Donor Umbilical Cord Blood Natural Killer Cells, Aldesleukin and Umbilical Cord Blood Transplant in Patients With Refractory Hematologic Cancers.

NCT ID: NCT00354172

Last Updated: 2017-12-28

Study Results

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

16 participants

Study Classification

INTERVENTIONAL

Study Start Date

2006-02-28

Study Completion Date

2008-08-31

Brief Summary

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RATIONALE: Giving chemotherapy, natural killer cells, aldesleukin, and total-body irradiation before a donor umbilical cord blood stem cell transplant helps stop the growth of abnormal cells and cancer cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine, mycophenolate mofetil, and methylprednisolone before and after transplant may stop this from happening.

PURPOSE: This clinical trial is studying how well giving fludarabine and cyclophosphamide together with total-body irradiation followed by donor umbilical cord blood natural killer cells, aldesleukin, and umbilical cord blood transplant works in treating patients with refractory hematologic cancer or other diseases.

Detailed Description

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OBJECTIVES:

Primary

* Determine the incidence of 6-month disease free survival. The primary laboratory objective is the measure of in vivo expansion of umbilical cord blood (UCB) derived natural killer cells (NK) after a fully ablative preparative regimen.

Secondary

* Determine the incidence of transplant-related mortality at 6 months after NK UCB + double UCBT
* Evaluate the pattern of chimerism after NK UCB + double UCBT
* Determine the incidence of neutrophil engraftment at day 42 after NK UCB + double umbilical cord blood transplantation (UCBT)
* Determine the incidence of platelet engraftment at 6 months after NK UCB + double UCBT
* Determine the incidence of acute graft-versus-host disease (GVHD) grade II-IV and grade III-IV at day 100 after NK UCB + double UCBT
* Determine the incidence of chronic GVHD at 1 year after NK UCB + double UCBT
* Determine the disease-free survival at 1 after NK UCB + double UCBT
* Determine the incidence of relapse at 1 after NK UCB + double UCBT

OUTLINE: This is a single arm, nonrandomized, open-label study.

* Myeloablative conditioning regimen: Patients receive fludarabine intravenously (IV) over 1 hour on days -18 to -16 and cyclophosphamide intravenously (IV) on days -18 and -17. Patients undergo total-body irradiation twice daily on days -16 to -13.
* Haploidentical umbilical cord blood (UCB) natural killer (NK) cell therapy and aldesleukin: Patients undergo haploidentical UCB-enriched NK cell (CD3- depleted) infusion on day -13. Patients then receive aldesleukin subcutaneously on days -13, -11, -9, -7, -5, and -3. Some patients may also receive methylprednisolone IV on days -1 and 0.
* UCB transplantation (UCBT): Patients undergo a single or double UCBT on day 0. Beginning on day 1, patients receive filgrastim (G-CSF) IV once daily until blood counts recover.
* Graft-vs-host disease (GVHD) prophylaxis: Patients receive cyclosporine IV over 2 hours 2-3 times daily beginning on day -1 and continuing until day 100, followed by a taper until day 180. Patients also receive mycophenolate mofetil IV or orally 2-3 times daily beginning on day -1 and continuing until day 30 (or 7 days after engraftment) in the absence of acute GVHD.

Conditions

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Leukemia Myelodysplastic Syndromes

Keywords

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adult acute myeloid leukemia with 11q23 (MLL) abnormalities adult acute myeloid leukemia with inv(16)(p13;q22) adult acute myeloid leukemia with t(15;17)(q22;q12) adult acute myeloid leukemia with t(16;16)(p13;q22) adult acute myeloid leukemia with t(8;21)(q22;q22) atypical chronic myeloid leukemia blastic phase chronic myelogenous leukemia childhood chronic myelogenous leukemia previously treated myelodysplastic syndromes recurrent adult acute myeloid leukemia recurrent childhood acute myeloid leukemia relapsing chronic myelogenous leukemia secondary acute myeloid leukemia secondary myelodysplastic syndromes childhood myelodysplastic syndromes

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Treated Patients

All patients receiving treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.

