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

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.

Conditions

• Hematologic Malignancy
• Acute Leukemia
• Remission
• Acute Myeloid Leukemia
• Acute Lymphoblastic Leukemia
• AML
• TP53
• Intrachromosomal Amplification of Chromosome 21
• Cytogenetic Abnormality
• CNS Leukemia
• Minimal Residual Disease
• Myelodysplasia
• Juvenile Myelomonocytic Leukemia
• Somatic Mutation
• PTPN11 Gene Mutation
• N-RAS Gene Amplification
• Neurofibromatosis 1
• NF1 Mutation
• CBL Gene Mutation
• Monosomy 7
• Chromosome Abnormality
• Fetal Hemoglobin
• Lymphoblastic Lymphoma
• High Grade Non-Hodgkin's Lymphoma, Adult

Interventions

DRUG

Fludarabine

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

DRUG

Busulfan

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

DRUG

Melphalan

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

DRUG

Rituximab

200 mg/m2 intravenous given once on day-1

DRUG

Levetiracetam

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.

BIOLOGICAL

Alpha/Beta T Cell-Depleted Hematopoietic Stem Cells

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.

DRUG

Thymoglobulin

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.

DRUG

Cyclophosphamide

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

Sponsors & Collaborators

• Masonic Cancer Center, University of Minnesota

  lead OTHER

Principal Investigators

• Margaret MacMillan · University of Minnesota Masonic Cancer Center

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Max Age

60 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2023-05-11

Primary Completion

2027-11-30

Completion

2030-11-30

FDA Drug

Yes

Countries

• United States

Study Locations