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Fludarabine and Cyclophosphamide in Treating Patients Who Are Undergoing Donor Stem Cell Transplant for Chronic Lymphocytic Leukemia or Waldenstrom's Macroglobulinemia

Phase 1
Completed
Conditions
Chronic Lymphocytic Leukemia
Interventions
Biological: alemtuzumab
Biological: anti-thymocyte globulin
Biological: filgrastim
Biological: rituximab
Biological: therapeutic allogeneic lymphocytes
Procedure: peripheral blood stem cell transplantation
Radiation: radiation therapy
Registration Number
NCT00281983
Lead Sponsor
German CLL Study Group
Brief Summary

RATIONALE: Giving chemotherapy before a donor bone marrow transplant helps stop the growth of cancer cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. Also, monoclonal antibodies, such as alemtuzumab, can find cancer cells and either kill them or deliver cancer-killing substances to them without harming normal 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 methotrexate, cyclosporine and mycophenolate mofetil after the transplant may stop this from happening. Once the donated stem cells begin working, the patient's immune system may see the remaining cancer cells as not belonging in the patient's body and destroy them (called graft-versus-tumor effect). Giving an infusion of the donor's white blood cells (donor lymphocyte infusion) may boost this effect.

PURPOSE: This phase I/II trial is studying the side effects of giving fludarabine together with cyclophosphamide and to see how well they work in treating patients who are undergoing donor stem cell transplant for B-cell chronic lymphocytic leukemia or Waldenström's macroglobulinemia.

Detailed Description

OBJECTIVES:

Primary

* Determine the feasibility and safety of induction therapy comprising fludarabine and cyclophosphamide followed by allogeneic stem cell transplantation in patients with high-risk B-cell chronic lymphocytic leukemia or lymphoplasmocytic lymphoma (Waldenstrom's macroglobulinemia).

Secondary

* Determine the incidence and kinetics of clinical and molecular remissions in patients treated with this regimen.

* Determine event-free and overall survival of patients treated with this regimen.

* Determine the duration of clinical and molecular remission in relation to the underlying cytogenetic deviation in patients treated with this regimen.

* Determine the kinetics and extent of lympho-hematopoietic donor chimerism in patients treated with this regimen.

OUTLINE: This is a multicenter, open-label, nonrandomized, pilot study.

* Cytoreductive therapy: Patients receive up to 3 courses of cytoreductive therapy comprising fludarabine IV and cyclophosphamide IV on days 1-3 (with or without rituximab IV on day 1). Patients refractory to fludarabine-containing therapy may receive alemtuzumab IV for 12 weeks OR any other cytotoxic salvage regimen for cytoreduction.

* Conditioning regimen: Patients receive 1 of the following conditioning regimens\*:

NOTE: \*Patients who did not achieve partial response after cytoreductive therapy receive regimen 3.

* Regimen 1: Patients receive fludarabine IV and cyclophosphamide IV on days -7 to -3. If stem cells are collected from an unrelated donor, patients also receive anti-thymocyte globulin (ATG) IV on days -4 to -1.

* Regimen 2: Patients undergo total-body irradiation on day -9. Patients then receive alemtuzumab IV on days -8 to -4 and fludarabine IV and cyclophosphamide IV on days -6 to -2.

* Regimen 3: Patients receive fludarabine IV on days -7 to -3, busulfan IV or orally on days -7 to -5, and cyclophosphamide IV on days -3 to -2. If stem cells are collected from an unrelated donor, patients also receive ATG on days -3 to -1.

* Allogeneic peripheral blood stem cell transplantation (PBSCT): Patients undergo allogeneic PBSCT on day 0. Patients receive filgrastim (G-CSF) subcutaneously daily starting on day 5 and continuing until blood count recover.

* Graft-versus-host-disease (GVHD) prophylaxis: Patients receive cyclosporine IV beginning on day -1 and continuing until approximately day 100. Patients treated with conditioning regimen 1 or 3 also receive methotrexate IV on days 1, 3, and 6 OR oral mycophenolate mofetil twice daily on days 0-50. Patients with evidence of residual disease at least 4 weeks after completion of cyclosporine undergo donor lymphocyte infusion (DLI).

* DLI: The donor T-lymphocytes are collected from the PBSCT donor without prior G-CSF mobilization. Patients receive DLI every 8 weeks in the presence of residual disease and the absence of GVHD.