Group Type EXPERIMENTAL

aldesleukin

Intervention Type BIOLOGICAL

10 Million units subcutaneous every other day on days -13 (after Natural killer cell graft), -11, -9, -7, -5, -3) If \< 45 kilograms (Kg) - interleukin (IL)-2 at 5 MU/m2

filgrastim

Intervention Type BIOLOGICAL

All patients will receive filgrastim (same as granulocyte-colony stimulating factor or G-CSF) 5 mcg/kg/day intravenous (IV) (dose rounded to vial size) based on the actual body weight beginning on day +1 after umbilical cord blood (UCB) infusion. Granulocyte Colony Stimulating Factor (G-CSF) will be administered daily until the absolute neutrophil count (ANC) exceeds 2.5 x 10\^9/L for three consecutive days and then discontinued. If the ANC decreases to \<1.0 x 10\^9/L, G-CSF will be reinstituted.

natural killer cell (NK) therapy

Intervention Type BIOLOGICAL

All CD3 depleted cells will be given (less those required for product monitoring). The minimum size of a starting NK cell unit will be 700 million mononuclear cells before processing. NK cells (CD56+) and NK cell precursors (CD34+/CD7-, CD34+/CD7+, CD34-/CD7+) will be monitored and reported but will not serve as lot release.

cyclophosphamide

Intervention Type DRUG

Cyclophosphamide to be administered with high volume fluid flush and mesna on days-18 and -17 after fludarabine. Cyclophosphamide 60 mg/kg/day intravenously (IV) x 2 days, total dose 120 mg/m2 (days -18 and -17)

cyclosporine

Intervention Type DRUG

Patients will receive cyclosporine (CSA) therapy beginning on day -1 maintaining a level of \>200 ng/mL. For adults the initial dose will be 2.5 mg/kg intravenously (IV) over 2 hours every 12 hours. For children \<40 kg the initial dose will be 2.5 mg/kg IV over 2 hours every 8 hours.

fludarabine phosphate

Intervention Type DRUG

Fludarabine 25 mg/m2/day intravenously (IV) x 3 days, total dose 75 mg/m2 (days -18 to -16)

methylprednisolone

Intervention Type DRUG

This modification will be enacted based on the engraftment stopping rule on all subsequent patients to stop natural killer (NK) cell reaction.

Methylprednisolone bolus 1000 mg intravenously (IV) will be administered day -1 and day 0 (before umbilical cord blood transplant) to suppress natural killer (NK) cell activity before transplant. Starting cyclosporin and mycophenolate mofetil (MMF) will also contribute to suppressing residual NK cell activity.

mycophenolate mofetil

Intervention Type DRUG

All patients will begin mycophenolate mofetil (MMF) on day -1. Patients

≥ 45 kilograms will receive MMF at the dose of 3 grams/day divided into 2 or 3 doses. Pediatric patient (\<45 kilograms) will receive MMF at the dose of 15 mg/kg. Use intravenous (IV) route between days -1 and +5, then, if tolerated, may change to by mouth (PO) between days +6 and +30.

Stop MMF at day +30 or 7 days after engraftment, whichever day is later, if no acute Graft Versus Host Disease. (Definition of engraftment is 1st day of 3 consecutive days of absolute neutrophil count \[ANC\] \> 0.5 x 10\^9 /L).

Umbilical Cord Blood Transplantation (UCBT)

Intervention Type PROCEDURE

The product is infused via intravenous (IV) drip directly into the central line without a needle, pump or filter.

Total body irradiation (TBI)

Intervention Type RADIATION

TBI 165 Gray (cGy) will be given twice daily for a total dose of 1320 cGy (days

-16 to -13).

Interventions

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aldesleukin

10 Million units subcutaneous every other day on days -13 (after Natural killer cell graft), -11, -9, -7, -5, -3) If \< 45 kilograms (Kg) - interleukin (IL)-2 at 5 MU/m2

Intervention Type BIOLOGICAL

filgrastim

All patients will receive filgrastim (same as granulocyte-colony stimulating factor or G-CSF) 5 mcg/kg/day intravenous (IV) (dose rounded to vial size) based on the actual body weight beginning on day +1 after umbilical cord blood (UCB) infusion. Granulocyte Colony Stimulating Factor (G-CSF) will be administered daily until the absolute neutrophil count (ANC) exceeds 2.5 x 10\^9/L for three consecutive days and then discontinued. If the ANC decreases to \<1.0 x 10\^9/L, G-CSF will be reinstituted.

Intervention Type BIOLOGICAL

natural killer cell (NK) therapy

All CD3 depleted cells will be given (less those required for product monitoring). The minimum size of a starting NK cell unit will be 700 million mononuclear cells before processing. NK cells (CD56+) and NK cell precursors (CD34+/CD7-, CD34+/CD7+, CD34-/CD7+) will be monitored and reported but will not serve as lot release.

Intervention Type BIOLOGICAL

cyclophosphamide

Cyclophosphamide to be administered with high volume fluid flush and mesna on days-18 and -17 after fludarabine. Cyclophosphamide 60 mg/kg/day intravenously (IV) x 2 days, total dose 120 mg/m2 (days -18 and -17)

Intervention Type DRUG

cyclosporine

Patients will receive cyclosporine (CSA) therapy beginning on day -1 maintaining a level of \>200 ng/mL. For adults the initial dose will be 2.5 mg/kg intravenously (IV) over 2 hours every 12 hours. For children \<40 kg the initial dose will be 2.5 mg/kg IV over 2 hours every 8 hours.

