Use of Busulfan as Conditioning Agent for a Second Stem Cell Transplant
Status:
Completed
Trial end date:
2010-04-06
Target enrollment:
Participant gender:
Summary
This protocol is designed for a single specific patient. It uses busulfan as a conditioning
agent in a second stem cell transplant procedure for a patient with chronic granulomatous
disease (CGD), a disorder in which a certain type of white cells, called myeloid cells, do
not function properly. This causes increased risk of serious bacterial and fungal infections
that can lead to organ dysfunction, such as kidney disease, as well as formation of
granulomas-non-cancerous masses that can cause obstructions in the esophagus, stomach, and
intestines, and block urine flow from the kidneys and bladder.). The child in this study has
previously undergone a stem cell transplant to treat CGD, and, as a result, he is now
producing normal lymphocytes (another type of white cell). However, the myeloid cells from
the donor did not engraft successfully, and the patient is still producing his own defective
myeloid cells. In this study, the child will undergo a second stem cell transplant in
combination with busulfan, a drug that targets myeloid cells, killing them to make way for
healthy, donated myeloid cells.
Treatment includes the following procedures:
- Medical evaluation to confirm that the patient is healthy enough to undergo the
transplantation
- Treatment with busulfan, injected through the patient's central venous line
- Stem cell transplantation through the central venous line
- Blood tests on days 25, 56, and 91 after the transplant to assess how many cells are of
donor origin
- Bone marrow aspiration on day 100, and then at 12, 24, and 36 months to assess how many
cells are of donor origin
- Pulmonary function (breathing) test at 12 and 24 months
- Physical examination and blood tests, weekly or twice weekly for the first 2 to 3 months
and at 4, 6, 12, 18, 24, 36, 48, and 60 months after transplant
- Treatment for graft-versus-host disease (GVHD), if this complication develops. GVHD is
the attack of lymphocytes from the donor against the patient's own cells. This is good
if it is against abnormal cells, but bad if serious damage occurs to the patient's vital
organs. GVHD is treated with steroids and cyclosporine, and possibly other drugs if
needed.
Phase:
Phase 1
Details
Lead Sponsor:
National Institute of Allergy and Infectious Diseases (NIAID)