Chronic Granulomatous Disease Clinical Trial
— MUNCHROfficial title:
HLA Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease
Verified date | October 2018 |
Source | Baylor College of Medicine |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 15 |
Est. completion date | November 24, 2017 |
Est. primary completion date | July 31, 2017 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility |
INCLUSION CRITERIA: CGD patients as documented by an abnormal NBT assay in a male patient and/or abnormal NADPH enzyme mutation confirmed by genetic analysis with abnormal NBT. Patients must not have an HLA genotype identical donor. Patients must have a 5/6 or 6/6 HLA-matched unrelated donor or a 5/6 or 6/6 HLA phenotype-matched related donor. Patients must have had at least one serious infection characteristic of those manifested in patients with CGD. Patients must not have active infection. An active infection may include the following: 1) clinical findings consistent with an infection such as fever, cavitary organ lesions, osteomyelitis; 2) progression of presumed infection based upon findings of diagnostic imaging (two or more studies at least 1 month a part). No cumulative organ dysfunction that, in the estimation of the treating physicians, will diminish the patient's likelihood to survive this procedure. Negative pregnancy test for post-pubertal female patients. Echocardiogram shortening fraction >/= 28%. DLCO 50% or greater predicted or FEV1 >/= 50% predicted. EXCLUSION CRITERIA: Active or uncontrolled infection (e.g. lung infection, cavitary organ lesions, osteomyelitis). Markedly elevated C reactive protein or sedimentation rate relative to patient's baseline. Invasive bone or bone marrow disease. Lack of potential hematologic blood product donors in the past (related to McLeod phenotype). |
Country | Name | City | State |
---|---|---|---|
United States | Texas Children's Hospital | Houston | Texas |
Lead Sponsor | Collaborator |
---|---|
Baylor College of Medicine | Texas Children's Hospital |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Percentage of Participants With Engraftment | To estimate the engraftment rate for patients with CGD using busulfan, cyclophosphamide, fludarabine and alemtuzumab (Campath 1H) as conditioning therapy for SCT from 5/6 or 6/6 HLA-matched unrelated or 5/6 or 6/6 HLA phenotype-matched related donors. | 28 days post transplant | |
Secondary | Number of Patients That Have Complete Donor Chimerism After Transplant. | To estimate the likelihood of complete donor chimerism for patients with CGD using busulfan, cyclophosphamide, fludarabine and alemtuzumab (Campath 1H) as conditioning therapy for SCT from 5/6 or 6/6 HLA-matched unrelated or 5/6 or 6/6 HLA phenotype-matched related donors. | 120 days post transplant | |
Secondary | Number of Patients That Have Acute GVHD and Regimen Related Morbidity/Mortality Post Transplant. | To estimate the risk for acute GVHD and regimen related morbidity/mortality for patients with CGD following stem cell transplant from 5/6 or 6/6 HLA matched unrelated or 5/6 or 6/6 HLA phenotype matched related donors. | Assessed between day 0 and day 100 post transplant | |
Secondary | Number of Patients That Have Chronic GVHD and Regimen Related Morbidity/Mortality Post Transplant. | To estimate the risk for chronic GVHD and regimen related morbidity/mortality for patients with CGD following stem cell transplant from 5/6 or 6/6 HLA matched unrelated or 5/6 or 6/6 HLA phenotype matched related donors. | Assessed between day 100 and day 365 post transplant |
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