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Clinical Trials/NCT00571662
NCT00571662
Completed
Phase 2

Allogeneic Peripheral Blood Stem Cell Transplantation With Minimally Myelosuppressive Regimen of Pentostatin and Low-dose Total-body Irradiation

University of Nebraska1 site in 1 country76 target enrollmentDecember 8, 2000

Overview

Phase
Phase 2
Intervention
Pentostatin
Conditions
Acute Myelogenous Leukemia
Sponsor
University of Nebraska
Enrollment
76
Locations
1
Primary Endpoint
Percent of Participants With Chimerism: Full Donor Chimerism Defined as >95% Donor CD3+ Cell in Blood as Assessed by DNA Fingerprinting
Status
Completed
Last Updated
2 years ago

Overview

Brief Summary

This is a continuation of a pilot study which is now regarded as a phase II trial with a plan to enroll an additional 40 patients (20 related and 20 unrelated donor transplants) with hematological malignancy assessing the safety and efficacy of a minimally myelosuppressive regimen with pentostatin and low-dose total body irradiation (TBI) followed by allogeneic peripheral blood stem cell transplantation (alloPSCT).

Detailed Description

This is a pilot study which began with a plan to enroll 50 patients (20 related and 30 unrelated donor transplants) with hematological malignancy assessing the safety and efficacy of a minimally myelosuppressive regimen with Pentostatin and low-dose total body irradiation (TBI) followed by allogeneic peripheral blood stem cell transplantation (alloPSCT). Patients with persistent or progressive malignancy after transplantation will be treated with GM-CSF (cytokine therapy) to assess its toxicity and potential therapeutic efficacy. Patients with persistent or progressive disease who fail or do not qualify for the cytokine therapy portion of the study will become candidates for donor leukocyte infusions. The purpose of this protocol remains a pilot study which is now regarded as a phase II trial with a plan to enroll 40 ADDITIONAL patients (20 related and 20 unrelated donor transplants) with hematological malignancy assessing the safety and efficacy of a modified version of the original preparative regimen of Pentostatin and low-dose total body irradiation (TBI) followed by allogeneic peripheral blood stem cell transplantation (alloPSCT). Patients who fail will become candidates for donor-leukocyte infusion (DLI). Primary Objectives 1. To determine the safety of treating hematological malignancies by establishing donor hematopoietic chimerism using pentostatin and low-dose total body irradiation followed by allogeneic peripheral blood stem cell transplantation. 2. To determine the immunomodulatory effects of pentostatin as part of the conditioning regimen for allogeneic peripheral blood stem cell transplantation. Secondary Objectives 1. To determine the incidence of infections after using a minimally myelosuppressive conditioning regimen. 2. To determine the kinetics of hematological and immunological reconstitution after allotransplantation with a minimally myelosuppressive conditioning regimen. 3. To determine the incidence of chronic GVHD after using allogeneic peripheral blood stem cell transplantation with a minimally myelosuppressive preparative regimen. 4. To evaluate the role of the preparative regimen and donor source (related versus unrelated) on inflammatory cytokine profiles. 5. To evaluate blood and where possible, biopsy specimens for a recently identified nuclear protein (molecular weight 44/46) in mononuclear cells obtained from study subjects. Interventions, evaluation, and follow up will include: Pentostatin 4 mg/m\^2/d intravenously once a day x 3 days will be administered with 1000 cc NS hydration before and after pentostatin ten days prior to stem cell infusion (days -10, -9, and -8). Total-body irradiation (TBI): TBI 2.0 Gy will be given on day -1. Antiemetics will be given as needed. Patients will receive one liter normal saline over 2 hours pre TBI. A bone marrow biopsy and aspiration with cytogenetics and flow cytometry will be performed on Day +28, Day +70 and 6, 12, 18 and 24 months following the transplant to monitor hematologic recovery. DNA fingerprinting will also be conducted at the same time at 3, 4, 5, 6, 12, 18, and 24 months to determine chimerism.

