Donor Stem Cell Transplant, Pentostatin, and Total-Body Irradiation in Treating Patients With Hematological Cancer

NCT ID: NCT00816413

Last Updated: 2023-08-14

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WITHDRAWN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

2008-09-30

Study Completion Date

2010-02-28

Brief Summary

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RATIONALE: Giving low doses of chemotherapy and total-body irradiation before a donor 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. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Removing the T cells from the donor cells before transplant and giving cyclosporine and mycophenolate mofetil after transplant may stop this from happening.

PURPOSE: This phase I/II trial is studying the side effects of giving a donor stem cell transplant after pentostatin and total-body irradiation and to see how well it works in treating patients with hematological cancer.

Detailed Description

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OBJECTIVES:

Primary

* To determine the safety of pentostatin and low-dose total body irradiation followed by T-cell-reduced unrelated donor peripheral blood stem cell transplantation, in terms of regimen-related toxicity, in patients with hematological malignancies.
* To evaluate the efficacy of this regimen, measured as engraftment rate and establishment of donor hematopoietic chimerism, in these patients.

Secondary

* To determine the incidence of acute and chronic graft-versus-host disease in patients treated with this regimen.

OUTLINE:

* Reduced-intensity preparative regimen: Patients receive pentostatin IV over 30 minutes once daily on days -10 to -8 and undergo low-dose total-body irradiation on day -1.
* Unrelated donor peripheral blood stem cell transplantation (PBSCT): Patients undergo T-cell-reduced donor PBSCT on day 0.
* Graft-versus-host disease (GVHD) prophylaxis: Patients receive cyclosporine IV over 2 hours twice daily on days -1, 0, and 1 and then orally twice daily on days 2-70 followed by a taper in the absence of GVHD. Patients also receive oral mycophenolate mofetil twice daily on days 0-27 followed by a taper.

Patients undergo bone marrow aspirate and biopsies and blood sample collection periodically for laboratory studies. Samples are analyzed for cytokines (i.e., IL-6, TNF-γ, IL-1β, and IL-10) by ELISA; phenotypic, molecular, and functional analysis of immunologic reconstitution markers (i.e., PHA, IL-2, IL-4, IL-10, IL-12, Fas, FasL, TNF, TGF-β, and IFN-γ) by flow cytometry; and cytogenetics by FISH.

After completion of study treatment, patients are followed periodically.

Conditions

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Chronic Myeloproliferative Disorders Leukemia Lymphoma Myelodysplastic Syndromes Nonmalignant Neoplasm

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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Donor Stem Cell Transplant, Pentostatin & Total-Body Irradiation for Hematological Cancer

This phase I/II trial is studying the side effects of giving a donor stem cell transplant after pentostatin and total-body irradiation and to see how well it works in treating patients with hematological cancer.

Group Type EXPERIMENTAL

cyclosporine

Intervention Type DRUG

2 mg/kg IV over 2 hours every 12 hours starting at 0700 on days -1, 0, and +1 (total of six doses).

mycophenolate mofetil

Intervention Type DRUG

15 mg/kg orally twice a day starting day 0 until day +27 then stopped without tapering in the absence of aGVHD then tapered over two months in the absence of aGVHD. Doses will be rounded to the nearest 250 mg.

pentostatin

Intervention Type DRUG

4 mg/m2/d IV over 30 minutes daily x 3 days, (days -10, -9, -8)to begin ten days prior to stem cell infusion (Day 0).

cytogenetic analysis

Intervention Type GENETIC

At days +28 and +70 post-transplant, patients' blood will be evaluated for CD3 and overall WBC chimerism.

fluorescence in situ hybridization

Intervention Type GENETIC

Mixed chimerism is defined as the detection of 95% or less donor T cells (CD3+), expressed as a proportion of the total T cell and WBC population as measured by DNA.

