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Donor Stem Cell Transplant in Treating Patients With High-Risk Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma

Phase 2
Completed
Conditions
Leukemia
Lymphoma
Interventions
Registration Number
NCT01027000
Lead Sponsor
Alliance for Clinical Trials in Oncology
Brief Summary

RATIONALE: Giving low doses of chemotherapy before a donor 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. Also, monoclonal antibodies, such as rituximab, can find cancer cells and either kill them or deliver cancer-killing substances to them without harming normal 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. Giving tacrolimus, sirolimus, and methotrexate after the transplant may stop this from happening.

PURPOSE: This phase II trial is studying how well donor stem cell transplant works in treating patients with high-risk chronic lymphocytic leukemia or small lymphocytic lymphoma.

Detailed Description

OBJECTIVES:

Primary

* To determine if this treatment can improve 2-year current progression-free survival (PFS) in the early disease cohort compared to historical controls. Specifically, we plan to study whether we can achieve 2-year PFS ≥ 70% and to exclude 2 year PFS ≤ 50%

Secondary

* To determine whether in the advanced disease cohort we can achieve 2-year current PFS ≥ 50% and to exclude 2-year PFS ≤ 30%

* To assess objective response rate.

* To assess the incidence of grade 2-4 and 3-4 acute graft-vs-host disease (GVHD).

* To assess the incidence of extensive chronic GVHD.

* To assess the incidence of treatment-related mortality at 100 days and 1 year

* To assess overall survival

* To assess donor chimerism for CD3+ cells at 1 and 2 years after transplantation

* To investigate the presence of donor antigen-specific T-cell clones before and after withdrawal of immune suppression.

* To compare the relapse profiles of patients with T-cell responses against CLL to those whose CLL cells are not reactive

* To prospectively examine the impact of high-risk genomic features and immune-based single nucleotide polymorphisms on response, toxicity, and 2-year PFS to reduced intensity allogeneic stem cell transplant

OUTLINE: This is a multicenter study.

* Preparative regimen: Patients receive 1 of 2 preparative regimens at the discretion of the participating institution.

* Preparative regimen 1: Patients receive rituximab IV on days -7, -1, 7, and 14 and fludarabine phosphate IV over 30 minutes and busulfan IV over 3 hours on days -5 to -2. .

* Preparative regimen 2: Patients receive rituximab IV on days -7, -1, 7, and 14, fludarabine phosphate IV over 30 minutes on days -5 to -2, and cyclophosphamide IV over 1-2 hours on days -5 to -3. Patients with matched unrelated donors also receive anti-thymocyte globulin IV over 4-6 hours on days -6 to -4.

* Graft-vs-host disease (GVHD) prophylaxis: Patients who receive preparative regimen 1 may receive either GVHD prophylaxis regimen 1 or 2; patients who receive preparative regimen 2 may only receive GVHD prophylaxis regimen 2.

* GVHD prophylaxis regimen 1: Patients receive tacrolimus either orally or IV and oral sirolimus beginning on day -2 and continuing until day 60, followed by a taper until day 180. Patients also receive methotrexate IV on days 1, 3, and 6.

* GVHD prophylaxis regimen 2: Patients receive tacrolimus either orally or IV beginning on day -2 and continuing until day 60, followed by a taper until day 180. Patients also receive methotrexate IV on days 1, 3, 6, and 11.

* Transplantation: Patients undergo allogeneic peripheral blood stem cell transplantation on day 0.

* Maintenance therapy: Patients receive rituximab IV at 3, 6, 9, and 12 months after transplantation.

Peripheral blood and bone marrow aspirate samples may be collected periodically for correlative laboratory studies.

Patients are followed up periodically for a maximum of 5 years from study entry.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
68
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Treatment (Combination of chemotherapy and transplant)rituximabSee detailed description
Treatment (Combination of chemotherapy and transplant)allogeneic stem cell transplantSee detailed description
Treatment (Combination of chemotherapy and transplant)busulfanSee detailed description
Treatment (Combination of chemotherapy and transplant)cyclophosphamideSee detailed description
Treatment (Combination of chemotherapy and transplant)fludarabine phosphateSee detailed description
Treatment (Combination of chemotherapy and transplant)methotrexateSee detailed description
Treatment (Combination of chemotherapy and transplant)sirolimusSee detailed description
Treatment (Combination of chemotherapy and transplant)tacrolimusSee detailed description
Primary Outcome Measures
NameTimeMethod
2-year Progression-free Survival in Early Disease Participants2 years post-registration

Percentage of participants who were alive and progression free at 2 years for participants with early disease stage. The 2 year progression free survival, with 95% confidence interval, was estimated using the Kaplan Meier method.

