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Clinical Trials/NCT04151706
NCT04151706
Completed
Phase 2

CD34 Selected Allogeneic Hematopoietic Cell Transplantation With Myeloablative Conditioning and CD8+ Memory T Cell Infusion For Patients With Myelodysplastic Syndrome, Acute Leukemia, and Chronic Myelogenous Leukemia

Robert Lowsky1 site in 1 country7 target enrollmentFebruary 27, 2020

Overview

Phase
Phase 2
Intervention
CD8+ Memory T Cell Infusion
Conditions
Acute Myeloid Leukemia
Sponsor
Robert Lowsky
Enrollment
7
Locations
1
Primary Endpoint
Number of Participants Achieving Graft-versus-Host Disease-Free and Relapse-Free Survival (GRFS) Through 1 Year Post-Transplant
Status
Completed
Last Updated
last year

Overview

Brief Summary

This study will evaluate combining stem cells from the patient's matched sibling donor (a standard CD34-selected transplant) with a second infusion of white blood cells called "CD8 memory T-cells" from their sibling donor.

Detailed Description

Primary Objective: To determine the rate of graft versus host disease (GvHD) free, relapse free survival (GRFS) at one year following CD34 selected allogeneic hematopoietic cell transplantation using myeloablative conditioning combined with an infusion of phenotypic CD8+ memory T cells from human leukocyte antigen (HLA) matched donors for patients with myelodysplastic syndrome (MDS), acute myeloid leukemia (AML), or acute lymphoblastic leukemia (ALL) and chronic myeloid leukemia (CML). Secondary Objective: To determine the rate of graft rejection, acute and chronic GvHD, non relapse mortality, relapse, overall survival, and disease free survival.

Registry
clinicaltrials.gov
Start Date
February 27, 2020
End Date
September 8, 2023
Last Updated
last year
Study Type
Interventional
Study Design
Single Group
Sex
All

Investigators

Responsible Party
Sponsor Investigator
Principal Investigator

Robert Lowsky

Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy)

Stanford University

Eligibility Criteria

Inclusion Criteria

  • Acute leukemia, in morphologic complete remission, OR myelodysplasia with \< 10% blasts in the marrow, and no circulating blasts that contain auer rods. Patients with chronic myelomonocytic leukemia (CMML) must have a WBC count ≤ 10,000 cells/μL and \< 10% blasts in the marrow.
  • Planned myeloablative conditioning regimen at Stanford University Medical Center.
  • Karnofsky or Lansky Performance Score ≥ 70%.
  • Must have an HLA related donor as follows: onor must be an 8/8 match for HLA A, B and C at intermediate (or higher) resolution, and DRB1 at high resolution using DNA based typing. The donors must be willing to receive G CSF followed by collection of cells by apheresis, and must meet the Program's criteria for donation.
  • Cardiac function: Ejection fraction at rest ≥ 40%.
  • Serum creatinine value of \< 1.5 mg/dL, or an estimated creatinine clearance greater than 50 mL/minute (using the Stanford calculator for eGFR available in EPIC)
  • Diffusing capacity of the lungs for carbon monoxide (DLCO) ≥ 50% (adjusted for Hgb)
  • Forced vital capacity (FVC) ≥ 50%.
  • Forced expiratory volume (FEV1) ≥ 50%.
  • Total bilirubin \< 2 times the upper limit of normal (ULN) (unless the elevated bilirubin is attributed to Gilbert's Syndrome)

Exclusion Criteria

  • Prior autologous or allogeneic hematopoietic stem cell transplant
  • Prior malignancies, except resected non melanoma or treated cervical carcinoma in situ. Cancer treated with curative intent ≥ 5 years previously is allowed. Cancer treated with curative intent \< 5 years previously will not be allowed unless approved by the Protocol Officer or one of the Protocol Chairs
  • Active central nervous system (CNS) involvement by malignant cells
  • Presence of fluid collection (ascites, pleural or pericardial effusion) that interferes with methotrexate clearance or makes methotrexate use contraindicated
  • Requirement for supplemental oxygen
  • Uncontrolled bacterial, viral or fungal infections (currently taking medication and with progression or no clinical improvement) at time of enrollment
  • History of uncontrolled autoimmune disease or on active treatment (defined as \> 5 mg prednisone daily)
  • Seropositive for HIV 1 or 2
  • Seropositive for HTLV I or -II
  • Active Hepatitis B or C viral replication by polymerase chain reaction (PCR)

