A Study Evaluating BPX-501 T Cells and AP1903 for Prevention of Graft Versus Host Disease (GVHD) After Haploidentical, Related, T Cell-Depleted Hematopoietic Cell Transplantation for Non-Malignant Diseases

BP-003

The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.

2015-02-01

2015-09-01

2018-01-01

Terminated

Early phase I

1

Inclusion Criteria: 1. Patient must meet eligibility criteria for allogeneic transplantation 2. 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 3. Males or females 4. Age \< 55 years old and \> 4 months 5. Diagnosis of a nonmalignant disorder considered treatable by HCT. 6. 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 7. If capable of reproduction, patient must agree to use contraception or abstinence to prevent pregnancy during the first year of enrollment and treatment. 8. Informed consent signed by patient (if ≥18 years old) or parent/guardian (if \<18 years old). 9. Fanconi anemia patients ONLY i) Patients must meet one of the following criteria to be eligible for this study: 1. 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. 2. Any patient with Fanconi anemia who requires red blood cell or platelet transfusions because of marrow failure 3. Any patient with Fanconi anemia who has a life-threatening bone marrow failure involving a single hematopoietic lineage. Exclusion Criteria: 1. Serious organ dysfunction 2. Pregnant or breast-feeding 3. Evidence of HIV infection 4. Bovine product allergy 5. Patients with an active infectious disease 6. Patients with Fanconi anemia with AML/MDS.

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2024-03-25