-Depleted Hematopoietic Stem CellsBiological

    Patients will be treated on the most medically appropriate regimen followed by an infusion at Day 0 of Alpha/Beta T Cell-Depleted Hematopoietic Stem Cells.

  • ThymoglobulinDrug

    Also called ATG

    rabbit anti-thymocyte globulin (rATG). Used in conditioning regimens for in vivo depletion of T cells, and the use of fludarabine model-based dosing to optimize dosing.

  • CyclophosphamideDrug

    Cyclophosphamide 60 mg/kg IV over 2 hours on days -3 and -2

Study groups in NCT05735717
Study groupRoleWhat they receive
Arm 1A: Fludarabine (flu), Total Body Irradiation (TBI), Flu/TBI Regimen, Closed to AccrualExperimentalPatients will be treated on the most medically appropriate regimen with a preference for Flu/TBI Arm followed by an infusion at Day 0 of Alpha/Beta T Cell-Depleted Hematopoietic Stem Cells.
Receives: Drug: Fludarabine; Drug: Rituximab; Drug: Levetiracetam; Biological: Alpha/Beta T Cell-Depleted Hematopoietic Stem Cells
Arm 2A: Fludarabine (flu), Busulfan (bu), Flu/Bu Regimen, Closed to AccrualExperimentalPatients will be treated on the most medically appropriate regimen with a preference for Flu/TBI Arm followed by an infusion at Day 0 of Alpha/Beta T Cell-Depleted Hematopoietic Stem Cells.
Receives: Drug: Fludarabine; Drug: Busulfan; Drug: Rituximab; Drug: Levetiracetam
Arm 3A: Fludarabine (flu), Busulfan (bu), Melphalan (Mel) Regimen for Pediatric Patients OnlyExperimentalFlu/Bu/Mel will the preference for patients with JMML or infants with leukemia. , Closed to Accrual
Receives: Drug: Fludarabine; Drug: Busulfan; Drug: Melphalan; Drug: Rituximab; Drug: Levetiracetam
Arm 1B: ATG, Fludarabine (flu), Total Body Irradiation (TBI), Flu/TBI RegimenExperimentalPatients will be treated on the most medically appropriate regimen with a preference for ATG/Flu/TBI Arm followed by an infusion at Day 0 of Alpha/Beta T Cell-Depleted Hematopoietic Stem Cells.
Receives: Drug: Fludarabine; Drug: Rituximab; Drug: Levetiracetam; Drug: Thymoglobulin
Arm 2B: ATG, Fludarabine (flu), Busulfan (bu), Flu/Bu RegimenExperimentalPatients will be treated on the most medically appropriate regimen with a preference for ATG/Flu/BU/TBI Arm followed by an infusion at Day 0 of Alpha/Beta T Cell-Depleted Hematopoietic Stem Cells.
Receives: Drug: Fludarabine; Drug: Busulfan; Drug: Rituximab; Drug: Levetiracetam; Drug: Thymoglobulin
Arm 3B: ATG, Fludarabine (flu), Busulfan (bu), Melphalan (Mel) Regimen for Pediatric Patients OnlyExperimentalATG/Flu/Bu/Mel will the preference for patients with JMML or infants with leukemia.
Receives: Drug: Fludarabine; Drug: Busulfan; Drug: Melphalan; Drug: Rituximab; Drug: Levetiracetam; Drug: Thymoglobulin
Arm 4B: ATG, Busulfan (BU), Cyclophosphamide (CY)ExperimentalPatients will be treated on the most medically appropriate regimen with a preference for ATG/BU/CY Arm followed by an infusion at Day 0 of Alpha/Beta T Cell-Depleted Hematopoietic Stem Cells.
Receives: Drug: Busulfan; Drug: Rituximab; Drug: Levetiracetam; Drug: Thymoglobulin; Drug: Cyclophosphamide
Arm 5B: ATG, Cyclophosphamide (CY), Total Body Irradiation (TBI)ExperimentalPatients will be treated on the most medically appropriate regimen with a preference for ATG/CY/TBI Arm followed by an infusion at Day 0 of Alpha/Beta T Cell-Depleted Hematopoietic Stem Cells.
Receives: Drug: Rituximab; Drug: Levetiracetam; Drug: Thymoglobulin; Drug: Cyclophosphamide

What is being measured

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

  1. Determine the rate of GVHD after alpha beta TCR depletion

    GVHD incidence after treatment.

    Measured: 100 days

Secondary outcomes (4)

  1. Transplant engraftment

    Monitor median rate of engraftment by 42 days.

    Measured: 42 days

  2. Graft Failure

    Determine the rate of graft failure by day 100 (defined as lack of achievement of an ANC >=500/mL with associated pancytopenia)

    Measured: 100 days

  3. Non-relapse mortality (NRM)

    Determine the incidence of non-relapse mortality (NRM) at 100 days and 1 year

    Measured: 12 months

  4. Overall survival (OS)

    Number of participants experiencing progression free survival at one year follow up

    Measured: 12 months

Timeline

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

  1. First posted21 Feb 2023
  2. Study start11 May 2023
  3. Primary completion30 Nov 2027 (estimated)
  4. Study completion30 Nov 2030 (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
Parallel groups
Masking
None (open label)
Enrolment
70 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05735717
Sponsor's protocol ID
2021LS061
Status last verified
Mar 2026
First submitted
10 Jan 2023
Last updated
6 Apr 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No: For the purposes of data and safety monitoring, this study is classified as high risk (investigator initiated under an IND). Therefore the following requirements will be fulfilled: * The Masonic Cancer Center Data and Safety Monitoring Council (DSMC) will review the study's progress at least quarterly. * The PI will comply with at least twice yearly monitoring of the project by the Masonic Cancer Center monitoring services. * The PI will oversee the submission of all reportable adverse events per the definition of reportable in Section 11.5 to the Masonic Cancer Center's SAE Coordinator, the University of Minnesota IRB, and the FDA.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Fludarabine, Busulfan and Melphalan in people with Hematologic Malignancy and Acute Leukemia, run by Masonic Cancer Center, University of Minnesota 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 9 inclusion and 6 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 6 Apr 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 Margaret MacMillan (612-626-2961, macmi002@umn.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Determine the rate of GVHD after alpha beta TCR depletion (time frame: 100 days). 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 NCT05735717.

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 6 Apr 2026.

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