A progression is defined as one of the following events:

* \>= 50% increase in the products of at least two lymph nodes on two consecutive determinations two weeks apart (at least one lymph node must be \>= 2 cm); appearance of new palpable lymph nodes.

* \>= 50% increase in the size of the liver and/or spleen as determined by measurement below the respective costal margin; appearance of palpable hepatomegaly or splenomegaly, which was not previously present.

* \> 50% increase in peripheral blood lymphocytes with an absolute increase \> 5000/μL.

* Transformation to a more aggressive histology (i.e., Richter's syndrome or prolymphocytic leukemia with \>= 56% prolymphocytes).

Secondary Outcome Measures
NameTimeMethod
Treatment-related Mortality6 months post-transplant
Acute Graft-vs-host Disease (GVHD)5 years post-registration
Overall Survival5 years post-registration
Chimerism for CD35 years post-registration
Response5 years post-registration
Chronic GVHD5 years post-registration

Trial Locations

Locations (25)

Cleveland Clinic Taussig Cancer Center

🇺🇸

Cleveland, Ohio, United States

Massachusetts General Hospital

🇺🇸

Boston, Massachusetts, United States

Dana-Farber/Brigham and Women's Cancer Center

🇺🇸

Boston, Massachusetts, United States

Dana-Farber/Harvard Cancer Center at Dana-Farber Cancer Institute

🇺🇸

Boston, Massachusetts, United States

Tunnell Cancer Center at Beebe Medical Center

🇺🇸

Lewes, Delaware, United States

Cancer Institute of New Jersey at Cooper - Voorhees

🇺🇸

Voorhees, New Jersey, United States

CCOP - Christiana Care Health Services

🇺🇸

Newark, Delaware, United States

UNMC Eppley Cancer Center at the University of Nebraska Medical Center

🇺🇸

Omaha, Nebraska, United States

Oklahoma University Cancer Institute

🇺🇸

Oklahoma City, Oklahoma, United States

City of Hope Comprehensive Cancer Center

🇺🇸

Duarte, California, United States

University of Chicago Cancer Research Center

🇺🇸

Chicago, Illinois, United States

Union Hospital of Cecil County

🇺🇸

Elkton, Maryland, United States

Holden Comprehensive Cancer Center at University of Iowa

🇺🇸

Iowa City, Iowa, United States

Siteman Cancer Center at Barnes-Jewish Hospital - Saint Louis

🇺🇸

Saint Louis, Missouri, United States

Monter Cancer Center of the North Shore-LIJ Health System

🇺🇸

Lake Success, New York, United States

Roswell Park Cancer Institute

🇺🇸

Buffalo, New York, United States

CCOP - North Shore University Hospital

🇺🇸

Manhasset, New York, United States

Don Monti Comprehensive Cancer Center at North Shore University Hospital

🇺🇸

Manhasset, New York, United States

Long Island Jewish Medical Center

🇺🇸

New Hyde Park, New York, United States

Arthur G. James Cancer Hospital and Richard J. Solove Research Institute at Ohio State University Comprehensive Cancer Center

🇺🇸

Columbus, Ohio, United States

New York Weill Cornell Cancer Center at Cornell University

🇺🇸

New York, New York, United States

Memorial Sloan-Kettering Cancer Center

🇺🇸

New York, New York, United States

H. Lee Moffitt Cancer Center and Research Institute at University of South Florida

🇺🇸

Tampa, Florida, United States

Florida Hospital Cancer Institute at Florida Hospital Orlando

🇺🇸

Orlando, Florida, United States

Wake Forest University Comprehensive Cancer Center

🇺🇸

Winston-Salem, North Carolina, United States

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