Expanded Access Protocol Using CD3+/CD19+ Depleted Unrelated or Related Donor Peripheral Stem Cells for Hematopoietic Stem Cell Transplant

What this Trial Is About

Researchers are studying a treatment approach for patients who need an allogeneic hematopoietic stem cell transplant (HSCT) but do not have a fully HLA-matched sibling donor. These patients have serious or life-threatening diseases such as leukemia, bone marrow failure syndromes, immunodeficiencies, metabolic diseases, and related disorders where HSCT is indicated. The study aims to expand access to a method that reduces complications associated with using unrelated or partially matched donors. The study uses the CliniMACs device to remove specific T cells (CD3+ and CD19+) from donor stem cells. This T cell depletion process aims to lower the risk of graft versus host disease (GVHD), a common complication when donor cells are not fully matched. Participants receive stem cells processed by this device from either unrelated or related donors who meet specific matching and screening criteria. This is an early phase 1 expanded access protocol designed for patients who are not eligible for other protocols using this technology. Participants will undergo the transplant after screening and consent. Researchers will monitor overall survival, occurrence of graft versus host disease, and graft failure up to one year after transplant. The study includes assessments of hematologic and organ function, infectious evaluations, and pregnancy testing where applicable. The total duration of follow-up includes at least one year post-transplant to evaluate these outcomes and safety.

Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC