Donor Natural Killer Cells and Aldesleukin in Treating Patients w/High Risk AML Undergoing Donor Stem Cell Transplant

NCT ID: NCT00303667

Last Updated: 2017-12-28

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

50 participants

Study Classification

INTERVENTIONAL

Study Start Date

2005-01-31

Study Completion Date

2011-03-31

Brief Summary

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RATIONALE: Giving chemotherapy, such as fludarabine phosphate and cyclophosphamide, and total body irradiation, before peripheral blood stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells and natural killer (NK) 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. Giving IL-2 (aldesleukin) after NK cell infusion may stimulate them to kill any remaining cancer cells.

PURPOSE: This phase I/II (currently enrolling in phase II) trial is studying how well a donor natural killer cell infusion works in treating patients who are undergoing donor stem cell transplant for acute myeloid leukemia.

Detailed Description

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OBJECTIVES:

Primary

* To determine the disease-free survival at 6 months and 1 year in patients with high-risk myeloid malignancies who undergo a reduced-intensity haploidentical hematopoietic stem cell transplantation (HSCT) supplemented with donor natural killer (NK) cells.

Secondary

* To evaluate the in vivo expansion of a donor CD3- CD19- selected NK cell product administered after a preparative regimen of cyclophosphamide, fludarabine, and total body irradiation (TBI) and HSCT in these patients.
* To determine the rate of graft failure defined by absolute neutrophil count (ANC) \< 500/mm³ by day 28.
* To determine the incidence of grade III-IV acute graft-versus-host disease (GVHD) at 6 months.
* To determine the rate of treatment-related mortality at day 100.
* To determine the incidence of chronic GVHD at 12 months.
* To determine the incidence of disease relapse at 12 months.
* To determine the incidence of post-transplant lymphoproliferative disorder at 12 months.

Correlative

* To correlate immune reconstitution of the in vivo expanded haploidentical NK cells with clinical outcomes.

OUTLINE: This is an open-label study.

Patients receive fludarabine intravenous (IV) over 1 hour on days -18 to -14 and cyclophosphamide IV over 2 hours on days -16 and -15. Patients receive cyclosporin A on Day -15 through Day -8. Patients undergo total body irradiation on day -13. Patients then receive an infusion of donor natural killer cells on day -12 and interleukin-2 subcutaneously on alternating days between days -12 to -2. Patients receive thymoglobulin (ATG) and undergo allogeneic peripheral blood stem cell transplantation on day 0.

After completion of study treatment, patients are followed periodically.

PROJECTED ACCRUAL: A total of 90 patients will be accrued for this study.

Conditions

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Acute Myelogenous Leukemia

Keywords

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NK cells natural killer cells immunotherapy hematopoietic cell transplant acute myelogenous leukemia

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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SCT w/Donor Natural Killer Cells - short schema

Patients with high risk myeloid malignancies undergoing allogeneic hematopoietic stem cell transplantation, receiving fludarabine phosphate (daily dose of 40mg/m\^2), cyclophosphamide (administered on Day -15 only), cyclosporin A, total body irradiation, natural killer cells, aldesleukin, and thymoglobulin.

Group Type EXPERIMENTAL

aldesleukin

Intervention Type BIOLOGICAL

Administered subcutaneously (SQ) 9 million units every other day beginning Day -12 through -2 (evening of natural killer cell infusion) for a total of 6 doses.

natural killer cells

Intervention Type BIOLOGICAL

Infusion given on Day -12; The targeted infused cell dose of CD3- CD19- selected NK product is within the range of 2-3 x 10\^7 cells/kg.

cyclophosphamide

Intervention Type DRUG

Administered intravenously (IV) 50 mg/kg on Day -15

fludarabine phosphate

Intervention Type DRUG

Administered intravenously (IV) 40 mg/m\^2 on Days -18 through -14

allogeneic hematopoietic stem cell transplantation

Intervention Type PROCEDURE

On day 0, patients will receive an allogeneic transplant using pool cells from the day -1 and day 0 PBSC which will be CD34+ selected as the donor graft. The graft will be infused over 15-60 minutes.

total body irradiation

Intervention Type RADIATION

Administered on Day -13, 200 cGy two times.

