Evaluating the Safety and Effectiveness of an Umbilical Cord Blood Stem Cell Transplant (BMT CTN 0604)
|Precursor B-Cell Lymphoblastic Leukemia-Lymphoma Leukemia, Myeloid, Acute Burkitt Lymphoma Lymphoma, B-Cell Lymphoma, Follicular Lymphoma, Large B-Cell, Diffuse||Biological: Hematopoietic Umbilical Cord Blood Stem Cell Transplantation Biological: GVHD prophylaxis||Phase 2|
|Study Design:||Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Multi-Center, Phase II Trial of Non-Myeloablative Conditioning (NST) and Transplantation of Umbilical Cord Blood (UCB) From Unrelated Donors in Patients With Hematologic Malignancies (BMT CTN #0604)|
- Overall Survival at 180 Days From the Time of Transplant [ Time Frame: Measured at Month 6 and Year 1 ]
- Neutrophil Recovery [ Time Frame: Measured at Days 28, 56, 90, and 100 ]Neutrophil recovery is defined as achieving an absolute neutrophil count ≥ 500/mm3 for three consecutive measurements on different days.
- Primary Graft Failure [ Time Frame: Measured at Day 100 ]Primary graft failure is defined as < 5% donor chimerism on all measurements prior to and day-100.
- Secondary Graft Failure [ Time Frame: Measured at Day 100 ]Secondary graft failure is defined initial recovery followed by neutropenia with < 5% donor chimerism.
- Platelet Recovery to 20K [ Time Frame: Measured at Days 56, 90, and 100 ]Platelet recovery is defined as the first day of a minimum of three consecutive measurements on different days such that the patient has achieved a platelet count >20,000/mm3 with no platelet transfusions in the preceding seven days.
- Donor Cell Engraftment [ Time Frame: Measured at Day 56 ]Marrow or Blood Sample. Donor cell engraftment is defined as donor chimerism ≥ 5% on Day ≥ 56 after transplantation. Chimerism should be evaluated on Days ~28, ~56, ~180, and ~365 after transplantation. Chimerism may be evaluated in whole blood or mononuclear fraction.
- Acute Graft-versus-host Disease (GVHD) [ Time Frame: Measured at Day 100 ]
- Chronic GVHD [ Time Frame: Measured at Year 1 ]
- Progression-free Survival [ Time Frame: Measured at Year 1 ]Progression-free survival is defined as the minimum time interval to relapse/ recurrence/progression, to death or to last follow-up.
- Treatment-related Mortality (TRM) [ Time Frame: Measured at 6 months and 1 year ]
- Incidence of Infections [ Time Frame: Measured at Year 1 ]Number of participants that experienced at least one infection.
- Platelet Recovery to 50K [ Time Frame: Measured at Days 56, 90, and 100 ]Platelet recovery is defined as the first day of a minimum of three consecutive measurements on different days such that the patient has achieved a platelet count >50,000/mm3 with no platelet transfusions in the preceding seven days.
|Study Start Date:||December 2008|
|Study Completion Date:||November 2013|
|Primary Completion Date:||April 2011 (Final data collection date for primary outcome measure)|
Experimental: Umbilical Cord Blood Transplantation
Participants will receive a double unit Hematopoietic Umbilical Cord Blood Stem Cell Transplantation using a non-myeloablative preparative regimen, GVHD prophylaxis.
Biological: Hematopoietic Umbilical Cord Blood Stem Cell Transplantation
Biological: GVHD prophylaxis
The transplant preparative regimen is listed below. The - sign is the number of days before the transplant.
GVHD prophylaxis regimen will consist of:
Day 0 is the day of the infusion of the umbilical cord blood graft units, which will be obtained from partially HLA-matched unrelated donors.
Beginning on Day 1, participants will receive G-CSF 5 mcg/kg/day until absolute neutrophil count (ANC) is greater than or equal to 2,000/mm^3 for three consecutive measurements, each on different days.
Leukemia and lymphoma are types of blood cancers. Chemotherapy is a common treatment option for people with these types of cancers, but if the cancer does not respond well to chemotherapy, or if the cancer returns, people may need to consider other options. A bone marrow transplant, which is a type of stem cell transplant in which healthy bone marrow is donated to a patient by a related or unrelated donor, is commonly used to treat leukemia and lymphoma. Recently, stem cell transplants using umbilical cord blood have become a viable option to treat these types of cancers. Traditionally, umbilical cord blood, which is the blood left over in the placenta after a baby is born, has been disposed of with the placenta. However, over the past few years, doctors have begun to collect and freeze the umbilical cord blood cells so that they may be used in stem cell transplant procedures at a later time.
Typically, people who are undergoing a stem cell transplant receive high doses of chemotherapy before the transplant to prepare their bodies to accept the donor stem cells. In this study, participants will undergo a new type of stem cell transplant called a nonmyeloablative transplant, which is a reduced intensity method of transplantation that does not require high doses of chemotherapy. The purpose of the study is to examine the safety and effectiveness of a nonmyeloablative stem cell transplant that uses umbilical cord blood as a treatment option for people with leukemia or lymphoma.
This study will enroll people with leukemia or lymphoma. Participants will be admitted to the hospital and will first receive a type of chemotherapy called cyclophosphamide, which will be given intravenously on the sixth day before the transplant. In addition, another type of chemotherapy, fludarabine, will be given intravenously each day for 5 days before the transplant. Three days before the transplant, participants will receive cyclosporine and mycophenolate mofetil (MMF), to help prevent the body from rejecting the stem cells and to help decrease the risk of developing a complication called graft-versus-host-disease (GVHD), which is an attack by the donor cells on the body's normal tissues. Some participants may receive tacrolimus instead of cyclosporine. After 6 days, participants will receive a small dose of radiation. The next day, participants will undergo the umbilical cord blood stem cell transplant.
Participants will remain in the hospital for approximately 2 to 3 months total, but possibly longer if there are complications. Beginning on the first day after the transplant, participants will receive daily injections of a growth factor called granulocyte-colony stimulating factor (G-CSF), which is a natural protein that increases the white blood cell count; G-CSF will be continued until a participant's white blood cell count is normal again. Participants will continue to receive MMF for 30 days and cyclosporine or tacrolimus for 180 days after the transplant. While participants are in the hospital, blood samples will be collected regularly to evaluate the response and possible side effects to treatment, including GVHD. If necessary, participants will receive platelet and red blood cell transfusions. At follow-up study visits 6 months and 1 year after the transplant, blood samples will be obtained. Study researchers will keep track of participants' medical condition through phone calls or mailings to participants and their doctors once a year for the rest of the participants' lives.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00864227
|United States, California|
|City of Hope National Medical Center|
|Duarte, California, United States, 91010|
|United States, Florida|
|University of Florida College of Medicine, Shands|
|Gainsville, Florida, United States, 32610-3633|
|H. Lee Moffitt Cancer Center|
|Tampa, Florida, United States, 33624|
|United States, Iowa|
|University of Iowa Hospitals and Clinics|
|Iowa City, Iowa, United States, 52242-1009|
|United States, Kansas|
|University of Kansas Hospital|
|Kansas City, Kansas, United States, 66160|
|United States, Massachusetts|
|Dana-Farber Cancer Institute (DFCI), Brigham & Women's Hospital|
|Boston, Massachusetts, United States, 02114|
|Dana-Farber Cancer Institute (DFCI), Massachusetts General Hospital|
|Boston, Massachusetts, United States, 02114|
|United States, Minnesota|
|University of Minnesota|
|Minneapolis, Minnesota, United States, 55455|
|United States, Missouri|
|Washington University, Barnes Jewish Hospital|
|St. Louis, Missouri, United States, 63110|
|United States, New York|
|Weill Cornell Medical College, NY Presbyterian Hospital|
|New York, New York, United States, 10065|
|United States, Ohio|
|Ohio State, Arthur G. James Cancer Hospital|
|Columbus, Ohio, United States, 43210|
|United States, Pennsylvania|
|University of Pennsylvania Cancer Center|
|Philadelphia, Pennsylvania, United States, 19104|
|United States, South Carolina|
|Medical University of South Carolina|
|Charleston, South Carolina, United States, 29425|
|United States, Texas|
|Texas Transplant Institute|
|San Antonio, Texas, United States, 78229|
|United States, Virginia|
|Virginia Commonwealth University, Medical College of Virginia (MCV) Hospital|
|Richmond, Virginia, United States, 23298|
|United States, Wisconsin|
|University of Wisconsin Hospital and Clinics|
|Madison, Wisconsin, United States, 53792-5156|
|Study Director:||Mary Horowitz, MD, MS||Center for International Blood and Marrow Transplant Research (CIBMTR), Medical College of Wisconsin|