protein analysis

Intervention Type GENETIC

Blood samples will be at baseline(day -11 or before) , days -8 and -1, prior to transplant on day 0, then weekly (days +7, +14, +21 and +28) post-transplant through day +28. Five ml's of blood will be drawn at each collection through a central line using heparinized vaccutainers. Samples will be spun down at 1200 g and plasma aspirated and stored in -80 oC until analyzed for the ELISA Assays being done for research purposes in this protocol.

flow cytometry

Intervention Type OTHER

Peripheral blood Peripheral blood flow cytometry for immunophenotyping including Th/c1 and Th/c2 subsets, mitogens (PHA, PWM), NK and LAK function studies, DCs, apoptosis assay of tumor cells, and lymphocyte subsets (CD4, CD8, CD19, CD56

immunoenzyme technique

Intervention Type OTHER

Peripheral blood for immunophenotyping including Th/c1 and Th/c2 subsets, mitogens (PHA, PWM), NK and LAK function studies, DCs, apoptosis assay of tumor cells, and lymphocyte subsets (CD4, CD8, CD19, CD56

laboratory biomarker analysis

Intervention Type OTHER

Donor mononuclear cells from the stem cell product (SCP) obtained with apheresis will be analyzed for surface markers, including CD3, CD4, CD8, CD19, CD14, CD56, TCR+CD8+ cells, Th/c1 and Th/c2, Fas and FasL, and DCs, as well as for apoptosis

reduced-intensity transplant conditioning procedure

Intervention Type OTHER

Allopurinol 300 mg orally once daily x 10 days, to begin one day prior to treatment with Pentostatin (day -11 to day -2) .

Pentostatin 4 mg/m2/d IV over 30 minutes daily x 3 days, (days -10, -9, -8)to begin ten days prior to stem cell infusion (Day 0) .

Pre and Post Pentostatin Hydration: 1000ml Normal Saline IV over 2 hours pre each dose of Pentostatin and 1000ml Normal Saline IV over 4 hours post each dose of Pentostatin on days -10, -9, -8.

Pre Pentostatin Recommended Antiemetics: Ondansetron 32 mg IV over 30 minutes to be given 30 min. before each dose of Pentostatin on days -10, -9, -8. Alternative ondansetron equivalent or other ancillary antiemetics may be used at the discretion of the treating physician.

Pred Forte Eye Drops: two drops in each eye every 4 hours while awake x 7 days after starting Pentostatin (day -10 to day -4).

nonmyeloablative allogeneic hematopoietic stem cell transplantation

Intervention Type PROCEDURE

Each patient will receive up to four IV infusions administered over 30 minutes of donor T cells at increasing cell doses given at intervals.

peripheral blood stem cell transplantation (PBSC)

Intervention Type PROCEDURE

PBSC are infused intravenously either by infusion or IV push on day 0 through a secure intravenous access (i.e. the double-lumen central catheter place pre transplant) according to institutional guidelines.

total-body irradiation

Intervention Type RADIATION

2.0 GY will be administered on day -1. Total-body irradiation (TBI) will consist of 2.0 GY at 8-12cGy/min via 6MV photons delivered AP/PA fields, without lung blocks or via lateral fields with lucite compensator along the head and neck region. TLD (thermal luminescent dosimetry) will be used to verify dose uniformity. It is anticipated that TBI will be given on day -1; however, the timing of TBI administration may be altered by factors beyond the control of the Principal Investigator because of the delivery of unrelated donor stem cells.

Interventions

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cyclosporine

2 mg/kg IV over 2 hours every 12 hours starting at 0700 on days -1, 0, and +1 (total of six doses).

Intervention Type DRUG

mycophenolate mofetil

15 mg/kg orally twice a day starting day 0 until day +27 then stopped without tapering in the absence of aGVHD then tapered over two months in the absence of aGVHD. Doses will be rounded to the nearest 250 mg.

Intervention Type DRUG

pentostatin

4 mg/m2/d IV over 30 minutes daily x 3 days, (days -10, -9, -8)to begin ten days prior to stem cell infusion (Day 0).

Intervention Type DRUG

cytogenetic analysis

At days +28 and +70 post-transplant, patients' blood will be evaluated for CD3 and overall WBC chimerism.