Thymoglobulin

Intervention Type BIOLOGICAL

intravenous (IV) 3 mg/kg on Day 0 (day of donor CD34 cell infusion)

Cyclosporin A

Intervention Type DRUG

1.5 mg/kg by mouth or intravenously for target dose range of 150-250; day -15 through day -8.

SCT w/Donor Natural Killer Cells - extended schema

Patients with high risk myeloid malignancies undergoing allogeneic hematopoietic stem cell transplantation, receiving fludarabine phosphate (daily dose of 35mg/m\^2), cyclophosphamide (administered on Days -15 and -16), cyclosporin A, total body irradiation, natural killer cells, aldesleukin, and thymoglobulin.

Group Type EXPERIMENTAL

aldesleukin

Intervention Type BIOLOGICAL

Administered subcutaneously (SQ) 9 million units every other day beginning Day -12 through -2 (evening of natural killer cell infusion) for a total of 6 doses.

natural killer cells

Intervention Type BIOLOGICAL

Infusion given on Day -12; The targeted infused cell dose of CD3- CD19- selected NK product is within the range of 2-3 x 10\^7 cells/kg.

allogeneic hematopoietic stem cell transplantation

Intervention Type PROCEDURE

On day 0, patients will receive an allogeneic transplant using pool cells from the day -1 and day 0 PBSC which will be CD34+ selected as the donor graft. The graft will be infused over 15-60 minutes.

total body irradiation

Intervention Type RADIATION

Administered on Day -13, 200 cGy two times.

Thymoglobulin

Intervention Type BIOLOGICAL

intravenous (IV) 3 mg/kg on Day 0 (day of donor CD34 cell infusion)

Cyclosporin A

Intervention Type DRUG

1.5 mg/kg by mouth or intravenously for target dose range of 150-250; day -15 through day -8.

cyclophosphamide

Intervention Type DRUG

Administered intravenously (IV) 50 mg/kg on Days -16 and -15

fludarabine phosphate

Intervention Type DRUG

Administered intravenously (IV) 35 mg/m\^2 on Days -18 through -14

Interventions

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aldesleukin

Administered subcutaneously (SQ) 9 million units every other day beginning Day -12 through -2 (evening of natural killer cell infusion) for a total of 6 doses.

Intervention Type BIOLOGICAL

natural killer cells

Infusion given on Day -12; The targeted infused cell dose of CD3- CD19- selected NK product is within the range of 2-3 x 10\^7 cells/kg.

Intervention Type BIOLOGICAL

cyclophosphamide

Administered intravenously (IV) 50 mg/kg on Day -15

Intervention Type DRUG

fludarabine phosphate

Administered intravenously (IV) 40 mg/m\^2 on Days -18 through -14

Intervention Type DRUG

allogeneic hematopoietic stem cell transplantation

On day 0, patients will receive an allogeneic transplant using pool cells from the day -1 and day 0 PBSC which will be CD34+ selected as the donor graft. The graft will be infused over 15-60 minutes.

Intervention Type PROCEDURE

total body irradiation

Administered on Day -13, 200 cGy two times.

Intervention Type RADIATION

Thymoglobulin

intravenous (IV) 3 mg/kg on Day 0 (day of donor CD34 cell infusion)

Intervention Type BIOLOGICAL

Cyclosporin A

1.5 mg/kg by mouth or intravenously for target dose range of 150-250; day -15 through day -8.

Intervention Type DRUG

cyclophosphamide

Administered intravenously (IV) 50 mg/kg on Days -16 and -15

Intervention Type DRUG

fludarabine phosphate

Administered intravenously (IV) 35 mg/m\^2 on Days -18 through -14

Intervention Type DRUG

Other Intervention Names

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Interleukin-2 IL-2 therapeutic allogeneic lymphocytes Cytoxan Fludara PBSC rabbit ATG CSA Cytoxan Fludara

Eligibility Criteria

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Inclusion Criteria

* 4.2 High risk acute myeloid leukemia (AML) fitting within one of the following disease groups:

