The protocol is designed to provide access to patients to the Clinimacs® CD34 TCRαβ system to prepare cells for an unlabeled indication using an HLA-compatible related or unrelated donor for allogenic transplant.
Allogeneic hematopoietic stem cell transplant (HSCT) is the only known curative therapy for a variety of non-malignant and malignant disorders1-4. Only about 25% of patients have an HLA-matched sibling and in about 16-75% (depending on ethnicity), a 10/10 HLA-matched unrelated donor can be identified. Engraftment failure is a dynamic complex process that may occur with this product due to unbalanced depletion of T-cells between recipient and donor, not only due to low number of CD34 cells. The use of mis-matched or haploidentical donors is associated with a higher risk of post-transplant mortality due to the development of severe graft versus host disease, graft failure and the development of infection due to the use of immunosuppression or T cell depletion. A total of 10 patients will be enrolled. The patients will be divided into 2 strata (haploidentical 7, unrelated donor 3).
Study Type
EXPANDED_ACCESS
CliniMACS System uses an automated antibody/magnetic beads column system that allows efficient depletion of TCRαβ and CD19+ cells without relevant loss of cell viability. Hematopoietic stem cells are retained in sufficient numbers to permit engraftment and hematopoietic reconstitution. Humanitarian Exemption IDE approval has been obtained for CD34+ cell selection as a method of T-cell depletion.
Children's Medical Center - Dallas
Dallas, Texas, United States
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