T-Regulatory Cell and CD3 Depleted Double Umbilical Cord Blood Transplantation in Hematologic Malignancies

NCT ID: NCT01163201

Last Updated: 2017-12-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE1/PHASE2

Study Classification

INTERVENTIONAL

Study Start Date

2014-01-31

Study Completion Date

2015-01-31

Brief Summary

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This is a unique dose-escalation trial that will titrate doses of umbilical cord blood (UCB) Treg and CD3+ Teff cells with the goal of infusing as many CD3+ Teff cells as possible without conferring grade II-IV acute graft-versus-host disease (GVHD).

In this study, the investigators propose to add UCB Treg and UCB CD3+ Teff cells to the two TCD UCB donor units with the goal of transplanting as many CD3+ Teff cells as possible without reintroducing risk of acute GVHD. The investigators hypothesize that Treg will permit the reintroduction of CD3+ Teff cells that will provide a bridge while awaiting HSC T cell recovery long term. The co-infusion of Treg will prevent GVHD without the need for prolonged pharmacologic immunosuppression.

Detailed Description

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Based on prior studies, the first patient will start at lowest dose combination (3 x 10\^6/kg of Treg and 3 x 10\^6/kg of CD3+ Teff cells).

One patient will be entered at each level with a minimum of 35 days to observe the patient prior to moving to the next dose level. (1) If GVHD does not occur, a "successful step", then the CD3+ Teff cell dose will increase to the next higher level for the next patient; (2) If GVHD occurs, a "failed step", then Treg dose will increase to the next higher level for the next patient. It would take a minimum of 5 (if no GVHD) and maximum of 9 patients (if GVHD is observed at each level) to complete all Treg:CD3+ Teff cell combinations.

An additional 10 patients will be enrolled to verify that this reflects the optimal combination and evaluate its safety profile.

Conditions

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Hematologic Malignancy Acute Myeloid Leukemia Acute Lymphocytic Leukemia Chronic Myelogenous Leukemia in Blast Crisis Anemia, Refractory, With Excess of Blasts Chronic Myeloproliferative Disease Chronic Lymphocytic Leukemia Small Lymphocytic Lymphoma Marginal Zone B-cell Lymphoma Follicular Lymphoma Lymphoplasmacytic Lymphoma Mantle-Cell Lymphoma Prolymphocytic Lymphoma Large Cell Non-Hodgkin's Lymphoma Lymphoblastic Lymphoma Burkitt's Lymphoma High Grade Non-Hodgkin's Lymphoma

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Treg Plus CD3+Teff Treatment

Includes dose adjustment of T regulatory (Treg) and CD3+ T effector (CD3+ Teff) cells in recipients of double UCB transplantation

Group Type EXPERIMENTAL

Treg cells

Intervention Type BIOLOGICAL

Given by infusion on Day 0 after transplantation - Five doses of Treg (3 x 10\^6/kg, 10 x 10\^6/kg, 30 x 10\^6/kg, 100 x 10\^6/kg and 300 x 10\^6/kg)

CD3+ Teff cells

Intervention Type BIOLOGICAL

Given by infusion on Day 0 after transplantation - 5 doses of CD3+ Teff cells (3 x 10\^6 cells/kg, 6 x 10\^6 cells/kg, 9 x 10\^6 cells/kg, 12 x 10\^6 cells/kg, and 15 x 10\^6 cells/kg with the latter dose representing the median number of CD3+ cells in two UCB unit grafts

Fludarabine

Intervention Type DRUG

Given intravenously on Days -8 through -6, 25 mg/m\^2 over 1 hour

Cyclophosphamide

Intervention Type DRUG

Given intravenously on Day -7 and -6, 60 mg/kg

Total body irradiation

Intervention Type RADIATION

Given on Days -4 through -2, 165 cGY twice a day.

Umbilical cord blood transplantation

Intervention Type BIOLOGICAL

Infusion given on day 0

Interventions

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Treg cells

Given by infusion on Day 0 after transplantation - Five doses of Treg (3 x 10\^6/kg, 10 x 10\^6/kg, 30 x 10\^6/kg, 100 x 10\^6/kg and 300 x 10\^6/kg)

Intervention Type BIOLOGICAL

CD3+ Teff cells

Given by infusion on Day 0 after transplantation - 5 doses of CD3+ Teff cells (3 x 10\^6 cells/kg, 6 x 10\^6 cells/kg, 9 x 10\^6 cells/kg, 12 x 10\^6 cells/kg, and 15 x 10\^6 cells/kg with the latter dose representing the median number of CD3+ cells in two UCB unit grafts

Intervention Type BIOLOGICAL

Fludarabine

Given intravenously on Days -8 through -6, 25 mg/m\^2 over 1 hour

Intervention Type DRUG

Cyclophosphamide

Given intravenously on Day -7 and -6, 60 mg/kg

Intervention Type DRUG

Total body irradiation

Given on Days -4 through -2, 165 cGY twice a day.

Intervention Type RADIATION

Umbilical cord blood transplantation

Infusion given on day 0

Intervention Type BIOLOGICAL

Other Intervention Names

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Fludara Cytoxan UCBT

Eligibility Criteria

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Inclusion Criteria

* Only patients requiring a double umbilical cord blood (UCB) transplant are to be considered for this study.

UCB Requirements

* Three UCB units are required - one for Treg production and two for UCB transplant. The unrelated UCB donors must be 4-6/6 HLA-A, B, DRB1 matched with the recipient (HLA matching using molecular techniques: A and B to antigen level resolution and DR to allele level resolution). Suitable UCB units will be selected according to the University Of Minnesota UCB Graft Selection Algorithm.
* Suitable UCB units must be ABO matched.

Disease Criteria:

* Patients aged 18 to 55 years
* Acute Myeloid Leukemia: with morphologically persistent disease in a representative bone marrow aspirate sample with ≤ 10% blasts after at least 1 cycles of chemotherapy (if patient refuses or is disqualified from alternative protocols), or in 3rd or higher complete remission (CR).
* Acute Lymphocytic Leukemia: with morphologically persistent disease in a representative bone marrow aspirate sample with ≤ 10% blasts after at least 1 cycles of chemotherapy, or in 3rd or higher CR
* Chronic Myelogenous Leukemia in Blast Crisis: with ≤10% residual blasts in the bone marrow aspirate after at least 1 cycle of induction chemotherapy in combination with a tyrosine kinase inhibitor (TKI)
* Refractory Anemia with Excess Blasts: (≤ 10%) in representative bone marrow aspirate sample of blasts after 1 cycle of induction chemotherapy. If treated with hypomethylating agents, patients are eligible if blast count is ≤ 10% after 4 cycles or evidence of stable or progressive disease after at least 2 cycles.
* Chronic Myeloproliferative Disease
* Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma, Marginal Zone B-Cell Lymphoma or Follicular Lymphoma: disease must be refractory after at least two chemotherapy regimens or is chemotherapy sensitive but has residual nodal disease of ≥ 5 cm
* Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia: disease must be refractory after at least two chemotherapy regimens or is chemotherapy sensitive but has residual nodal disease of ≥ 5 cm
* Large Cell Non-Hodgkin's Lymphoma: disease must be refractory after at least two chemotherapy regimens or is chemotherapy sensitive but has residual nodal disease of ≥ 5 cm
* Lymphoblastic Lymphoma, Burkitt's Lymphoma, and other High-Grade NHL: disease must be refractory after at least two chemotherapy regimens or is chemotherapy sensitive but has residual nodal disease of ≥ 5 cm
* Performance Status, Age, and Organ Function
* Adequate performance status defined as a Karnofsky score ≥ 80%
* Adequate organ function defined as:

* Renal: creatinine \< 2.0 mg/dL,
* Hepatic: bilirubin, AST/ALT, ALP \< 5 x upper limit of normal,
* Pulmonary function: DLCOcorr \> 50% normal,
* Cardiac: left ventricular ejection fraction \> 45%
* Voluntary written informed consent signed before performance of any study-related procedure not part of normal medical care

Exclusion Criteria

* Available medically suitable HLA-identical related donor
* Active infection at time of transplantation (including active infection with Aspergillus or other mold within 30 days)
* History of HIV infection
* Pregnant or breast feeding. The agents used in this study may be teratogenic to a fetus and there is no information on the excretion of agents into breast milk. Females of childbearing potential must have a blood test or urine study within 14 days prior to registration to rule out pregnancy
* Prior myeloablative transplant within the last 6 months
* Extensive prior therapy including \> 12 months alkylator therapy or \> 6 months alkylator therapy with extensive radiation
* Patients who have received Y-90 ibritumomab (Zevalin) or I-131 tositumomab (Bexxar) as part of their salvage therapy (not eligible for myeloablative umbilical cord blood transplant)
Minimum Eligible Age

18 Years

Maximum Eligible Age

55 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Masonic Cancer Center, University of Minnesota

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Claudio Brunstein, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Masonic Cancer Center, University of Minnesota

Locations

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Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Countries

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United States

Other Identifiers

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MT2009-03

Identifier Type: OTHER

Identifier Source: secondary_id

0910M73595

Identifier Type: OTHER

Identifier Source: secondary_id

2009LS018

Identifier Type: -

Identifier Source: org_study_id