* Primary induction failure defined as no complete remission (CR) after two or more induction cycles. For primary induction failure (PIF) or refractory AML, the patient must have \<5% circulating blasts (and \<1000 absolute circulating blasts) beyond Day 28 after last chemo. During work-up period if circulating blasts rise above these levels, the patient is ineligible. The use of hydrea or other non-induction cytotoxic agents is not allowed to reduce blasts and achieve this eligibility. If the blasts are higher than these limits, the patient should be treated on an alternative therapeutic protocol or receive another reinduction attempt. (See section 7 regarding final check of blast status within 7 days of preparative regimen start).
* Relapsed AML with low disease burden must have less than 5% marrow blasts at time of enrollment for patients who did not receive re-induction or measured at least 28 days from the start of reinduction therapy for patients who did receive re-induction (maximum of 2 re-induction attempts). Patients who have relapsed more than 12 months following a prior HCT and did not reach CR following one re-induction cycle but have less than 10% marrow blasts are eligible.
* CR3 or greater. This will include CRp defined as CR without platelet recovery to 100,000/mcL.
* CR1 or CR2 with high risk features (therapy induced, prior myelodysplastic syndrome \[MDS\] or myeloproliferative disease \[MPD\], high risk cytogenetic or molecular phenotype) with no available alternate (sibling, unrelated donor \[URD\] or umbilical cord blood \[UCB\]) donors.

Patients with prior central nervous system (CNS) involvement are eligible provided that it has been treated and CFS must be clear for at least 2 weeks prior to enrollment.

* Available related HLA-haploidentical donor (3-5 of 6 HLA, A, B and DRB1 matched)
* Karnofsky performance status \> 50
* Pulmonary Function: oxygen saturation ≥ on room air and diffusion lung capacity for carbon monoxide (DLCOcor) ≥ 40%
* Cardiac Function: Ejection fraction (EF) ≥ 30%, no uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities.
* Able to be off prednisone or other immunosuppressive medications for at least 3 days prior to Day 0
* Women of child bearing potential must have a negative pregnancy test within 14 days prior to study registration and agree to use adequate birth control during study treatment
* Human anti-mouse antibody (HAMA) monitoring: All subjects will be questioned about prior exposure to antibody therapy (including OKT3, rituximab, trastuzumab, etc).

* For subjects with no prior antibody therapy exposure, no further action will be taken
* For subjects who have received previous antibody therapies 10 ml of serum will be drawn before starting therapy. The presence of HAMA will not preclude proceeding with treatment.
* Voluntary written consent signed before performance of any study related procedure not part of the normal medical care.

Exclusion Criteria

* Biphenotypic leukemia
* New or progressive pulmonary infiltrates on screening chest x-ray or chest computed tomography (CT) scan that has not been evaluated with bronchoscopy, if feasible. Infiltrates attributed to infection must be stable/improving (with associated clinical improvement) after 1 week of appropriate therapy (2 weeks for presumed or documented fungal infections). Surgical resection waives any waiting requirements.
* Uncontrolled bacterial or viral infections. Chronic asymptomatic viral hepatitis is allowed.
* Known hypersensitivity to any of the study agents used
* Received other investigational drugs within the 14 days before enrollment

Donor Selection:

* 12-75 years of age
* \> 40 kilogram body weight
* In general good health as determined by the evaluating physician
* Donors must be HLA-A, B, DRB1 haploidentical (3-5/6 antigens HLA, A, B, DRB1) match to recipient. Patients and donors will be typed for HLA-A, B and C using at least intermediate resolution DNA techniques for DRB1 at high (allele) resolution. KIR B genotyping will be done on all haploidentical donors, and when feasible, the donor with the most favorable KIR gene profile will be used.
* Able and willing to have up to 4 separate apheresis collections per formed
* Not pregnant
* Human immunodeficiency virus (HIV): HIV-1, HIV-2 negative; HTLV-1, HTLV-2 negative, Hepatitis B and C negative
* Voluntary written consent
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 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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Sarah Cooley, MD

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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UMN-MT2003-23

Identifier Type: OTHER

Identifier Source: secondary_id

UMN-IRB-0405M60481

Identifier Type: OTHER

Identifier Source: secondary_id

2004LS042

Identifier Type: -

Identifier Source: org_study_id