After completion of study treatment, patients are followed periodically.

PROJECTED ACCRUAL: A total of 70 patients will be accrued for this study.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
100
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Allogeneic stem cell transplantationalemtuzumab1. Cytoreductive therapy for inducing a state of partial remission: FC or FC-R or alternative salvage regimens (e.g. Alemtuzumab) 2. Conditioning regimen: FC +/- ATG (Arm A) or FC/Busulfan +/- ATG (Arm C: refractory patients only) 3. allogeneic-PBSCT (from HLA-identical donor) 4. GVHD prophylaxis: CSA + MTX or MMF 5. +/- DLI (Donor lymphocyte infusions)
Allogeneic stem cell transplantationtherapeutic allogeneic lymphocytes1. Cytoreductive therapy for inducing a state of partial remission: FC or FC-R or alternative salvage regimens (e.g. Alemtuzumab) 2. Conditioning regimen: FC +/- ATG (Arm A) or FC/Busulfan +/- ATG (Arm C: refractory patients only) 3. allogeneic-PBSCT (from HLA-identical donor) 4. GVHD prophylaxis: CSA + MTX or MMF 5. +/- DLI (Donor lymphocyte infusions)
Allogeneic stem cell transplantationmethotrexate1. Cytoreductive therapy for inducing a state of partial remission: FC or FC-R or alternative salvage regimens (e.g. Alemtuzumab) 2. Conditioning regimen: FC +/- ATG (Arm A) or FC/Busulfan +/- ATG (Arm C: refractory patients only) 3. allogeneic-PBSCT (from HLA-identical donor) 4. GVHD prophylaxis: CSA + MTX or MMF 5. +/- DLI (Donor lymphocyte infusions)
Allogeneic stem cell transplantationanti-thymocyte globulin1. Cytoreductive therapy for inducing a state of partial remission: FC or FC-R or alternative salvage regimens (e.g. Alemtuzumab) 2. Conditioning regimen: FC +/- ATG (Arm A) or FC/Busulfan +/- ATG (Arm C: refractory patients only) 3. allogeneic-PBSCT (from HLA-identical donor) 4. GVHD prophylaxis: CSA + MTX or MMF 5. +/- DLI (Donor lymphocyte infusions)
Allogeneic stem cell transplantationperipheral blood stem cell transplantation1. Cytoreductive therapy for inducing a state of partial remission: FC or FC-R or alternative salvage regimens (e.g. Alemtuzumab) 2. Conditioning regimen: FC +/- ATG (Arm A) or FC/Busulfan +/- ATG (Arm C: refractory patients only) 3. allogeneic-PBSCT (from HLA-identical donor) 4. GVHD prophylaxis: CSA + MTX or MMF 5. +/- DLI (Donor lymphocyte infusions)
Allogeneic stem cell transplantationfilgrastim1. Cytoreductive therapy for inducing a state of partial remission: FC or FC-R or alternative salvage regimens (e.g. Alemtuzumab) 2. Conditioning regimen: FC +/- ATG (Arm A) or FC/Busulfan +/- ATG (Arm C: refractory patients only) 3. allogeneic-PBSCT (from HLA-identical donor) 4. GVHD prophylaxis: CSA + MTX or MMF 5. +/- DLI (Donor lymphocyte infusions)
Allogeneic stem cell transplantationrituximab1. Cytoreductive therapy for inducing a state of partial remission: FC or FC-R or alternative salvage regimens (e.g. Alemtuzumab) 2. Conditioning regimen: FC +/- ATG (Arm A) or FC/Busulfan +/- ATG (Arm C: refractory patients only) 3. allogeneic-PBSCT (from HLA-identical donor) 4. GVHD prophylaxis: CSA + MTX or MMF 5. +/- DLI (Donor lymphocyte infusions)
Allogeneic stem cell transplantationmycophenolate mofetil1. Cytoreductive therapy for inducing a state of partial remission: FC or FC-R or alternative salvage regimens (e.g. Alemtuzumab) 2. Conditioning regimen: FC +/- ATG (Arm A) or FC/Busulfan +/- ATG (Arm C: refractory patients only) 3. allogeneic-PBSCT (from HLA-identical donor) 4. GVHD prophylaxis: CSA + MTX or MMF 5. +/- DLI (Donor lymphocyte infusions)