Registry
clinicaltrials.gov
Start Date
December 8, 2000
End Date
December 30, 2008
Last Updated
2 years ago
Study Type
Interventional
Study Design
Single Group
Sex
All

Investigators

Responsible Party
Sponsor

Eligibility Criteria

Inclusion Criteria

  • Age 19-75 years
  • Patients who relapse after autologous stem cell transplantation.
  • Patients who are candidates for an autologous or conventional allogeneic stem cell transplantation from a disease standpoint but who do not qualify functionally (from the point of view of organ function, or performance status) for a myeloablative protocol.
  • Any patient, where in the opinion of the primary treating oncologist, nonmyleoablative therapy would be the treatment option in the best patients interest providing the patient fits all other eligibility criteria for this protocol.
  • Identification of a matched related or unrelated stem cell donor
  • Acute myelogenous leukemia first complete remission with high-risk cytogenetics\>second complete remission minimal residual disease (\<10% blasts\*). Acute lymphocytic leukemia first complete remission with high-risk cytogenetics \>second complete remission minimal residual disease (\<10% blasts\*). Chronic myelogenous leukemia first chronic phase, accelerated phase (\<10% blasts\*)blast phase with minimal residual disease (\<10% blasts\*)second chronic phase. Chronic lymphocytic leukemia recurrence after the front line regimen (related donor transplant), chemorefractory disease (unrelated donor transplant),T-CLL in partial remission or any minimal residual disease. Myelodysplastic syndromes refractory anemia with or without ringed sideroblasts,RAEB, RAEB-T, and CMML (\< than 10% blasts\*). \*both in peripheral blood and bone marrow
  • Multiple myeloma - after receiving at least one regimen of prior chemotherapy
  • Non-Hodgkin's Lymphomas:
  • Small Lympho(plasma)cytic Lymphoma (B-SLL, B-LPL): recurrence after a front line regimen (related donor transplant), or chemorefractory disease (related or unrelated donor transplant). Follicular Low-Grade Lymphoma, Marginal Zone Lymphomas (splenic, nodal, or extranodal/MALT type): chemorefractory disease or \> 2 prior regimens. Mantle Cell Lymphoma: first complete or partial remission, refractory disease, or failed prior ASCT. Diffuse Large B-cell Lymphoma, Follicular Large cell Lymphoma, Peripheral T-cell Lymphoma, Anaplastic Large Cell Lymphoma: refractory disease, or failed prior ASCT. Burkitt or Acute Lymphoblastic Lymphomas: high-risk disease in remission, chemosensitive persistent or recurrent disease. Cutaneous T-cell Lymphomas: (Mycosis Fungoides, Sezary Syndrome): chemorefractory disease of \> 2 prior regimens
  • Hodgkins Disease: refractory or persistent disease and not candidate for ASCT, or failed prior ASCT.

Exclusion Criteria

  • Age \> 75 years and \< 19 years
  • progressive disease within 8 weeks of prior therapy or within 12 weeks after prior autologous stem cell transplantation
  • Active CNS malignancy (patients with known positive CSF cytology or parenchymal lesions visible by CT or MRI)
  • Fertile men or women unwilling to use appropriate contraceptive techniques during and for 12 months following treatment
  • Females who are pregnant
  • Patients who are HIV seropositive
  • Active uncontrolled infection or immediate life-threatening condition at the time of enrollment
  • Significant Organ dysfunction:
  • Calculated Creatinine Clearance \<55ml/min
  • cardiac ejection fraction \<40%, NYHA class II or greater cardiac disease.

Arms & Interventions

Cohort I

Pentostatin to be administered intravenously on days - 10, -9, and -8 at a dose of 4mg/m2/day

Intervention: Pentostatin

Cohort I

Pentostatin to be administered intravenously on days - 10, -9, and -8 at a dose of 4mg/m2/day

Intervention: Total-body irradiation (TBI)

Cohort I

Pentostatin to be administered intravenously on days - 10, -9, and -8 at a dose of 4mg/m2/day

Intervention: Cyclosporine A (CsA)

Cohort I

Pentostatin to be administered intravenously on days - 10, -9, and -8 at a dose of 4mg/m2/day

Intervention: Mycophenolate Mofetil (MMF)

Cohort I

Pentostatin to be administered intravenously on days - 10, -9, and -8 at a dose of 4mg/m2/day

Intervention: G-CSF

Outcomes

Primary Outcomes

Percent of Participants With Chimerism: Full Donor Chimerism Defined as >95% Donor CD3+ Cell in Blood as Assessed by DNA Fingerprinting

Time Frame: days +28 and +70

the efficacy of the regimen as determined by engraftment rate and establishment of donor hematopoietic chimerism at day +28 and day +70.

Toxicity for the Combination of Pentostatin and Low Dose Total Body Irradiation (TBI)

Time Frame: Conditioning regimen to count recovery (D + 28 post transplant)

Secondary Outcomes

  • Incidence of Acute and Chronic Graft-versus-host Disease(twice weekly until day 100 up to 1 year post transplant)
  • Responses to Therapy(every 6 mo. up to 2 years)
  • Kinetics of Immunologic Reconstitution(at day 100 post transplantation)

Study Sites (1)

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