Intervention Type GENETIC

fluorescence in situ hybridization

Mixed chimerism is defined as the detection of 95% or less donor T cells (CD3+), expressed as a proportion of the total T cell and WBC population as measured by DNA.

Intervention Type GENETIC

protein analysis

Blood samples will be at baseline(day -11 or before) , days -8 and -1, prior to transplant on day 0, then weekly (days +7, +14, +21 and +28) post-transplant through day +28. Five ml's of blood will be drawn at each collection through a central line using heparinized vaccutainers. Samples will be spun down at 1200 g and plasma aspirated and stored in -80 oC until analyzed for the ELISA Assays being done for research purposes in this protocol.

Intervention Type GENETIC

flow cytometry

Peripheral blood Peripheral blood flow cytometry for immunophenotyping including Th/c1 and Th/c2 subsets, mitogens (PHA, PWM), NK and LAK function studies, DCs, apoptosis assay of tumor cells, and lymphocyte subsets (CD4, CD8, CD19, CD56

Intervention Type OTHER

immunoenzyme technique

Peripheral blood for immunophenotyping including Th/c1 and Th/c2 subsets, mitogens (PHA, PWM), NK and LAK function studies, DCs, apoptosis assay of tumor cells, and lymphocyte subsets (CD4, CD8, CD19, CD56

Intervention Type OTHER

laboratory biomarker analysis

Donor mononuclear cells from the stem cell product (SCP) obtained with apheresis will be analyzed for surface markers, including CD3, CD4, CD8, CD19, CD14, CD56, TCR+CD8+ cells, Th/c1 and Th/c2, Fas and FasL, and DCs, as well as for apoptosis

Intervention Type OTHER

reduced-intensity transplant conditioning procedure

Allopurinol 300 mg orally once daily x 10 days, to begin one day prior to treatment with Pentostatin (day -11 to day -2) .

Pentostatin 4 mg/m2/d IV over 30 minutes daily x 3 days, (days -10, -9, -8)to begin ten days prior to stem cell infusion (Day 0) .

Pre and Post Pentostatin Hydration: 1000ml Normal Saline IV over 2 hours pre each dose of Pentostatin and 1000ml Normal Saline IV over 4 hours post each dose of Pentostatin on days -10, -9, -8.

Pre Pentostatin Recommended Antiemetics: Ondansetron 32 mg IV over 30 minutes to be given 30 min. before each dose of Pentostatin on days -10, -9, -8. Alternative ondansetron equivalent or other ancillary antiemetics may be used at the discretion of the treating physician.

Pred Forte Eye Drops: two drops in each eye every 4 hours while awake x 7 days after starting Pentostatin (day -10 to day -4).

Intervention Type OTHER

nonmyeloablative allogeneic hematopoietic stem cell transplantation

Each patient will receive up to four IV infusions administered over 30 minutes of donor T cells at increasing cell doses given at intervals.

Intervention Type PROCEDURE

peripheral blood stem cell transplantation (PBSC)

PBSC are infused intravenously either by infusion or IV push on day 0 through a secure intravenous access (i.e. the double-lumen central catheter place pre transplant) according to institutional guidelines.

Intervention Type PROCEDURE

total-body irradiation

2.0 GY will be administered on day -1. Total-body irradiation (TBI) will consist of 2.0 GY at 8-12cGy/min via 6MV photons delivered AP/PA fields, without lung blocks or via lateral fields with lucite compensator along the head and neck region. TLD (thermal luminescent dosimetry) will be used to verify dose uniformity. It is anticipated that TBI will be given on day -1; however, the timing of TBI administration may be altered by factors beyond the control of the Principal Investigator because of the delivery of unrelated donor stem cells.