Allogeneic stem cell transplantationradiation therapy1. Cytoreductive therapy for inducing a state of partial remission: FC or FC-R or alternative salvage regimens (e.g. Alemtuzumab) 2. Conditioning regimen: FC +/- ATG (Arm A) or FC/Busulfan +/- ATG (Arm C: refractory patients only) 3. allogeneic-PBSCT (from HLA-identical donor) 4. GVHD prophylaxis: CSA + MTX or MMF 5. +/- DLI (Donor lymphocyte infusions)
Allogeneic stem cell transplantationbusulfan1. Cytoreductive therapy for inducing a state of partial remission: FC or FC-R or alternative salvage regimens (e.g. Alemtuzumab) 2. Conditioning regimen: FC +/- ATG (Arm A) or FC/Busulfan +/- ATG (Arm C: refractory patients only) 3. allogeneic-PBSCT (from HLA-identical donor) 4. GVHD prophylaxis: CSA + MTX or MMF 5. +/- DLI (Donor lymphocyte infusions)
Allogeneic stem cell transplantationcyclophosphamide1. Cytoreductive therapy for inducing a state of partial remission: FC or FC-R or alternative salvage regimens (e.g. Alemtuzumab) 2. Conditioning regimen: FC +/- ATG (Arm A) or FC/Busulfan +/- ATG (Arm C: refractory patients only) 3. allogeneic-PBSCT (from HLA-identical donor) 4. GVHD prophylaxis: CSA + MTX or MMF 5. +/- DLI (Donor lymphocyte infusions)
Allogeneic stem cell transplantationcyclosporine1. Cytoreductive therapy for inducing a state of partial remission: FC or FC-R or alternative salvage regimens (e.g. Alemtuzumab) 2. Conditioning regimen: FC +/- ATG (Arm A) or FC/Busulfan +/- ATG (Arm C: refractory patients only) 3. allogeneic-PBSCT (from HLA-identical donor) 4. GVHD prophylaxis: CSA + MTX or MMF 5. +/- DLI (Donor lymphocyte infusions)
Allogeneic stem cell transplantationfludarabine phosphate1. Cytoreductive therapy for inducing a state of partial remission: FC or FC-R or alternative salvage regimens (e.g. Alemtuzumab) 2. Conditioning regimen: FC +/- ATG (Arm A) or FC/Busulfan +/- ATG (Arm C: refractory patients only) 3. allogeneic-PBSCT (from HLA-identical donor) 4. GVHD prophylaxis: CSA + MTX or MMF 5. +/- DLI (Donor lymphocyte infusions)
Primary Outcome Measures
NameTimeMethod
Safety as measured by a treatment-related mortality of < 25% at 2 years following transplant
Feasibility as measured by the proportion of eligible patients completing the transplant procedure successfully
Secondary Outcome Measures
NameTimeMethod
Event-free and overall survival at 5 years following transplant
Clinical remission rate by NIH criteria at 12 months following transplant
Minimal residual disease negativity rate as measured by high-resolution flow or CDR PCR at 12 months following transplant
Chimerism as measured by STR-PCR at 12 months following transplant

Trial Locations

Locations (13)

Charite - Universitaetsmedizin Berlin - Campus Benjamin Franklin

🇩🇪

Berlin, Germany

University Hospital of Leipzig

🇩🇪

Leipzig, Germany

Klinikum der Universitaet Regensburg

🇩🇪

Regensburg, Germany

Comprehensive Cancer Center Ulm at Universitaetsklinikum Ulm

🇩🇪

Ulm, Germany

Medizinische Hochschule Hannover

🇩🇪

Hannover, Germany

University Hospital Schleswig-Holstein - Kiel Campus

🇩🇪

Kiel, Germany

Maisonneuve-Rosemont Hospital

🇨🇦

Montreal, Quebec, Canada

Universitaetsklinikum Goettingen

🇩🇪

Goettingen, Germany

Clinic for Bone Marrow Transplantation and Hematology and Oncology

🇩🇪

Idar-Oberstein, Germany

Universitaetsklinikum Essen

🇩🇪

Essen, Germany

Universitaets-Kinderklinik Heidelberg

🇩🇪

Heidelberg, Germany

Universitaetsklinikum des Saarlandes

🇩🇪

Homburg, Germany

Asklepios Klinik St. Georg

🇩🇪

Hamburg, Germany

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