Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease (MUNCHR)
Chronic Granulomatous Disease
About this trial
This is an interventional treatment trial for Chronic Granulomatous Disease focused on measuring Stem Cell Transplant, Chronic Granulomatous Disease, Fludarabine, Busulfan, Cyclophosphamide
Eligibility Criteria
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).
Sites / Locations
- Texas Children's Hospital
Arms of the Study
Arm 1
Experimental
Allogeneic unrelated transplant
Conditioning from Day -9 to Day -1. Stem cells given on Day 0. Busulfan, alemtuzumab, cyclophosphamide, fludarabine, cyclosporine, stem cell infusion.