Arms & Interventions

fTBI/Thiotepa/fludarabine

Participants will be infused on Day 0 with donor derived CD34+ selected cells combined with CD8+CD45RA- T cells {CD Memory T Cells} following a standard myeloablative conditioning regimen that might consist of fTBI, Thiotepa, and Fludarabine or Busulfan and Cyclophosamide.

Intervention: CD8+ Memory T Cell Infusion

fTBI/Thiotepa/fludarabine

Participants will be infused on Day 0 with donor derived CD34+ selected cells combined with CD8+CD45RA- T cells {CD Memory T Cells} following a standard myeloablative conditioning regimen that might consist of fTBI, Thiotepa, and Fludarabine or Busulfan and Cyclophosamide.

Intervention: Thiotepa

fTBI/Thiotepa/fludarabine

Participants will be infused on Day 0 with donor derived CD34+ selected cells combined with CD8+CD45RA- T cells {CD Memory T Cells} following a standard myeloablative conditioning regimen that might consist of fTBI, Thiotepa, and Fludarabine or Busulfan and Cyclophosamide.

Intervention: Fludarabine

fTBI/Thiotepa/fludarabine

Participants will be infused on Day 0 with donor derived CD34+ selected cells combined with CD8+CD45RA- T cells {CD Memory T Cells} following a standard myeloablative conditioning regimen that might consist of fTBI, Thiotepa, and Fludarabine or Busulfan and Cyclophosamide.

Intervention: Hyperfractionated TBI

fTBI/Thiotepa/fludarabine

Participants will be infused on Day 0 with donor derived CD34+ selected cells combined with CD8+CD45RA- T cells {CD Memory T Cells} following a standard myeloablative conditioning regimen that might consist of fTBI, Thiotepa, and Fludarabine or Busulfan and Cyclophosamide.

Intervention: Busulfan

fTBI/Thiotepa/fludarabine

Participants will be infused on Day 0 with donor derived CD34+ selected cells combined with CD8+CD45RA- T cells {CD Memory T Cells} following a standard myeloablative conditioning regimen that might consist of fTBI, Thiotepa, and Fludarabine or Busulfan and Cyclophosamide.

Intervention: Cyclophosphamide

Outcomes

Primary Outcomes

Number of Participants Achieving Graft-versus-Host Disease-Free and Relapse-Free Survival (GRFS) Through 1 Year Post-Transplant

Time Frame: 1 year

The rate of participants who do not experience GvHD and also do not experience relapse are collectively considered to be GRFS. Relapse will be assessed according to the myelodysplastic syndrome or leukemia response criteria. The participants will be assessed for GRFS though 1 year post transplant. The outcome will be reported as the number of participants, a number without dispersion.

Secondary Outcomes

  • Number of Participants Experiencing Graft Rejection Through 1 Year Post-Transplant(1 year)
  • Number of Participants Experiencing Acute Graft-versus-Host Disease (GvHD) Through 1 Year Post-Transplant(1 year)
  • Number of Participants With Chronic, Steroid-Requiring Graft-versus-Host Disease (GvHD) Through 1 Year Post-Transplant(1 year)
  • Number of Participants With Non-Relapse Mortality Through 1 Year Post-Transplant Without Recurrence of Myelodysplastic Syndrome or Leukemia(1 year)
  • Number of Participants Who Experience Relapse Through 1 Year Post-Transplant(1 year)
  • Overall Survival (OS)(1 year)

Study Sites (1)

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