MT2012-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

I'm interested

up to 50 Years old
This study is NOT accepting healthy volunteers
Inclusion Criteria:

• Diagnosis of immunodeficiency or histiocytic disorder including the following:
• Severe combined immunodeficiency (SCID - all variants)
• Second bone marrow transplant (BMT) for SCID (after graft rejection)
• Omenn's Syndrome
• Reticular dysgenesis
• Wiskott-Aldrich syndrome
• Major histocompatibility complex (MHC) Class II deficiency (bare lymphocyte syndrome)
• Hyper IgM Syndrome (CD40 Ligand Deficiency)
• Common variable immunodeficiency (CVID) with severe phenotype
• Chronic Granulomatous Disease (CGD)
• Other severe Combined Immune Deficiencies (CID)
• Hemophagocytic Lymphohistiocytosis (HLH)
• X-linked Lymphoproliferative Disease (XLP)
• Chediak-Higashi Syndrome (CHS)
• Griscelli Syndrome
• Langerhans Cell Histiocytosis (LCH)
• Acceptable stem cell sources include:
• HLA identical or 1 antigen matched sibling donor eligible to donate bone marrow
• HLA identical or up to a 1 antigen mismatched unrelated BM donor
• Sibling donor cord blood with acceptable HLA match and cell dose as per current institutional standards
• Single unrelated umbilical cord blood unit with 0-2 antigen mismatch and minimum cell dose of >5 x 10^7 nucleated cells/kg as per current institutional guidelines
• Double unrelated umbilical cord blood units that are:
• up to 2 antigen mismatched to the patient
• up to 2 antigen mismatched to each other
• minimum cell dose of at least one single unit must be ≥ 3.5 x 10^7 nucleated cells/kg
• combined dose of both units must provide a total cell dose of ≥ 5 x 10^7 nucleated cells/kg
• Age: 0 to 50 years
• Adequate organ function and performance status. Exclusion Criteria
• pregnant or breastfeeding
• active, uncontrolled infection and/or HIV positive
• acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy

Drug: Alemtuzumab 0.3 mg, Drug: Cyclophosphamide, Drug: Busulfan, Biological: Stem Cell Transplantation, Drug: Fludarabine phosphate 40 mg, Drug: Melphalan, Drug: Alemtuzumab 0.2 mg, Drug: Busulfan, Drug: Fludarabine phosphate 30 mg, Drug: MESNA

SCID, Omenn's Syndrome, Reticular Dysgenesis, Wiskott-Aldrich Syndrome, Bare Lymphocyte Syndrome, Common Variable Immunodeficiency, Chronic Granulomatous Disease, CD40 Ligand Deficiency, Hyper IgM Syndrome, X-linked Lymphoproliferative Disease, Hemophagocytic Lymphohistiocytosis, Griscelli Syndrome, Chediak-Higashi Syndrome, Langerhan's Cell Histiocytosis

immunodeficiency disorder, histiocytic disorder, Clinics and Surgery Center (CSC)

Timothy Krepski -
Christen Ebens
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