Chronic Granulomatous Disease Clinical Trial
Official title:
HLA Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease
This study is for patients with chronic granulomatous disease (CGD), which is a disorder of
the immune system that puts them at risk for severe infections. CGD is caused by a genetic
defect that stops or prevents the white blood cells from killing certain bacteria and fungi.
This condition cannot presently be cured by standard treatment with drugs or surgery.
Medicine including antibiotics, antifungals, and interferon gamma, may help some patients
with CGD; however even with continuous treatment, most patients with CGD will have chronic
and recurrent infections. Transfusion of white blood cells may help overcome infection, but
white cell transfusions lead to allergic reactions and fever and the benefit of transfusion
lasts only a matter of hours. Ultimately, chronic infections can damage or injure the body
organs. Injury to the lung or liver can lead to lung or liver failure and death. Medicines
used to treat infection can damage body organs too. Infections may become resistant to
antibiotic or antifungal treatment, and infections not responding to treatment can be deadly.
It is now known that under specific conditions and with special treatment, blood stem cells
(the cells that make blood) can be transplanted from one person to another. Stem cell
transplantation has been done for patients with CGD who have a healthy sibling and who share
the same immune type (HLA type) as the patient. Stem cell transplantation allows healthy or
normal white cells from the stem cell donor to grow or develop in the patient's bone marrow.
These healthy white cells can fight infection and prevent future infections for a patient
with CGD.
Patients on this study will receive stem cells from a related or unrelated donor. The donor
will be closely matched to the patient's immune type but the donor is not a sibling. The
reason this treatment is investigational is that we do not know the likelihood of benefit
that the patient will receive. It is possible that they will have great benefit, like some of
the patients who have been transplanted from a brother or sister. It is possible that the
side-effects of treatment may be too severe so that the transplant won't work.
The purpose of this research study is to evaluate whether or not patients with CGD treated
with a stem cell transplant from a non-matched and/or non-related donor can have a good
outcome from the procedure with an acceptable number of side-effects.
In order to transplant stem cells we will need to give the patient drugs or high-dose
chemotherapy to kill or destroy most of the blood forming and immune cells in the bone
marrow. This is necessary to allow the donor stem cells to live and grow (engraft) in the
bone marrow space. After the drug treatment is completed, the patient will be given the stem
cells from the donor. The drug treatment is as follows:
Day -9 Busulfan
Day -8 Busulfan
Day -7 Busulfan
Day -6 Busulfan
Day -5 Alemtuzumab, Fludarabine, Cyclophosphamide
Day -4 Alemtuzumab, Fludarabine, Cyclophosphamide
Day -3 Alemtuzumab, Fludarabine, Cyclophosphamide
Day -2 Alemtuzumab, Fludarabine, Cyclosporine, Cyclophosphamide
Day -1 REST
Day 0 Stem cell infusion
The day after the chemotherapy treatment is completed, the patient will receive the healthy
stem cells by vein, like a blood transfusion. Once in the bloodstream, the marrow cells will
go to the bone marrow and grow.
It is also possible that if the marrow takes, it will cause a disease known as
graft-versus-host disease (GVHD). To prevent GVHD, we will give the patient cyclosporine and
Methotrexate. Methotrexate will be administered on Days 1, 3, 6 and 11 after the transplant.
The cyclosporine therapy will continue for a longer period of time, however if the patient
does not develop GVHD, it will be discontinued by 6 months after the stem cell transplant.
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