Lymphoid Hematological Malignancies Clinical Trial
Official title:
Umbilical Cord Blood Transplant for Children With Lymphoid Hematological Malignancies (UCALL)
The purpose of this study is to determine the safety and effectiveness of UCBT to treat
patients with lymphoid hematological diseases and to see if this treatment can decrease the
incidence of leukemia relapse, GVHD and infections.
These patients have a type of blood cell disorder that is very hard to cure. This treatment
that is being used in this trial is known as a stem cell transplant. This treatment might
help the patient live longer without the disease. It uses much stronger doses of drugs and
radiation to kill the diseased cells that could be given without the transplant. We also
think that the healthy cells from the donor may help fight any diseased cells left after the
transplant.
For the transplant to take place, we will administer stem cells from a 'donor' whose cells
best 'match' the patient's. In this study umbilical cords will be the source of the stem
cells. Before the transplant, two very strong drugs plus total body irradiation will be
given to as preconditioning. This treatment will kill most of your blood-forming cells in
the bone marrow. The patient will then get then healthy stem cells.
If the patient has the disease in the central nervous system (CNS), they will receive
radiation to the head and spine before starting the conditioning. This is to try to get
disease control in the CNS. Radiation will not be given for children under 2 yrs old.
Currently, many umbilical cord blood units are available in public banks for transplantation
in patients lacking bone marrow donors. UCB transplants (UCBT) may offer several advantages
over adult bone marrow or peripheral blood stem cell transplants, including:
1. rapid availability,
2. absence of donor risk,
3. low risk of transmissible infectious diseases,
4. low risk of acute GvHD (Graft vs. Host Disease)
The three main causes of death after umbilical cord blood transplantation for these kind of
disorders are graft failure, infection and disease relapse.
In this study we are trying to address these three problems:
To help improve engraftment we will add the drug Fludarabine to Cytoxan and total body
irradiation. Fludarabine is a very strong medicine. We will try to decrease infections and
reduce leukemia relapse by using fludarabine instead of antithymocyte globulin (ATG).
After the eligible criterion for treatment has been met and a suitable UCB stem donor has
been found, the patient will have a central line placed.
Research Therapy:
After placement of the central line, the following chemotherapy will be given to after
admission to the hospital and before the infusion of the umbilical cord blood stem cells:
- 9 - 6 days before the infusion: Total Body Irradiation (TBI) in two fractions ("doses")
per days.
- 5 - 2 days before the infusion: Cytoxan given daily for 4 days, over 1 hour as an
intravenous infusion. Mesna will be given per standards. Mesna is a drug given to
decrease the side effects of Cytoxan. It will be given daily as an intravenous infusion
while the patient receives the Cytoxan.
- 4 - 2 days before the infusion: Fludarabine given daily for 3 days over 1 hour as an
intravenous infusion.
Stem cell transplant (intravenous infusion of the UCB stem cells) - defined as day 0 of the
treatment. All other "numbered" days relate to this infusion date. For example, Day 1 is the
first day after the stem cell transplant.
The following medications will be given to help decrease side effects from the chemotherapy
and UCB infusion: Cyclosporine A (CSA) will be given starting 3 days prior to the stem cell
infusion. It will be given daily over 2 hours every 12 hours, after the infusion and then
tapered if no GVHD is present.
Administration of Mycophenolate mofetil (MMF) will start on the day the stem cell infusion
is completed and will continue daily for 45 days, unless the patient develops GvHD.
Intravenous immunoglobulin's (IVIG) will be given monthly until GVHD therapy is stopped and
there is evidence that the patient's body is producing antibodies.
Study Evaluations: Various study evaluations will be performed before and during the trial.
Follow Up: After year 1, the patient will be asked to return to the clinic once a year for
consultations and bone marrow tests. A follow up bone marrow biopsy and aspirate will be
done 1 and 2 years after transplant. Consultations with specialists will be similar to the
ones the patient had before the transplant.
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Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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