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NCT Number: NCT05735717

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

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Key information

Conditions

Hematologic Malignancy AML Acute Leukemia Acute Lymphoblastic Leukemia Acute Myeloid Leukemia Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Refractory Anemia, Refractory, with Excess of Blasts Bone Marrow Diseases CBL Gene Mutation CNS Leukemia Chromosome 7, monosomy Chromosome Aberrations Chromosome Abnormality Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cytogenetic Abnormality Fetal Hemoglobin Genetic Diseases, Inborn Hematologic Diseases Hematologic Neoplasms Hemic and Lymphatic Diseases Hemoglobinopathies Heredodegenerative Disorders, Nervous System High Grade Non-Hodgkin's Lymphoma, Adult Immune System Diseases Immunoproliferative Disorders Infections Influenza, Human Intrachromosomal Amplification of Chromosome 21 Juvenile Myelomonocytic Leukemia Leukemia Leukemia, Lymphoid Leukemia, Myeloid Leukemia, Myeloid, Acute Leukemia, Myelomonocytic, Juvenile Lymphatic Diseases Lymphoblastic Lymphoma Lymphoproliferative Disorders Minimal Residual Disease Monosomy 7 Myelodysplasia Myelodysplastic Syndromes Myelodysplastic-Myeloproliferative Diseases N-RAS Gene Amplification NF1 Mutation Neoplasm, Residual Neoplasms Neoplasms by Histologic Type Neoplasms by Site Neoplasms, Nerve Tissue Neoplastic Processes Neoplastic Syndromes, Hereditary Nerve Sheath Neoplasms Nervous System Diseases Neurocutaneous Syndromes Neurodegenerative Diseases Neurofibroma Neurofibromatoses Neurofibromatosis 1 Neuromuscular Diseases Orthomyxoviridae Infections PTPN11 Gene Mutation Pathologic Processes Pathological Conditions, Signs and Symptoms Peripheral Nervous System Diseases Precursor Cell Lymphoblastic Leukemia-Lymphoma RNA Virus Infections Remission Respiratory Tract Diseases Respiratory Tract Infections Somatic Mutation TP53 Thalassemia Virus Diseases beta-Thalassemia

Age range

Up to 60 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

University of Minnesota Masonic Cancer Center

Minneapolis, Minnesota, 55455, United States

Location status: Recruiting

Location contact

Margaret MacMillan

CONTACT

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.
  • 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

Exclusion criteria

  • 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

Treatment and study plan

Fludarabine

Drug

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

busulfan

Drug

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

melphalan

Drug

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

Rituximab

Drug

200 mg/m2 intravenous given once on day-1

Levetiracetam

Drug

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.

Other names: Keppra

Alpha/Beta T Cell-Depleted Hematopoietic Stem Cells

Biological

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.

Thymoglobulin

Drug

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.

Other names: ATG

Cyclophosphamide

Drug

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

Primary outcomes

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

    Time frame: 100 days

    GVHD incidence after treatment.

Secondary outcomes

  1. Transplant engraftment

    Time frame: 42 days

    Monitor median rate of engraftment by 42 days.

  2. Graft Failure

    Time frame: 100 days

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

  3. Non-relapse mortality (NRM)

    Time frame: 12 months

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

  4. Overall survival (OS)

    Time frame: 12 months

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

Study contacts

Contact information is provided by the study sponsor or research team.

Margaret MacMillan

CONTACT

[email protected]

612-626-2961

Sponsors and collaborators

Lead sponsor

Masonic Cancer Center, University of Minnesota

Other

Registry information

Official study 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

Important dates

Study start
2023
Primary completion
2027
Study completion
2030
First posted
Feb 21, 2023
Registry last updated
Apr 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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