Trial Information

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)

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.