Intervention Type DRUG

fludarabine phosphate

Fludarabine 25 mg/m2/day intravenously (IV) x 3 days, total dose 75 mg/m2 (days -18 to -16)

Intervention Type DRUG

methylprednisolone

This modification will be enacted based on the engraftment stopping rule on all subsequent patients to stop natural killer (NK) cell reaction.

Methylprednisolone bolus 1000 mg intravenously (IV) will be administered day -1 and day 0 (before umbilical cord blood transplant) to suppress natural killer (NK) cell activity before transplant. Starting cyclosporin and mycophenolate mofetil (MMF) will also contribute to suppressing residual NK cell activity.

Intervention Type DRUG

mycophenolate mofetil

All patients will begin mycophenolate mofetil (MMF) on day -1. Patients

≥ 45 kilograms will receive MMF at the dose of 3 grams/day divided into 2 or 3 doses. Pediatric patient (\<45 kilograms) will receive MMF at the dose of 15 mg/kg. Use intravenous (IV) route between days -1 and +5, then, if tolerated, may change to by mouth (PO) between days +6 and +30.

Stop MMF at day +30 or 7 days after engraftment, whichever day is later, if no acute Graft Versus Host Disease. (Definition of engraftment is 1st day of 3 consecutive days of absolute neutrophil count \[ANC\] \> 0.5 x 10\^9 /L).

Intervention Type DRUG

Umbilical Cord Blood Transplantation (UCBT)

The product is infused via intravenous (IV) drip directly into the central line without a needle, pump or filter.

Intervention Type PROCEDURE

Total body irradiation (TBI)

TBI 165 Gray (cGy) will be given twice daily for a total dose of 1320 cGy (days

-16 to -13).

Intervention Type RADIATION

Other Intervention Names

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IL-2 interleukin-2 granulocyte colony-stimulating factor (G-CSF) NK cells Cytoxan cyclosporin Fludara Medrol Solu-Medrol Depo-Medrol methylprednisolone sodium succinate mycophenolic acid allogeneic transplant radiation

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of 1 of the following:

* Acute myeloid leukemia with active leukemia (i.e., not in complete remission \[CR\]), defined by light microscopy (bone marrow) and having failed ≥ 1 round of standard chemotherapy
* Chronic myelogenous leukemia with myeloid blast crisis not in second chronic phase after ≥ 1 course of standard chemotherapy and imatinib mesylate
* Myelodysplastic syndromes (MDS) or other myeloproliferative disorders more than 10% blasts after ≥ 1 course of standard chemotherapy
* Unrelated umbilical cord blood (UCB) donor(s) available - Each unit must be 4-6/6 HLA-A, -B, and -DRB1 matched with the recipient (and to each other if 2 units are utilized) (for UCB graft) AND 3/6 HLA-A, -B, and -DRB1 matched with the recipient (for UCB natural killer \[NK\] cells)
* Karnofsky performance status (PS) 80-100% (adult patients) or Lansky PS 50-100% (pediatric patients)
* Creatinine ≤ 2.0 mg/dL (adult patients) OR creatinine clearance \> 40 mL/min (pediatric patients)
* Bilirubin, aspartate aminotransferase (AST), alanine aminotransferase (ALT), Alkaline phosphatase ≤ 5 times upper limit of normal (ULN)
* Corrected Carbon Monoxide Diffusing Capacity (DLCO) \> 50% normal OR oxygen saturation \> 92% (in pediatric patients who cannot undergo pulmonary function tests)
* Left ventricular ejection fraction ≥ 45%

Exclusion Criteria

* Pregnant or nursing
* Positive pregnancy test (Fertile patients must use effective contraception)
* History of HIV infection
* Active infection at time of transplantation

* Active infection with Aspergillus or other mold within the past 120 days
* Less than 6 months since prior myeloablative transplant (≤ 18 years old)
* Prior myeloablative allotransplant or autologous transplant (\> 18 years old)
* No prior extensive therapy including \> 12 months of any alkylator chemotherapy or \> 6 months of alkylator therapy with extensive radiation (e.g., mantle irradiation for Hodgkin's lymphoma)
* Prior radiation therapy that would make the patient ineligible for total-body irradiation
Maximum Eligible Age

45 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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Jeffrey Miller, MD

Role: PRINCIPAL_INVESTIGATOR

Masonic Cancer Center, University of Minnesota

Locations

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Masonic Cancer Center at University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Countries

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United States

Other Identifiers

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MT2005-18

Identifier Type: OTHER

Identifier Source: secondary_id

0509M73449

Identifier Type: OTHER

Identifier Source: secondary_id

UMN-2005LS058

Identifier Type: -

Identifier Source: org_study_id