Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders (MASCI)
Severe Combined Immunodeficiency Disease, Severe Primary Immunodeficiency Disorder, Undefined T Cell Deficiency Disorder
About this trial
This is an interventional treatment trial for Severe Combined Immunodeficiency Disease focused on measuring Severe Combined Immunodeficiency Disease, Severe Primary Immunodeficiency Disorder, Undefined T cell Deficiency Disorder, Wiskott-Aldrick Syndrome, Allogeneic stem cell transplant, Fludarabine, monoclonal antibodies
Eligibility Criteria
Inclusion Criteria:
- Patients with a diagnosis of: Severe combined immunodeficiency disease
This includes patients whose SCID is characterized by gene specific mutations as well as patients with clinically severe combined immunodeficiency without a defined genetic cause in which the diagnosis will be determined by a combination of clinical course with lymphocyte quantification and function assays.
OR
Severe primary immunodeficiency disorder, including undefined T cell deficiency disorder, Wiskott-Aldrich syndrome, and other severe immunodeficiencies for which satisfactory conventional therapy does not exist.
- Availability of an HLA mismatched (up to one haplotype) family member or an HLA matched or mismatched (up to one antigen) unrelated donor.
- Creatinine < 2.5 x normal for age.
- Life expectancy greater than 6 weeks
- Lansky/Karnofsky greater than or equal to 70%
Exclusion Criteria:
- Patients with an HLA matched related donor
- Patients with symptomatic cardiac disease, or evidence of significant cardiac disease by echocardiogram (i.e., shortening fraction less than 25%)
- Patients with known allergy to rat serum products
- Patients with a severe infection that on evaluation by the Principal Investigator precludes ablative chemotherapy or successful transplantation
- HIV positive
- Pregnant
Sites / Locations
- Texas Children's Hospital
Arms of the Study
Arm 1
Experimental
Participants With SCID or Primary Immunodeficiency Disorder
all patient will receive an allogeneic transplant with the following conditioning regimen Campath -1H, Fludarabine, Anti-CD45