This phase II trial studies how well a donor stem cell transplant with alpha/beta T cell and CD19+ B cell depleted stem cells works in treating patients with primary immunodeficiencies. Sometimes the transplanted cells from a donor can attack the body's normal cells (called graft-versus-host disease). Removing the T cells from the donor cells before the transplant may stop this from happening. B cells sometimes can contribute to development of a virus that leads to enlarged lymph nodes. This is known as Epstein Bar virus associated lymphoproliferative disorder. Removing B cells before the transplant may stop this from happening. A donor stem cell transplant with alpha/beta T cell and CD19+ B cell depleted stem cells may reduce some of the complications of the transplant and decrease the time it takes for the new stem cells to establish a new immune system.
Study sponsor and potential other locations can be found on ClinicalTrials.gov for NCT02990819.
PRIMARY OBJECTIVES:
I. To determine event free survival and stable engraftment > 20% donor cells at one year for patients with primary immunodeficiencies (PID) who receive unrelated or partially matched related donor peripheral stem cell grafts with conditioning which have been alpha beta T depleted and CD19 depleted.
SECONDARY OBJECTIVES:
I. Evaluation of incidence and severity of acute and chronic graft versus (vs) host disease (GVHD) at one and two years.
II. Evaluation of incidence of mixed chimerism (for patients who receive conditioning) at one and two years. III. Evaluation of primary and secondary graft rejection at one and two years.
IV. Evaluation of immune reconstitution at one and two years.
OUTLINE:
Patients receive standard of care conditioning regimen comprising busulfan, fludarabine, thiotepa or cyclophosphamide, and anti-thymocyte globulin on days -7 to -2 or days -9 to -2. Patients then undergo allogeneic hematopoietic stem cell transplant by receiving alpha/beta T cell and CD19+ B cell depleted stem cells intravenously (IV) on day 0. Patients who are positive for Epstein-Barr virus (EBV) also receive rituximab on day 1.
After completion of study treatment, patients are followed up for 2 years.
Lead OrganizationChildren's Hospital of Philadelphia
Principal InvestigatorTimothy Steven Olson
