Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome
About this trial
This is an interventional treatment trial for Primary Immune Deficiency Disorders focused on measuring Severe Combined Immune Deficiency, Congenital T-cell Defect, Congenital T-cell Deficiency, Chronic Granulomatous Disease, Shwachman Diamond Syndrome, Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Sickle Cell Disease, Thalassemia, Mucopolysaccharidosis, Sphingolipidoses
Eligibility Criteria
Inclusion Criteria:
- Patient must meet eligibility criteria for allogeneic transplantation
- Lack of suitable conventional donor (10/10 allele matched related or unrelated donor) or presence of rapidly progressive disease not permitting time to identify an unrelated donor
- Males or females
- Age < 55 years old and > 4 months
- Diagnosis of a nonmalignant disorder considered treatable by HCT.
HLA typing will be performed at high resolution (allele level) for the HLA-A, -B, Cw, DRBl, and DQB1 loci.
i. A minimum match of 5/10 is required. ii. The donor and recipient must be identical, as determined by high resolution typing, in at least one allele of each of the following
- If capable of reproduction, patient must agree to use contraception or abstinence to prevent pregnancy during the first year of enrollment and treatment.
- Informed consent signed by patient (if ≥18 years old) or parent/guardian (if <18 years old).
Fanconi anemia patients ONLY i) Patients must meet one of the following criteria to be eligible for this study:
- Any patient with Fanconi anemia and bone marrow failure involving 2 of the following 3 lineages: granulocyte count <0.5 x 109/L, platelet count <20 x 109/L, or hemoglobin <8 g/dL.
- Any patient with Fanconi anemia who requires red blood cell or platelet transfusions because of marrow failure
- Any patient with Fanconi anemia who has a life-threatening bone marrow failure involving a single hematopoietic lineage.
Exclusion Criteria:
- Serious organ dysfunction
- Pregnant or breast-feeding
- Evidence of HIV infection
- Bovine product allergy
- Patients with an active infectious disease
- Patients with Fanconi anemia with AML/MDS.
Sites / Locations
- Fred Hutchinson Cancer ResearchCenter
Arms of the Study
Arm 1
Experimental
BPX-501 and Rimiducid
Single administration of BPX-501 T cells post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7