Intervention Type RADIATION

Other Intervention Names

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Gengraf, Neoral, and Sandimmune CellCept Nipent

Eligibility Criteria

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Inclusion Criteria

Acute myeloid leukemia (AML) meeting any of the following criteria:

Antecedent hematologic disorder Therapy related Primary induction failure

In first complete remission (CR1) with poor-risk cytogenetics, as defined by the following:

del(5q)/-5 del(7q)/-7 abn(3q) t(6;9) del(20q) del(17p)

+13 Complex karyotype t(9;22) = 11q23 rearrangement In second complete remission (CR2) or greater

Acute lymphoblastic leukemia meeting any of the following criteria:

In CR1 with WBC \> 50,000/mm³ at diagnosis

In CR1 with poor-risk cytogenetics (i.e., t\[9;22\], t\[1;19\], t\[4;11\]) AND meets at least 1 of the following criteria:

19-75 years of age AND received prior high-dose chemotherapy, total-body irradiation (TBI), or a radiation dose that precludes administration of 12 Gy of TBI = 50-75 years of age = 19-75 years of age with hematopoietic stem cell transplantation (HSCT) comorbidity index ≥ 3 CNS or testicular involvement at diagnosis No CR within 4 weeks of initial treatment Primary induction failure In CR2 or greater

Myelodysplastic syndromes meeting the following criteria:

Intermediate-2 or high-risk category as determined by International Prognostic Scoring System Not considered a candidate for intensive or standard chemotherapy or HSCT

Chronic myelogenous leukemia meeting any of the following criteria:

First chronic phase AND \< 40 years of age First chronic phase AND no hematologic response after 3 months of imatinib mesylate therapy First chronic phase AND never achieved a complete cytogenetic response during imatinib mesylate therapy First chronic phase AND loss of previously documented response Accelerated phase Blast crisis phase Chronic myeloproliferative disorder (i.e., polycythemia vera, essential thrombocythemia, myelofibrosis) Bone marrow blasts \> 5% and/or other evidence of progression to acute leukemia Chronic myelomonocytic leukemia Severe aplastic anemia Failed prior antithymocyte globulin and cyclosporine immunosuppressive therapy

Mantle cell lymphoma meeting any of the following criteria:

In CR1 In first partial remission (PR1) In CR2 or greater In second PR (PR2) or greater

Indolent non-Hodgkin lymphoma OR chronic lymphocytic leukemia meeting either of the following criteria:

In CR2 or greater In PR2 or greater Lymphoblastic lymphoma In CR1 or greater

Must have minimal residual disease as defined by either of the following:

No more than 5% blasts in blood and/or bone marrow (in patients with acute leukemia/MDS) No bulky adenopathy (\> 5 cm masses) and/or \< 20% bone marrow involvement by lymphoma (in patients with lymphoma) No progressive disease within 8 weeks of most recent prior therapy OR within 12 weeks of prior autologous HSCT No active CNS malignancy (i.e., known positive CSF cytology or parenchymal lesions visible by CT scan or MRI) HLA-matched unrelated peripheral blood stem cell donor available Meets the University of Nebraska Medical Center's or the National Marrow Donor Program's criteria for donors Matched at 7/8 or 8/8 HLA-A, B, C, or DRβ1 loci by molecular typing If match is not at allele level, suitability for donation requires discussion with and approval by the principal investigator Not an identical twin Karnofsky performance status 60-100% Creatinine clearance ≥ 55 mL/min Total bilirubin ≤ 2 times upper limit of normal (ULN) (unless due to Gilbert's disease or malignancy) ALT and AST ≤ 4 times ULN DLCO ≥ 40% FEV1/FVC ratio ≥ 50% of predicted Cardiac ejection fraction ≥ 40%Prior cytoreductive chemotherapy or irradiation to areas of bulky disease allowed, as determined by the primary physician in consultation with the study investigators
Minimum Eligible Age

19 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Cancer Institute (NCI)

NIH

Sponsor Role collaborator

University of Nebraska

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Robert Bociek, MD

Role: PRINCIPAL_INVESTIGATOR

University of Nebraska

Other Identifiers

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P30CA036727

Identifier Type: NIH

Identifier Source: secondary_id

View Link

0164-07-FB

Identifier Type: -

Identifier Source: org_study_id

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